Two pediatric brain cancers that are challenging to treat if they recur, medulloblastoma and ependymoma, are the target of a clinical trial using a new type of therapy. A multi-institutional, international team led by researchers at Baylor College of Medicine, Texas Children’s Hospital and the Hospital for Sick Children (SickKids) has developed a novel approach that delivers appropriately-targeted chimeric antigen receptor (CAR) T cell therapy directly into the cerebrospinal fluid that surrounds the tumor.
The researchers report in the journal Nature Medicine that this approach was effective in treating these cancers in mouse models of human disease. The findings support further clinical studies to evaluate this strategy to treat pediatric brain cancers, the most common cause of cancer death in childhood. Indeed, a first-in-child clinical trial currently is recruiting patients at Texas Children’s Hospital and Baylor College of Medicine to test the safety and anti-tumor efficacy of this approach….
