Cancer Program / en Fri, 01 Aug 25 14:13:45 -0400 Genomics points to potential targeted treatment options for pediatric gliomas /news/genomics-points-potential-targeted-treatment-options-pediatric-gliomas <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Fri, 01 Aug 2025 18:13:45 +0000 tulrich@broadinstitute.org 5559231 at Genomic score predicts patients' progression to multiple myeloma /news/genomic-score-predicts-patients-progression-multiple-myeloma <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Wed, 21 May 2025 21:00:58 +0000 tulrich@broadinstitute.org 5558731 at #WhyIScience Q&A: A computational biologist’s world travels have shaped her approach to research /news/whyiscience-qa-computational-biologists-world-travels-have-shaped-her-approach-research <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Wed, 11 Dec 2024 14:57:24 +0000 Corie Lok 5557961 at Targeted drug treatment leads tumor cells to imitate viral infection /news/targeted-drug-treatment-leads-tumor-cells-imitate-viral-infection <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Thu, 11 Jul 2024 18:15:09 +0000 arinavetta 5557116 at Q&A: New approaches are needed to find better cancer drug targets /news/qa-new-approaches-are-needed-find-better-cancer-drug-targets <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Mon, 22 Jan 2024 15:00:00 +0000 adicorat 5556306 at Bayer and ӳý extend cancer therapy research collaboration /news/bayer-and-broad-institute-extend-cancer-therapy-research-collaboration <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Thu, 02 Nov 2023 14:10:43 +0000 Corie Lok 5555956 at A research team searches for every gene that helps tumors evade immunotherapy /news/research-team-searches-every-gene-helps-tumors-evade-immunotherapy <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Wed, 11 Oct 2023 15:00:00 +0000 adicorat 1282681 at UV radiation plays role in development of rare leukemia in the skin /news/uv-radiation-plays-role-development-rare-leukemia-skin <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Tue, 13 Jun 2023 13:30:27 +0000 tulrich@broadinstitute.org 1282361 at Scientists use genetics to dig into a tumor’s past /news/scientists-use-genetics-dig-tumors-past <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Thu, 20 Apr 2023 15:05:38 +0000 tulrich@broadinstitute.org 1282126 at Lung cancer’s molecular features shed light on immunotherapy response /news/lung-cancers-molecular-features-shed-light-immunotherapy-response <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> <span class="field field--name-uid field--type-entity-reference field--label-hidden"> <span>By Tom Ulrich</span> </span> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" class="datetime">August 1, 2025</time> </span> <div class="hero-section container"> <div class="hero-section__row row"> <div class="hero-section__content hero-section__content_left col-6"> <div class="hero-section__breadcrumbs"> <div class="block block-system block-system-breadcrumb-block"> <nav class="breadcrumb" role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/news">News</a> </li> </ol> </nav> </div> </div> <div class="hero-section__title"> <div class="block block-layout-builder block-field-blocknodelong-storytitle"> <span class="field field--name-title field--type-string field--label-hidden"><h1>Genomics points to potential targeted treatment options for pediatric gliomas</h1> </span> </div> </div> <div class="hero-section__description"> <div class="block block-layout-builder block-field-blocknodelong-storybody"> <div class="clearfix text-formatted field field--name-body field--type-text-with-summary field--label-hidden field__item"><p>Findings suggest that previously-approved precision medicines may benefit children with certain childhood brain tumors.</p> </div> </div> </div> <div class="hero-section__author"> <div class="block block-layout-builder block-extra-field-blocknodelong-storyextra-field-author-custom"> By Beth Dougherty, Dana-Farber Cancer Institute </div> </div> <div class="hero-section__date"> <div class="block block-layout-builder block-field-blocknodelong-storycreated"> <span class="field field--name-created field--type-created field--label-hidden"><time datetime="2025-08-01T14:13:45-04:00" title="Friday, August 1, 2025 - 14:13" class="datetime">August 1, 2025</time> </span> </div> </div> </div> <div class="hero-section__right col-6"> <div class="hero-section__image"> <div class="block block-layout-builder block-field-blocknodelong-storyfield-image"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"> <article class="media media--type-image media--view-mode-multiple-content-types-header"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <picture> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop_xl/public/news/images/2022/DMG.jpeg?itok=fzytTJnT 