UCSF Scientists Develop Brain-Targeted Therapy for Deadly Childhood Tumors

Engineered Cells Attack Brain Tumors

Researchers engineered immune cells called B-SYNC CAR-T to remain inactive until they reach the brain. These modified cells completely eliminated diffuse midline glioma in two different mouse models tested by the team. The therapy provided lasting protection against tumor regrowth when cancer cells were re-introduced weeks later. Diffuse midline glioma grows into the brainstem and causes death within a year for nearly all diagnosed children.

Previous Research Led to Breakthrough

Professor Hideho Okada and Professor Wendell Lim built on earlier work with an E-SYNC therapy reported in 2021. That previous treatment used synNotch to identify a mutated protein found on glioblastoma cells before triggering an attack. The new B-SYNC approach limits CAR expression specifically within the brain to avoid side effects elsewhere. Conventional CAR-T treatments have historically struggled to overcome these aggressive brain tumors effectively.

Clinical Trials Begin Soon

The promising laboratory results have prompted scientists to prepare for human clinical trials at UCSF Health. Researchers expect these trials potentially beginning as early as April 2027 to test the therapy in patients. Professor Okada stated that limiting CAR expression where it matters allowed consistent total shutdown of tumors in the lab. This targeted approach offers a new way forward for treating one of the most difficult childhood cancers.