April 02, 2026
A Stanford Medicine clinical trial has demonstrated that engineered immune cells, known as CAR-T cells, can shrink tumors, restore neurological function, and in one case, eliminate all detectable traces of a deadly childhood brain cancer. The trial focused on children with aggressive brain and spinal cord tumors, including diffuse intrinsic pontine glioma, a condition historically considered universally fatal. The study’s findings were published in Nature on November 13, and in October the therapy received a regenerative medicine advanced therapy designation, allowing for expedited regulatory review.
Among the 11 participants, nine experienced clinical benefits. Functional improvements were observed in nine children, while four had tumor volumes reduced by over 50%. Remarkably, one patient exhibited a complete response, with brain scans showing no residual tumor. Though it is too early to confirm a cure, this child remains healthy four years after diagnosis.
Lead author Michelle Monje, MD, PhD, Milan Gambhir Professor in Pediatric Neuro-Oncology at Stanford Medicine, emphasized the significance: “This is a universally lethal disease for which we’ve found a therapy that can cause meaningful tumor regressions and clinical improvements… I’m hopeful he has been cured.”
The study illustrates the potential of CAR-T cell therapy to overcome some of the most intractable pediatric solid tumors. By demonstrating durable tumor regression and neurological recovery, this research provides a pathway for developing optimized, life-saving immunotherapies for children with historically incurable brain cancers.
SOURCE: https://med.stanford.edu/news/all-news/2024/11/car-t-brain-cancer.html
CREDITS: STANFORD MEDICINE