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="754" height="503"/> <source srcset="/files/styles/multiple_ct_header_desktop/public/news/images/2022/DMG.jpeg?itok=X4LKDbZu 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="736" height="520"/> <source srcset="/files/styles/multiple_ct_header_laptop/public/news/images/2022/DMG.jpeg?itok=GnO45Je- 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="641" height="451"/> <source srcset="/files/styles/multiple_ct_header_tablet/public/news/images/2022/DMG.jpeg?itok=52daQv6W 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="706" height="417"/> <source srcset="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu 1x" media="all and (max-width: 539px)" type="image/jpeg" width="499" height="294"/> <img loading="eager" width="499" height="294" src="/files/styles/multiple_ct_header_phone/public/news/images/2022/DMG.jpeg?itok=lEP_noFu" alt="MRI of a diffuse midline glioma" title="MRI of a diffuse midline glioma" typeof="foaf:Image" /> </picture> </div> <div class="media-caption"> <div class="media-caption__credit"> Credit: Case courtesy of Dr. Jeremy Jones, radiopaedia.org. From the case rID: 68487 </div> <div class="media-caption__description"> MRI of a diffuse midline glioma </div> </div> </article> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block block-better-social-sharing-buttons block-social-sharing-buttons-block"> <div style="display: none"><link rel="preload" href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg" as="image" type="image/svg+xml" crossorigin="anonymous" /></div> <div class="social-sharing-buttons"> <a href="https://www.facebook.com/sharer/sharer.php?u=/taxonomy/term/594/feed&title=" target="_blank" title="Share to Facebook" aria-label="Share to Facebook" class="social-sharing-buttons-button share-facebook" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#facebook" /> </svg> </a> <a href="https://twitter.com/intent/tweet?text=+/taxonomy/term/594/feed" target="_blank" title="Share to X" aria-label="Share to X" class="social-sharing-buttons-button share-x" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#x" /> </svg> </a> <a href="mailto:?subject=&body=/taxonomy/term/594/feed" title="Share to Email" aria-label="Share to Email" class="social-sharing-buttons-button share-email" target="_blank" rel="noopener"> <svg aria-hidden="true" width="32px" height="32px" style="border-radius:100%;"> <use href="/modules/contrib/better_social_sharing_buttons/assets/dist/sprites/social-icons--no-color.svg#email" /> </svg> </a> </div> </div> <div class="block block-layout-builder block-field-blocknodelong-storyfield-content-paragraphs"> <div class="field field--name-field-content-paragraphs field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--text-with-sidebar text-with-sidebar"> <div class="field field--name-field-sidebar field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related people</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/bios/pratiti-mimi-bandopadhayay">Pratiti “Mimi” Bandopadhayay</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-menu sidebar-menu"> <div class="sidebar-menu__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related programs</p> </div> <div class="field field--name-field-links field--type-link field--label-hidden field__items"> <div class="field__item"><a href="/cancer">Cancer Program</a></div> </div> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--sidebar-articles sidebar-articles"> <div class="sidebar-articles__col"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Related news</p> </div> <div class="field field--name-field-content-reference field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><article about="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1921px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2022/FOXR2-image_v2.png?itok=weUeTbiK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/png" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2022/FOXR2-image_v2.png?itok=-LwVBHcy 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/png" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2022/FOXR2-image_v2.png?itok=WmHAUtVW 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/png" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8 1x" media="all and (max-width: 539px)" type="image/png" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2022/FOXR2-image_v2.png?itok=9wxil0u8" alt="Susanna Hamilton, ӳý Communications" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/scientists-identify-key-gene-turned-most-cancer-types" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Scientists