CAR-T therapy, short for chimeric antigen receptor T-cell therapy, is already transforming treatment for blood cancers like lymphoma and leukemia and is now being tested in early trials for multiple sclerosis. In this approach, a person’s own T cells (or sometimes donor T cells) are collected and reengineered in the lab with a special “chimeric” receptor on their surface. Once infused back into the body, these “designer” T cells can specifically hunt down B cells, including those hiding in the brain and spinal cord that may drive progressive MS. This is a key difference since current B-cell depleting antibody treatments don’t reach effectively into the central nervous system.

Like any powerful therapy, CAR-T is not without risks. Side effects can include serious infections, cytokine release syndrome (fever, chills, body aches), and even neurological symptoms like confusion or seizures. Still, the early results in MS patients are remarkable, bringing fresh hope for those with progressive disease. Beyond MS, CAR-T is being explored across autoimmune neurological diseases from myasthenia gravis to stiff person syndrome, opening a promising new frontier in care.

Barry Singer MD, Director of the MS Center for Innovations in Care, interviews:

Jeffrey Dunn MD, Clinical Professor and Chief of Neuroimmunology in the Department of Neurology & Neurological Sciences at Stanford University

Amanda Piquet MD, Professor of Neurology and Céline Dion Foundation Endowed Chair at the University of Colorado Anschutz School of Medicine