%0 Journal Article
%A Konitsioti, Agni M.
%A Prüss, Harald
%A Laurent, Sarah
%A Fink, Gereon Rudolf
%A Heesen, Christoph
%A Warnke, Clemens
%T Chimeric antigen receptor T-cell therapy for autoimmune diseases of the central nervous system: a systematic literature review
%J Journal of neurology
%V 271
%N 10
%@ 0367-004X
%C [Darmstadt]
%I Steinkopff
%M FZJ-2025-00573
%P 6526 - 6542
%D 2024
%Z Funding Open Access funding enabled and organized by Projekt DEAL.
%X Importance: B-cell-targeting monoclonal antibodies have demonstrated safety and efficacy in multiple sclerosis or anti-aquaporin-4 IgG positive neuromyelitis optica spectrum disorder. However, these therapies do not facilitate drug-free remission, which may become possible with cell-based therapies, including chimeric antigen receptor (CAR) T cells. CAR T-cell therapy holds promise for addressing other antibody-mediated CNS disorders, e.g., MOG-associated disease or autoimmune encephalitis.Objective: To provide an overview of the current clinical knowledge on CAR T-cell therapy in central nervous system autoimmunity.Evidence review: We searched PubMed, Embase, Google Scholar, PsycINFO, and clinicaltrials.gov using the terms 'CAR T cell' and 'multiple sclerosis/MS' or 'neuromyelitis optica/spectrum diseases/NMOSD' or 'MOG-associated disease/MOGAD 'or' autoimmune encephalitis' or 'neuroimmunology'.Findings: An ongoing phase I clinical trial has indicated the safety and benefits of anti-BCMA CAR T cells in 12 patients with AQP4-IgG seropositive neuromyelitis optica spectrum disorder. Case reports involving two individuals with progressive multiple sclerosis and one patient with stiff-person syndrome demonstrated a manageable safety profile following treatment with anti-CD19 CAR T cells. Recruitment has commenced for two larger studies in MS, and a phase I open-label basket study is underway to evaluate BCMA-directed CAR T cells in various antibody-associated inflammatory diseases, including MOG-associated disease. Preclinical research on NMDA receptor antibody autoimmune encephalitis treated with chimeric autoantibody receptor T cells generated promising data.Conclusions and relevance: There is minimal evidence of the benefits of CAR T-cell therapy in individuals with central nervous system-directed autoimmunity. Nevertheless, multicenter controlled clinical trials with a manageable safety profile appear feasible and are warranted due to very promising case experiences.Keywords: Antibody-mediated CNS disorders; CAR T-cell therapy; Central nervous system autoimmunity; Multiple sclerosis; NMOSD.
%F PUB:(DE-HGF)16
%9 Journal Article
%$ 39276207
%U <Go to ISI:>//WOS:001321346700002
%R 10.1007/s00415-024-12642-4
%U https://juser.fz-juelich.de/record/1037240