Activity of PDGFRA inhibitor Avapritinib in H3K27M Diffuse Midline Glioma

ABSTRACT: PDGFRA is commonly altered in pediatric high grade gliomas including in histone 3 lysine 27-mutated diffuse midline gliomas (H3K27M DMG), a disease with almost no long-term survivors. We describe effective CNS penetration of a PDGFRA inhibitor with pharmacologically relevant concentrations in brain tumor tissue resulting in dramatic clinical effect and minimal side effects in a cohort of 9 pediatric high grade glioma cases.

ORGANISM(S): Homo sapiens

PROVIDER: GSE222457 | GEO | 2026/01/01

REPOSITORIES: GEO