Chimeric Antigen Receptor T (CAR-T) Cell Therapy in Autoimmune Neurological Disease
A systematic review assessed 19 studies on CAR-T therapy targeting CD19, CD20, or B-cell maturation antigen in autoimmune neurological diseases. Efficacy signals were strongest in certain conditions like generalized myasthenia gravis and neuromyelitis optica spectrum disorder, with mixed results in multiple sclerosis. Lower rates of severe side effects compared to oncological uses were noted, though significant concerns like cytopenias emerged.
- Tags
- #MSResearch
- Most relevant to
- —
Why it matters
This review highlights the potential of CAR-T therapy for treating autoimmune neurological disorders, signaling a shift in therapeutic approaches but raising expectations cautiously due to the preliminary nature of findings and the variability in study outcomes.
What this does not prove
Evidence is constrained by the small size and uncontrolled nature of the studies, which makes broad recommendations premature. Variability in reported outcomes also limits the reliability of conclusions.
Next milestone
No next milestone was established from the available source.
Study facts
- Study design
- Systematic review
- Participants / samples
- 19 · basis not reported
- Randomised
- No
- Controlled
- Not reported
- Primary endpoint met
- Not reported
- Relevant MS type
- Not reported
- Publication date
- 2026-09-29
- Evidence reviewed
- Abstract only
- Regulatory approval
- Not reported
- Research areas
- Not classified
Original sources
- Primary evidenceChimeric antigen receptor T cell therapy in autoimmune neurological disease: current status of efficacy and safety. ↗DOI: 10.1136/bmjno-2026-001611
Supporting passages (10)
findingsRelapse was reported across several conditions, with follow-up generally limited to under two years.
limitationsCurrent evidence remains limited to small, largely uncontrolled studies with heterogeneous outcome reporting.
publication datePublication date: 2026-09-29
study designThis scoping review, conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA)-ScR guidelines, examined published clinical studies from January 2023 to January 2026 in which CAR-T cells targeting CD19, CD20 or B-cell maturation antigen were used to treat autoimmune neurological disease, identifying 19 eligible studies.
subjectsEfficacy signals were most consistent in refractory acetylcholine receptor antibody-positive generalised myasthenia gravis and Aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder, with encouraging but more preliminary results reported in multiple sclerosis, Lambert-Eaton myasthenic syndrome, myelin oligodendrocyte glycoprotein antibody-associated disease, chronic inflammatory demyelinating polyneuropathy, stiff-person syndrome and autoimmune encephalitis.
sample sizeThis scoping review, conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA)-ScR guidelines, examined published clinical studies from January 2023 to January 2026 in which CAR-T cells targeting CD19, CD20 or B-cell maturation antigen were used to treat autoimmune neurological disease, identifying 19 eligible studies.
randomizedCurrent evidence remains limited to small, largely uncontrolled studies with heterogeneous outcome reporting.
peer reviewedThis scoping review, conducted according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA)-ScR guidelines, examined published clinical studies from January 2023 to January 2026 in which CAR-T cells targeting CD19, CD20 or B-cell maturation antigen were used to treat autoimmune neurological disease, identifying 19 eligible studies.
interventionChimeric antigen receptor T (CAR-T) cell therapy, initially developed for haematological malignancy, has been repurposed to more comprehensively deplete pathogenic B-cell and plasma cell populations in autoimmune disease, raising the possibility of durable, treatment-free remission.
follow upwith follow-up generally limited to under two years.
AI assessment, not yet reviewed by a person · version 1 · Community votes are separate from evidence review.