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Avapritinib is FDA approved for stromal tumors and systemic mastocytosis. A collaboration across three institutions has now shown this drug may have promise in high grade glioma (HGG) because it can cross the blood-brain barrier and target PDGFRA mutations, which occur in about 15% of pediatric HGG cases. Based on positive preclinical results, the drug was given to a small group of patients through an expanded access program, and three out of seven had tumor shrinkage. Thanks to these positive real world results, HGG was included as an eligible condition for an ongoing Phase 1 pediatric trial testing the drug in multiple solid tumor types.