CAR T cells as novel therapeutic strategy for multiple sclerosis and other neuroimmune disorders
Year: 2025
Type: article
Abstract: Chimeric antigen receptor (CAR) T-cell therapy is rapidly emerging as a transformative approach for treating multiple sclerosis (MS) and other neuroimmune disorders such as neuromyelitis optica spectrum disorder (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), and myasthenia gravis (MG), alongside several other rare neuroimmunological conditions currently being evaluated in compassionate-use or early-phase studies. These conditions are driven in part by autoreactive B cells that sustain chronic inflammation and progressive tissue damage. While current immunomodulatory therapies have improved clinical outcomes, they often require lifelong administration and fail to effectively eliminate compartmentalized inflammation within the central nervous system. Recent advances in CD19- and BCMA-directed CAR T-cell therapy, initially developed for hematologic malignancies, demonstrate the potential to achieve targeted, durable B-cell depletion and immune reprogramming in autoimmune diseases. Preclinical models and early-phase clinical trials have shown promising efficacy, including reduced relapse rates, stabilization of disability progression, and decreased autoantibody levels, alongside a favorable safety profile with lower rates of high-grade cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) compared to oncologic applications. This review synthesizes the current evidence supporting the use of CAR T-cell therapy in neuroinflammatory diseases and explores its potential to redefine treatment paradigms by shifting from chronic immunosuppression to long-term immune tolerance, creating a favorable environment for repair mechanisms. Realizing the full therapeutic promise of CAR T-cells in autoimmune neurology will require sustained research in heterogeneous populations and across disease spectrums.
Source: Journal of Neuroinflammation
Institutions: University of Southern Denmark, Humboldt-Universität zu Berlin, Slagelse Hospital, Charité - Universitätsmedizin Berlin, Medical University of Warsaw, Medizinische Hochschule Hannover, University of Connecticut, UConn Health, Klinički Centar Crne Gore, Universidade Estadual de Campinas (UNICAMP), Brazilian Institute of Neuroscience and Neurotechnology, Inserm, Université Paris-Saclay, The University of Sydney, Heinrich Heine University Düsseldorf, Palacký University Olomouc, Universitat de Vic - Universitat Central de Catalunya, Universitat Autònoma de Barcelona, Vall d'Hebron Hospital Universitari, Centre d'Esclerosi Múltiple de Catalunya
Language: English
Subfield: Pathology and Forensic Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $4,090
OpenAlex ID: W7117558538