[CAR-T cell therapy for systemic autoimmune rheumatic diseases: a new frontier in immune reset therapy]
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Updated: Oct 30, 2026

Generation of Human Chimeric Antigen Receptor Regulatory T Cells
Published on: January 3, 2025
1Department of Rheumatology and Clinical Immunology, Graduate School of Medicine, Kyoto University.
Abstract:
Chimeric antigen receptor T (CAR-T) cell therapy, originally established for hematologic malignancies, has recently attracted attention as a novel therapeutic strategy for systemic autoimmune rheumatic diseases. By targeting CD19, CAR-T cells induce broad depletion of autoreactive B cells, leading to immune reconstitution. In systemic autoimmune rheumatic diseases, B cells play a central role not only in autoantibody production but also in antigen presentation and cytokine-mediated immune responses. Therefore, comprehensive elimination of B cells is considered an effective approach for disease control. Early clinical studies have demonstrated high remission rates in refractory cases, including systemic lupus erythematosus, with some patients achieving drug-free remission, suggesting deeper disease control beyond that attainable with conventional therapies. In terms of safety, cytokine release syndrome and neurotoxicity are generally milder than those observed in hematologic malignancies. In addition, a characteristic toxicity known as local immune effector cell-associated toxicity syndrome (LICATS) has been reported, presenting as transient inflammation localized to previously affected organs. Future challenges include optimizing CAR-T cell design with an emphasis on safety and controllability, as well as determining the optimal timing of treatment. CAR-T cell therapy holds curative potential and may redefine the treatment paradigm for systemic autoimmune rheumatic diseases.
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