Cell子刊：CAR-T细胞疗法又攻克一种自身免疫病

Core Viewpoint - The study demonstrates the safety and potential of BCMA-targeted CAR-T cell therapy in treating refractory chronic inflammatory demyelinating polyneuropathy (CIDP) and provides insights into the molecular mechanisms of disease remission [3][10]. Group 1: CAR-T Cell Therapy Overview - Since 2017, the FDA has approved seven CAR-T cell therapies for treating blood cancers, showing strong therapeutic effects [2]. - CAR-T cell therapy has also shown promising results in treating various autoimmune diseases, including systemic lupus erythematosus and myasthenia gravis [2]. Group 2: CIDP and Treatment Challenges - CIDP is a chronic autoimmune disease affecting the peripheral nervous system, with first-line treatments including intravenous immunoglobulin and steroids [2]. - Approximately 15% of CIDP patients do not respond to current treatment methods [2]. Group 3: Research Findings - The study published in Cell by researchers from Huazhong University of Science and Technology confirmed the safety and potential of BCMA-targeted CAR-T cell therapy in CIDP [3][5]. - The therapy was administered to two patients, both showing manageable safety profiles; one patient experienced disease relapse after 12 months, while the other maintained clinical remission for over 24 months [5][10]. - Multi-omics analysis was conducted on patient samples to understand the molecular mechanisms behind the therapy's efficacy and the differing patient responses [5]. Group 4: Mechanisms of Disease Relapse - Disease relapse in one patient was associated with the reactivation of pathogenic B cells and the reappearance of autoantibodies [6][7]. - Metabolic reprogramming of B cells characterized by excessive glycolysis was linked to disease relapse, which can be regulated by factor RFX5 [6][7].