A promising Stanford Medicine treatment for deadly pediatric brain and spinal cord cancers received a Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) to expedite the FDA approval process. By expediting approval, more children will be able to benefit from the treatment sooner.
The RMAT designation is open to regenerative medicine therapies, such as cell therapy. Researchers must go through a selective admissions process and meet specific criteria, including providing evidence that the therapy can improve patient survival or meet other unmet medical needs for a serious or life-threatening disease or condition. Since the program began in 2017, 128 therapies have been selected for the designation out of the 279 projects invited to apply.
The designation gives researchers access to regulatory experts to refine and improve their research, making the FDA approval process more efficient. Experts may work with researchers on creating flexible, innovative clinical trial designs, such as comparing several investigational drugs; identifying an adequate study population and sample size; and determining appropriate endpoints for various clinical development phases.
Crystal Mackall, MD, Center Director for the Parker Institute for Cancer Immunotherapy at Stanford and co-leader of the Stanford Cancer Institute Cancer Immunotherapy Program, and Stanford Cancer Institute member Michelle Monje, MD, PhD, the Milan Gambhir Professor in Pediatric Neuro-Oncology and professor of neurology at Stanford Medicine, are leading an ongoing clinical trial to evaluate the therapy’s safety and efficacy. Interim results were published in Nature, and Stanford Medicine published an in-depth article about the treatment.
