Chimeric antigen receptor T-cell therapy in pediatric neurological autoimmune diseases: mechanisms, clinical applications, and future perspectives.

Pediatric neurological autoimmune diseases (PNADs) are a result of immune system abnormalities that target the central and peripheral nervous systems, leading to various neurological dysfunctions in children. The limitations of current immunotherapies underscore the necessity for more efficacious treatment interventions. The objective of this review is to examine the fundamental principles, recent advancements, and clinical applications of chimeric antigen receptor (CAR) T cell therapy in the treatment of pediatric neurological autoimmune diseases (PNADs). By specifically targeting and reducing pathogenic B cells, CAR-T therapy has the potential to reset the immune system. Growing evidence from preclinical animal studies, case reports, and early clinical trials suggests that CAR-T cell therapy has therapeutic potential in managing autoimmune diseases such as myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) and systemic lupus erythematosus (SLE). However, several challenges remain, including the high cost of treatment, safety concerns such as cytokine release syndrome and neurotoxicity, and a lack of long-term safety data in pediatric populations. Future research should prioritize optimizing the management and evaluation model for the entire process, as well as refining CAR design and development, to enhance the safe and effective clinical application of CAR T-cell therapy in patients with PNADs.