Handbook of clinical neurology · 2026
The future unfolded: CAR T cells and the transformation of treatment algorithms in autoimmune neurology.
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Mougiakakos Dimitrios, Dalakas Marinos C
Аннотация
Chimeric antigen receptor (CAR) T-cell therapy has revolutionized the treatment of hematologic malignancies, and its application in neuroimmunologic autoimmune diseases is now emerging as a promising therapeutic avenue. Neuroimmunologic diseases such as multiple sclerosis, myasthenia gravis, neuromyelitis optica spectrum disorder, and stiff-person syndrome have shown varying degrees of response to traditional immunomodulatory therapies, but a significant proportion of patients remain refractory to treatment. In recent studies, anti-CD19 CAR T cells have shown encouraging results in targeting B cells, a key driver of autoimmune pathogenesis. CAR T-cells can penetrate the central nervous system and overcome the limitations of conventional B-cell depleting therapies such as rituximab, particularly in accessing ectopic lymphoid follicles that maintain compartmentalized inflammation. In early clinical cases, CAR T-cell treatment has resulted in marked clinical improvements, including significant reductions in symptoms and durable disease remission, with manageable side-effects. In addition, advances in allogeneic CAR T cell constructs and chimeric autoantibody receptor T cells offer additional avenues for precision-targeted therapies. These developments underscore the potential of CAR T cells to reshape the treatment landscape for refractory neuroimmunologic autoimmune diseases and warrant further controlled trials and regulatory exploration.