Neuro-Oncology • Vol 27 • No Supplement_5
EXTH-143. Preclinical Efficacy of Brain Penetrant, Single Agent Avapritinib in <i>PDGFRA</i> EcDNA-amplified Glioblastoma
November 2025 • Artem Berezovsky, Indrani Datta, Katherine Gurdziel, Andrea Transou, Susan M. Irtenkauf, Laura Hasselbach, Ruicong She, S. Rosenfeld, James M. Snyder…
Abstract The high prevalence of platelet derived growth factor receptor alpha (PDGFRA) alterations and its mitogenic function in oligodendrocyte precursor cells (OPC) make PDGFRA a promising therapeutic target in adult glioblastoma (GBM). Towards validating PDGFRA as an essential driver of GBM tumor maintenance, we have isolated subpopulations from a patient-derived model (HF3253), harboring two alterations in PDGFRA: internal deletion leading to constitutive activation and extrachromosomal (ecDNA) amplification d…