Chimeric Antigen Receptor T-Cell Therapy in Neuroautoimmune Disease

Abstract

Chimeric Antigen Receptor (CAR) T-cell therapy, originally developed for cancer treatment, is emerging as a promising therapeutic strategy for refractory neuroautoimmune diseases. By selectively targeting and eliminating autoreactive immune cells, particularly B cells, CAR T-cell therapy offers the potential for deeper and more durable remission compared with conventional immunosuppressive treatments. Early clinical evidence in disorders such as multiple sclerosis (MS), myasthenia gravis (MG), and neuromyelitis optica spectrum disorder (NMOSD) has demonstrated encouraging outcomes, including significant reductions in pathogenic antibodies, sustained disease remission, and successful central nervous system penetration with manageable toxicity profiles. Ongoing clinical trials are evaluating various CAR T-cell platforms, including CD19-, BCMA-, and chimeric autoantibody receptor (CAAR)-based approaches, across a broader range of neuroimmunological conditions. Despite these advances, important challenges remain, including treatment-related toxicities, manufacturing complexity, optimal target selection, long-term durability of response, and cost-effectiveness. Overall, CAR T-cell therapy represents a rapidly evolving and potentially transformative approach in neuroimmunology, with the capacity to redefine treatment paradigms for severe autoimmune neurological diseases.