Researchers report encouraging early results from a first-in-human clinical trial led by Children’s National Hospital using a new T-cell immunotherapy for children and young adults with some of the deadliest brain tumors, including diffuse intrinsic pontine glioma (DIPG) and relapsed central nervous system (CNS) tumors. These findings, published in Nature Medicine, are particularly significant given the challenges of treating pediatric brain tumors, which remain the leading cause of cancer-related deaths in children. Immunotherapies have been shown to work in blood cancers but rarely succeed in solid tumors, especially brain tumors.

“This study represents an important step toward developing safer and more effective T-cell therapies for children with devastating brain cancers,” said Catherine Bollard, MBChB, MD, senior vice president and chief research officer at Children’s National, and co-senior author of the study. “Even in this early-stage trial focused on safety, we were encouraged to see lasting clinical benefit in several patients who otherwise had very few options.”