Chimeric antigen receptor T-cell therapy in neurological disorders: Emerging applications, evidence, and challenges

Author: Jin Jun Luo, Shaun Wang, Xiaohong Si

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Journal of the Neurological Sciences | Chimeric antigen receptor T-cell therapy in neurological disorders: Emerging applications, evidence, and challenges

Article Link: https://doi.org/10.1016/j.jns.2026.125982

Originally developed for blood cancers, chimeric antigen receptor (CAR) T-cell therapy represents a paradigm shift in neurology from lifelong immunosuppression toward a full immune reset.

Early clinical studies and observations show potentially sustained remission in specific antibody-mediated diseases, including myasthenia gravis, multiple sclerosis, and neuromyelitis optica spectrum disorder. However, the utilization of this therapy in immune-mediated neuropathies is still largely theoretical, and the intervention cannot target genetic or metabolic neuromuscular disorders.