identify a key gene that is turned on in most cancer types</span> </a> </div> </article> </div> <div class="field__item"><article about="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node"> <div class="field field--name-field-image field--type-entity-reference field--label-hidden field__item"><article class="media media--type-image media--view-mode-multiple-ct-sidebar-link-with-image"> <div class="field field--name-field-media-image field--type-image field--label-hidden field__item"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape"><picture> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1921px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop_xl/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=_TXkvwgK 1x" media="all and (min-width: 1601px) and (max-width: 1920px)" type="image/jpeg" width="104" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 1340px) and (max-width: 1600px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_desktop/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=OfFQxArz 1x" media="all and (min-width: 800px) and (max-width: 1339px)" type="image/jpeg" width="87" height="104"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_tablet/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=D6SsFx9H 1x" media="all and (min-width: 540px) and (max-width: 799px)" type="image/jpeg" width="285" height="186"/> <source srcset="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe 1x" media="all and (max-width: 539px)" type="image/jpeg" width="220" height="186"/> <img loading="eager" width="220" height="186" src="/files/styles/multiple_ct_sidebar_link_with_image_phone/public/news/images/2020/image001-5.jpg?h=d3e04ee7&itok=o_R_HRBe" alt="Spheroids grown from a high-grade glioma patient-derived cell line" title="Spheroids grown from a high-grade glioma patient-derived cell line" typeof="foaf:Image" /> </picture></a> </div> </article> </div> <div class="node__content"> <a href="/news/massive-study-reveals-how-hypermutated-malignant-brain-tumors-many-mutations-escape" class="node__title"><span class="field field--name-title field--type-string field--label-hidden">Massive study reveals how “hypermutated" malignant brain tumors with many mutations escape chemotherapy and immunotherapy</span> </a> </div> </article> </div> </div> </div> </div> </div> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>A collaborative study between Dana-Farber Cancer Institute and the ӳý found that 8.9 percent of children with glioma, the most common form of pediatric brain tumor, have alterations in the fibroblast growth factor receptor (FGFR) family of proteins, and that these gliomas may be sensitive to existing U.S. Food and Drug Administration (FDA)-approved inhibitors that broadly block FGFR.</p> <p>The work opens the door to potential targeted therapy for these tumors, particularly for pediatric low-grade gliomas (pLGG), though more research is needed to improve the efficacy of these treatments and to test them in pediatric clinical trials. Currently there are no FDA-approved therapies or clinical trials specifically for FGFR-altered pediatric gliomas.</p> <p>“This research was motivated by the patients we see in our Jimmy Fund Clinic and others around the world who have been diagnosed with pediatric gliomas with FGFR-alterations and want to know if existing targeted medicines are an option,” says co-senior author <a href="/bios/pratiti-mimi-bandopadhayay">Pratiti "Mimi" Bandopadhayay</a>, a physician-scientist at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and an institute member in the <a href="/cancer">Cancer Program</a> at ӳý. “The results of this study have given us insights that will impact children with FGFR-altered gliomas seeking care in the clinic today.”</p> <p>The research was published today in <a href="https://www.nature.com/articles/s41467-025-61820-z" target="_blank"><em>Nature Communications</em></a>.</p> <p>Similar research at Dana-Farber led to the recent approval of tovorafenib for childhood BRAF-altered gliomas. “We’re following that same strategy with this research but with a focus on FGFR-altered pediatric gliomas,” says Bandopadhayay. “It is a real area of need for families.”</p> <p>This study, which was also led by Dana-Farber pathologist and ӳý associate member Keith Ligon, performed genomic analyses on 11,635 gliomas from three existing datasets across all ages. The analysis revealed that 8.9 percent of pediatric gliomas harbor FGFR alterations. The most common alterations found were in the FGFR1 and FGFR2 genes and included both point mutations and structural variants. The team concluded that FGFR1-altered pLGGs were the most frequent subgroup of FGFR-altered gliomas in children.