ArticleCancer cell2025
Effective targeting of PDGFRA-altered high-grade glioma with avapritinib.
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Article in Cancer cell, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.
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34citing papers in PubMed
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Who cites it
34 citing papers in PubMed.
- The DIPG/DMG National Tumor Board: The power of advocacy.Neuro-oncology pediatrics · 2026Article
- Understanding DMG: current treatment options and prospective solutions using nanoparticles.Drug delivery and translational research · 2026Review
- IDH-Mutant Diffuse Glioma: From Metabolic Origins to Targeted Therapy.Journal of clinical medicine · 2026Review
- Combinatorial Targeting of Avapritinib-Driven MAP Kinase Activation in High-Grade Glioma.Research square · 2026Article
- Delivery of ATSP-7041 by Minimally Invasive Nasal Depot (MIND) to Target Diffuse Intrinsic Pontine Glioma.Molecular cancer therapeutics · 2026Article
- Microglia in diffuse midline glioma contribute to extracellular matrix remodelling and cancer cell invasion.Cell death & disease · 2026Article
- Disrupting MED8-dependent epigenetic reprogramming augments avapritinib sensitivity in PDGFRA-driven glioma.Journal of experimental & clinical cancer research : CR · 2026Article
- Advances in targeted therapies for pediatric tumors.Acta pharmacologica Sinica · 2026Article
- Co-targeting CDK4/6 and MEK reverses mesenchymal transition in therapy-refractory BRAF-altered pediatric high-grade glioma.Journal of experimental & clinical cancer research : CR · 2026Article
- Single-cell transcriptome analysis indicated that immune-related programmed cell death modification features could predict the clinical outcomes of patients with ovarian cancer.Translational oncology · 2026Article
- Systematic design of combination therapy by targeting master regulators of coexisting diffuse midline glioma cell states.Nature genetics · 2026Article
- Diffuse Midline Gliomas: Clinical, Diagnostic, and Therapeutic Perspectives.Biomedicines · 2026Review
- Avapritinib for targeting KIT D816V-mutated acute myeloid leukemia relapsed after allogeneic hematopoietic stem cell transplant with extramedullary and central nervous system involvement.Annals of hematology · 2026Article
- Recent Advances in Targeted Therapies for Adult Gliomas.Current neurology and neuroscience reports · 2026Review
- Mapping kinase-dependent tumor immune adaptation with multiplexed single-cell CRISPR screens.bioRxiv : the preprint server for biology · 2026Article
- Overcoming Chemoresistance in Glioblastoma: Mechanisms, Therapeutic Strategies, and Functional Precision Medicine.International journal of molecular sciences · 2026Review
- The oncogenome of the domestic cat.Science (New York, N.Y.) · 2026Article
- Neutrophil Percentage-to-Albumin Ratio as a Novel Prognostic Biomarker in Adult Diffuse Gliomas: Retrospective Study Integrating 3 Machine Learning Models and Cox Regression.JMIR medical informatics · 2026Article
- Disrupting PDGFRA-driven immune evasion in glioma: vaccine-based strategies on the horizon.Frontiers in oncology · 2026Review
- Single-nucleus transcriptomics of spinal ependymoma subtypes recognizes intratumoral heterogeneity.Neuro-oncology · 2026Article
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Authors and funding
83 authors.
Lisa MayrDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA; Department of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Sina NeyaziDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Kallen SchwarkDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Maria TrissalDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Alexander BeckCenter for Neuropathology and Prion Research, Ludwig Maximilians University Munich, Faculty of Medicine, Muenchen, 80539 Bayern, Germany.
Jenna LabelleDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Sebastian K EderDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA; St. Anna Children's Hospital, Department of Pediatrics and Adolescent Medicine, Medical University of Vienna and St. Anna Children's Cancer Research Institute (CCRI), 1090 Vienna, Austria.
Liesa Weiler-WichtlDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Joana G MarquesDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Carlos A O de Biagi-JuniorDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Costanza Lo CascioDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Owen ChapmanSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Sunita SridharSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Rishaan KenkreSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Aditi DuttaSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Shanqing WangSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Jessica WangSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Olivia HackDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Andrezza NascimentoDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Cuong M NguyenDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Sophia CastellaniDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Jacob S RozowskyDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Andrew GrovesDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Eshini PanditharatnaDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Gustavo Alencastro Veiga CruzeiroDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Rebecca D HaaseDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Kuscha TabatabaiDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Sibylle MadlenerDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Jack WaddenDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Tiffany AdamDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Seongbae KongDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Madeline MicleaDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Tirth PatelDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Katharina BrucknerDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria; Department of Neurosurgery, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Daniel SenfterDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Anna LämmererDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria; Center for Cancer Research and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria; Research Cluster "Translational Cancer Therapy Research", University of Vienna and Medical University of Vienna, 1090 Vienna, Austria.
