Abstract Details

Title CAR-T Cell Therapy for Multiple Sclerosis: A Systematic Review of Emerging Clinical, Safety, and Biomarker Evidence

Topic Multiple Sclerosis

Presentation(s) P11 - Poster Session 11 (11:45 AM-12:45 PM)

Poster/Presentation
Number 19-002

Objective To systematically evaluate the efficacy, safety, and biomarker effects of chimeric antigen receptor (CAR) T-cell therapy in multiple sclerosis (MS).

Background

Current MS disease-modifying therapies are immunomodulatory and seldom induce deep or durable remission. The success of anti-CD20 monoclonal antibodies has highlighted B-cell depletion as an effective strategy. CAR-T cells engineered to target CD19 or autoreactive T-cell receptors may provide sustained immune reset and durable disease control.

Design/Methods A systematic review was performed according to PRISMA guidelines, including published case reports, case series, and early-phase clinical trials up to October 2025. Eligible studies reported outcomes of CD19- or peptide–MHC–directed CAR-T therapy in MS. Data extracted included demographics, CAR design, adverse events, MRI and clinical outcomes, and relapse-free survival. Risk of bias was assessed using the ROBINS-I tool.

Results Seventeen publications (N=27 patients) met inclusion criteria. Most patients had highly active or progressive MS refractory to conventional therapies. Universal B-cell depletion was achieved. Low-grade cytokine release syndrome occurred in approximately 30% of patients; no grade ≥3 neurotoxicity was reported. Among 13 patients who followed ≥6 months, 11 achieved “no evidence of disease activity” (NEDA). Cerebrospinal fluid neurofilament light chain levels decreased after treatment, supporting a biologic effect. However, all available studies were uncontrolled, heterogeneous, and at serious risk of bias.

Conclusions Early reports suggest CAR-T-cell therapy can induce prolonged remission in treatment-refractory MS with manageable short-term toxicity. Nonetheless, evidence remains preliminary, limited by small sample sizes and publication bias. Long-term safety—particularly persistent hypogammaglobulinemia, infection risk, and secondary malignancies—remains uncertain. Controlled trials with extended follow-up are essential to define the true therapeutic potential and safety profile of CAR-T therapy in MS.

Authors/Disclosures
Daniel V. De Siqueira Lima, Jr., MD PRESENTER	Dr. De Siqueira Lima has nothing to disclose.
José Felipe Lacerda Fernandes	José Felipe Lacerda Fernandes has nothing to disclose.
Luiza Villarim	No disclosure on file
Arthur F. Vasconcelos, MD	Mr. Vasconcelos has nothing to disclose.
BIANCA ETELVINA S. OLIVEIRA, Sr.	Dr. OLIVEIRA has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MERCK. Dr. OLIVEIRA has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NOVARTIS. Dr. OLIVEIRA has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AMGEN. Dr. OLIVEIRA has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NOVARTIS. Dr. OLIVEIRA has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MERCK.

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