Chimeric Antigen Receptor T-Cell Therapy and Autoimmune Diseases in the Nervous System

Chimeric antigen receptor T-cell (CAR-T) therapy, a revolutionary immunotherapy originally developed for hematologic malignancies, has recently gained attention for its potential in treating autoimmune diseases. Increasing evidence suggests that CAR-T cells can precisely target pathogenic immune populations, offering durable remission and immune homeostasis restoration in neuroimmunological disorders such as myasthenia gravis (MG), neuromyelitis optica spectrum disorder (NMOSD), and multiple sclerosis (MS).

Relevant publications and clinical trial data up to September 2025 were systematically reviewed to summarize the mechanisms, therapeutic targets, safety profiles, and translational applications of CAR-T therapy in autoimmune diseases of the nervous system.

Preclinical and early clinical studies demonstrate that CD19- and BCMA-directed CAR-T therapies effectively deplete autoreactive B cells with significant symptom improvement and minimal cytokine release syndrome or neurotoxicity. Novel constructs such as chimeric autoantibody receptor (CAAR)-T and CAR-regulatory T (CAR-Treg) cells enhance specificity and immune tolerance. Innovations including allogeneic "off-the-shelf" CAR-T, in vivo CAR engineering, and CRISPR-based safety switches further optimize therapeutic potential and accessibility.

CAR-T therapy represents a promising frontier for refractory neuroautoimmune diseases. By precisely modulating immune networks, it offers a pathway toward long-term remission and personalized immunotherapy in clinical neuroimmunology.