An international team of clinical collaborators, led by physician scientists from Dana-Farber Cancer Institute, performed a first-ever clinical test of the targeted therapy avapritinib in pediatric and young patients with a form of high-grade glioma. They found that the drug, already FDA-approved for certain adult cancers, was generally safe and resulted in tumor reduction visible on brain scans, as well as clinical improvement, in 3 out of 7 patients.
The study was published in Cancer Cell.
Pediatric-type high-grade gliomas are currently incurable brain tumors with median survival times less than 18 months after initial diagnosis.
Avapritinib is a small molecule that crosses the blood-brain-barrier and targets platelet derived growth factor alpha (PDGFRA), which is overactive in some pediatric high-grade…