Avapritinib shows promise for pediatric high-grade gliomas

Researchers have reported early success using a new therapy called avapritinib to treat high-grade gliomas in children. A team from Dana-Farber Cancer Institute led a clinical test with seven young patients. The study showed that the drug was generally safe and helped reduce tumors in three of them. Pediatric high-grade gliomas are aggressive brain tumors that often have a poor prognosis, with most patients living less than 18 months after diagnosis. Avapritinib targets a specific protein linked to the growth of cancer cells. The team plans to start a larger trial to evaluate this treatment in more patients. Dr. Mariella Filbin from Dana-Farber noted that previous attempts to target the protein were not successful due to the limitations of earlier drugs. Avapritinib is a next-generation treatment designed to overcome these challenges. Initial research indicated that it could reduce tumor growth in laboratory models. The study was conducted in collaboration with other clinical partners and was inspired by the hope of improving patient outcomes. Future research will focus on which genetic changes in tumors can help predict how well patients will respond to avapritinib. Dr. Filbin expressed optimism about finding new treatment options for children facing this difficult disease. She believes that avapritinib could lead to creative combination therapies that work better together.