Alex's Lemonade Stand Foundation for Childhood Cancer

Neuroblastoma is the most common solid tumor found outside the brain in children and remains one of the deadliest childhood cancers. While new treatments have improved outcomes for some patients, many children still experience relapses, especially those with aggressive forms of the disease. One of the most promising areas of cancer research is immunotherapy, which uses the body’s own immune system to fight cancer. In neuroblastoma, a medicine called anti-GD2 immunotherapy has helped some children live longer, but unfortunately, many tumors eventually return.

Pediatric high-grade gliomas (pHGGs) are highly aggressive, incurable brain tumors that primarily affect children. Our research group previously identified an important growth factor receptor, PDGFRA, which is overactive and causes uncontrolled growth of pHGG cancer cells. We developed a new treatment approach for patients diagnosed pHGG with PDGFRA gene alterations using the small-molecule PDGFRA inhibitor avapritinib. This drug effectively targeted patient-derived pHGG cell lines and pHGG mouse models.