Jeffrey SupkoDepartment of Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Armin S GuntnerInstitute for Analytical and General Chemistry, Johannes Kepler University, 4040 Linz, Austria.
Hana PalovaCentral European Institute of Technology, Masaryk University, 60177 Brno, Czech Republic.
Jakub NeradilDepartment of Experimental Biology, Faculty of Science, Masaryk University, 61137 Brno, Czech Republic; International Clinical Research Center, St. Anne's University Hospital, Brno, Czech Republic.
Natalia StepienDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Daniela Lötsch-GojoDepartment of Neurosurgery, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Walter BergerCenter for Cancer Research and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria; Research Cluster "Translational Cancer Therapy Research", University of Vienna and Medical University of Vienna, 1090 Vienna, Austria.
Ulrike LeissDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Verena RosenmayrDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Christian DorferDepartment of Neurosurgery, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Karin DieckmannDepartment of Radiotherapy, Medical University of Vienna, 1090 Vienna, Austria.
Andreas PeyrlDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Amedeo A AziziDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Alicia BaumgartnerDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA; Department of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria.
Ondrej SlabyCentral European Institute of Technology, Masaryk University, 60177 Brno, Czech Republic; Department of Biology, Faculty of Medicine, Masaryk University, 62500 Brno, Czech Republic.
Petra PokornaCentral European Institute of Technology, Masaryk University, 60177 Brno, Czech Republic.
Louise M ClarkLurie Family Imaging Center, Center for Biomedical Imaging in Oncology, Dana-Farber Cancer Institute, Boston, MA 02210, USA.
Amy CameronLurie Family Imaging Center, Center for Biomedical Imaging in Oncology, Dana-Farber Cancer Institute, Boston, MA 02210, USA.
Quang-De NguyenLurie Family Imaging Center, Center for Biomedical Imaging in Oncology, Dana-Farber Cancer Institute, Boston, MA 02210, USA.
Hiroaki WakimotoDepartment of Neurosurgery, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.
Frank DuboisDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Noah F GreenwaldBroad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Department of Cancer Biology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02115, USA.
Pratiti BandopadhayayDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Rameen BeroukhimBroad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Department of Cancer Biology and Medical Oncology, Dana-Farber Cancer Institute, and Harvard Medical School, Boston, MA 02115, USA.
Keith LigonDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA.
Christof KrammDivision of Pediatric Hematology and Oncology, University Medical Center Göttingen, 37099 Göttingen, Germany.
Annika BronsemaClinic of Pediatric Hematology and Oncology, University Medical Center Hamburg-Eppendorf, 20251 Hamburg, Germany.
Simon BaileyGreat North Childrens Hospital and Newcastle University, Newcastle upon Tyne, UK; Newcastle Hospitals NHS Foundation Trust, NE1 4LP Newcastle, UK.
Ana Guerreiro StucklinDepartment of Oncology and Children's Research Center, University Children's Hospital Zurich, 8008 Zurich, Switzerland.
Sabine MuellerDepartments of Pediatrics, Neurology, and Neurological Surgery, University of California San Francisco, San Francisco, CA 94143, USA.
Mary SkrypekDepartment of Pediatric Hematology-Oncology, Children's Minnesota, Minneapolis, MN 55404, USA.
Nina MartinezDepartment of Neurology & Neurological Surgery, Jefferson University, Philadelphia, PA 19107, USA.
Daniel C BowersDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.
David T W JonesHopp Children's Cancer Center Heidelberg (KiTZ), 69120 Heidelberg, Germany; Pediatric Glioma Research Group, German Cancer Research Center (DKFZ), 69120 Heidelberg, Germany.
Chris JonesDivision of Molecular Pathology, Institute of Cancer Research, SM2 5NG London, UK.
Natalie JägerHopp Children's Cancer Center Heidelberg (KiTZ) & Division of Pediatric Neurooncology, German Cancer Research Center (DKFZ), 69120 Heidelberg, Germany.
Jaroslav SterbaDepartment of Pediatric Oncology, University Hospital Brno and Faculty of Medicine, Masaryk University, 662630 Brno, Czech Republic.
Leonhard MüllauerDepartment of Pathology, Medical University of Vienna, 1090 Vienna, Austria.
