Targeting PDGFRA in high-grade glioma
Platelet-derived growth factor receptor alpha (PDGFRA) is commonly altered in pediatric highgrade glioma (HGG). The researchers detect PDGFRA mutations and/or amplifications in 15% of cases, suggesting PDGFRA as a therapeutic target.
They report that the PDGFRA/ KIT inhibitor avapritinib demonstrates on target activity, brain penetrance, and survival benefit in pre-clinical H3K27M DMG models.
In pediatric HGG patients, avapritinib treatment is well tolerated and results in radiographic and clinical response in a subset of patients.
https://www.cell.com/cancer-cell/fulltext/S1535-6108(25)00070-4