</p> <p>Co-first authors and Bandopadhayay lab postdoctoral fellows April Apfelbaum and Eric Morin leveraged the genomic data to develop the first-ever models of FGFR-altered gliomas consisting of spherical collections of living FGFR-altered neural stem cells. They used the models to determine that FGFR alterations can induce the development of the tumors.</p> <p>Tests of FGFR inhibitors in the preclinical models showed that the tumors were sensitive to the inhibitors.</p> <p>“This is the first preclinical data showing that existing FGFR inhibitors might be viable therapeutics for FGFR-altered pediatric gliomas,” says Apfelbaum.</p> <p>They followed their preclinical model tests with a retrospective look at a small number of cases of pediatric patients with FGFR-altered gliomas who were treated with FGFR inhibitors. They found that patients with pLGGs frequently experienced stable disease after treatment with these medicines.</p> <p>pLGGs are the most common central nervous system cancer among children. Patients typically survive into adulthood after treatment because the tumors stop growing over time, but current standard treatment with chemotherapy and surgery result in long-term challenges including mental health disorders, vision changes or loss, seizures, and behavioral challenges.</p> <p>“We are eager to find precision medicines with fewer side effects than current standard of care treatment for these patients,” says Bandopadhayay.</p> <p>The team plans continued research to improve the efficacy and brain penetrance of FGFR inhibitors and to test these medicines in clinical trials. They also plan to investigate their finding that FGFR expression may play a role in normal brain development to determine if that finding should influence treatment strategies.</p> <p><em>Adapted from <a href="https://www.dana-farber.org/newsroom/news-releases/2025/dana-farber-research-shows-promise-for-targeted-therapies-for-pediatric-gliomas">a press release issued by Dana-Farber Cancer Institute</a>.</em></p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro paragraph--view-mode--default"> <div class="field field--name-field-paragraph field--type-entity-reference-revisions field--label-hidden field__items"> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Funding</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Support for this study was provided by the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center Pediatric Low-Grade Glioma Program, the Everest Centre for Low-grade Paediatric Brain Tumours, Team Kermit and the Jared Branfman Sunflowers for Life Fund, Team Brainstorms, Pediatric Brain Tumor Foundation, V Foundation, Team Jack Foundation, Jack’s Drive 55, Helen Gurley Brown Foundation, Damon Runyon-St. Jude Foundation, the Swedish Childhood Cancer Fund, 3000 Miles to the Cure, Else Kröner Excellence Fellowship, the Lady Davis Institute, the Faculty of Medicine and Health Sciences at McGill University, Alex’s Lemonade Stand, the Dietmar Hopp Foundation, the Astro Brain Tumour Fund, and the German Childhood Cancer Foundation.</p> </div> </div> </div> <div class="field__item"> <div class="paragraph paragraph--type--table-outro-row paragraph--view-mode--default"> <div class="clearfix text-formatted field field--name-field-heading field--type-text field--label-hidden field__item"><p>Paper Cited</p> </div> <div class="clearfix text-formatted field field--name-field-text field--type-text-long field--label-hidden field__item"><p>Apfelbaum AA, Morin E, et al. <a href="http://www.nature.com/articles/s41467-025-61820-z" target="_blank">A diverse landscape of FGFR alterations and co-mutations suggests potential therapeutic strategies in pediatric low-grade gliomas</a>. <em>Nature Communications</em>. Online July 31, 2025. DOI: 10.1038/s41467-025-61820-z.</p> </div> </div> </div> </div> </div> </div> </div> </div> </div> </div> <div class="content-section container"> <div class="content-section__main"> <div class="block-node-broad-tags block block-layout-builder block-field-blocknodelong-storyfield-broad-tags"> <div class="block-node-broad-tags__row"> <div class="block-node-broad-tags__title">Tags:</div> <div class="field field--name-field-broad-tags field--type-entity-reference field--label-hidden field__items"> <div class="field__item"><a href="/broad-tags/cancer-program" hreflang="en">Cancer Program</a></div> <div class="field__item"><a href="/broad-tags/cancer" hreflang="en">Cancer</a></div> <div class="field__item"><a href="/broad-tags/pratiti-mimi-bandopadhayay" hreflang="en">Pratiti "Mimi" Bandopadhayay</a></div> </div> </div> </div> </div> </div> Tue, 18 Apr 2023 14:51:33 +0000 kzusi@broadinstitute.org 1282071 at