Christine HaberlerDivision of Neuropathology and Neurochemistry, Department of Neurology, Medical University of Vienna, 1090 Vienna, Austria.
Chandan Kumar-SinhaDepartment of Pathology, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Arul ChinnaiyanDepartment of Pathology, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Rajen ModyDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA.
Lukas ChavezSanford Burnham Prebys Medical Discovery Institute, San Diego, CA 92037, USA.
Julia FurtnerDivision of Neuroradiology and Musculoskeletal Radiology, Department of Biomedical Imaging and Image-Guided Therapy, Medical University of Vienna, 1090 Vienna, Austria; Research Center of Medical Image Analysis and Artificial Intelligence, Danube Private University, 3500 Krems an der Donau, Austria.
Carl KoschmannDepartment of Pediatrics, University of Michigan Medical School, Ann Arbor, MI 48109, USA. Electronic address: ckoschma@med.umich.edu.
Johannes GojoDepartment of Pediatrics and Adolescent Medicine, Comprehensive Center for Pediatrics and Comprehensive Cancer Center, Medical University of Vienna, 1090 Vienna, Austria. Electronic address: johannes.gojo@meduniwien.ac.at.
Mariella G FilbinDepartment of Pediatric Oncology, Dana-Farber/Boston Children's Cancer and Blood Disorder Center and Harvard Medical School, Boston, MA 02215, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA. Electronic address: mariella.filbin@childrens.harvard.edu.
Funding
MICHIGAN MEDICAL SCIENTIST TRAINING PROGRAMT32GM007863 · NIGMS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI COLLINS, KATHLEEN L. · 1985 to 2024
$38.3MTargeting the Vasular SystemP50CA165962 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI Samuel Kent McBrayer · 2013 to 2026
$33.5MShared Resource Core 2: Clinical Artificial Intelligence CoreU54CA274516 · NCI · DANA-FARBER CANCER INST · PI DAPHNE A. HAAS-KOGAN · 2023 to 2026
$8.1MHow the 3D Architecture of the Brain Shapes Cancer Cell Fate DecisionsDP2NS127705 · NINDS · DANA-FARBER CANCER INST · PI FILBIN, MARIELLA GRUBER · 2021 to 2024
$2.7MTargeting EGFR/FOXG1-mediated resistance to ONC201 in H3K27M-mutant diffuse midline gliomaR01NS124607 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI Carl J Koschmann · 2022 to 2026
$2.2MThe role of ATRX mutation in the epigenetic dysregulation of cell cycle in pediatric high-grade gliomaR01NS119231 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI KOSCHMANN, CARL J · 2021 to 2025
$1.9MNCI NIH HHS P50 CA165962NCI NIH HHS U54 CA274516NIGMS NIH HHS T32 GM007863NINDS NIH HHS DP2 NS127705NINDS NIH HHS R01 NS119231NINDS NIH HHS R01 NS124607
Abstract
PDGFRA is crucial to tumorigenesis and frequently genomically altered in high-grade glioma (HGG). In a comprehensive dataset of pediatric HGG (n = 261), we detect PDGFRA mutations and/or amplifications in 15% of cases, suggesting PDGFRA as a therapeutic target. We reveal that the PDGFRA/KIT inhibitor avapritinib shows (1) selectivity for PDGFRA inhibition, (2) distinct patterns of subcellular effects, (3) in vitro and in vivo activity in patient-derived HGG models, and (4) effective blood-brain barrier penetration in mice and humans. Furthermore, we report preliminary clinical real-world experience using avapritinib in pediatric and young adult patients with predominantly recurrent/refractory PDGFRA-altered HGG (n = 8). Our early data demonstrate that avapritinib is well tolerated and results in radiographic response in 3/7 cases, suggesting a potential role for avapritinib in the treatment of HGG with specific PDGFRA alterations. Overall, these translational results underscore the therapeutic potential of PDGFRA inhibition with avapritinib in HGG.
Indexed as
Antineoplastic AgentsBrain NeoplasmsGliomaProtein Kinase InhibitorsPyrazolesReceptor, Platelet-Derived Growth Factor alphaAdolescentAdultAnimalsAvapritinibBlood-Brain BarrierCell Line, TumorChildChild, PreschoolFemaleHumansAntineoplastic AgentsAvapritinibProtein Kinase InhibitorsPyrazolesPyrrolesReceptor, Platelet-Derived Growth Factor alphaTriazinesavapritinibbrain penetrancediffuse midline gliomaglioblastomahigh-grade gliomaPDGFRA alterationPDGFRA amplificationPDGFRA inhibitorPDGFRA mutationtyrosine kinase inhibitor
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