CAR-T Cell Therapy and MS: An Updated Look at This Emerging Treatment

Sponsored by Bristol Myers Squibb

Multiple Sclerosis_Clinical Trials_MS doctors discussing lab results

3 Sep 2026 | ~04:37 Engagement Time

Author

Kathy Costello , Nurse Practitioner

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Our Immune System

The immune system is our body’s army of cells and processes that protects us from harmful invaders like bacteria, viruses and parasites. Some immune cells are produced that are considered “auto-reactive” meaning that they misrecognize our own cells as invaders. Fortunately, healthy regulatory mechanisms usually eliminate or control these autoreactive cells before they can cause harm. However, in some people, the regulatory mechanisms that keep the immune system functioning without auto-reactivity malfunction and the autoreactive cells can multiply and provoke an autoimmune condition such as multiple sclerosis (MS).  

What Happens in MS

MS is a complex disease of the central nervous system or CNS (the brain and the spinal cord) that is orchestrated by a malfunctioning immune system. In MS, the body’s own immune system causes acute  inflammation – in the form of attacks (relapses) and also chronic inflammation within the CNS that is considered to be a contributor to MS progression.  

Targets of the malfunctioning immune system include the myelin sheath wrapped around many nerves in the CNS, the cells that make myelin and also the nerve fibers (axons)  that are beneath the myelin. Research over the past 2 decades indicates that the immune system attacks come from immune cells circulating in the bloodstream, and also from within the CNS where chronic inflammation becomes established. The chronic inflammation within the CNS appears to be driven by activation of certain cells – including B cells, certain T cells, astrocytes and microglia. Identification of these cells as part of MS immune pathology has led to the identification of  potential treatment targets – and research on new treatment strategies.  

CAR-T Cell Therapy

CAR-T cell therapy  is currently approved and used to treat blood cancers and more recently has become  a treatment of great interest in MS and other autoimmune conditions such as Systemic Lupus, Myasthenia Gravis, Stiff Person Syndrome, Neuromyelitis Optica Spectrum Disorder and several others. In CAR-T cell therapy researchers modify and amplify the activity of certain cytotoxic (killer) T cells so that they can more readily identify and eliminate cells considered to be contributors to MS inflammation and damage. The “CAR” or chimeric antigen receptor essentially gives the T cell better vision to “see” its target. It can then interact with the target and destroy it or suppress its activity.  

In MS, both T cells  and B cells  are contributors to the MS disease process, but B cell activity and antibody damage seems of very high importance. Thus CAR-T cell therapy directed at B cells is of significant interest. More recently, researchers also have interest in suppressing the MS immune response with CAR-T regulatory T cells. CAR-T cells may also be able to work  within the CNS – which could be important  for treating chronic inflammation and helping those with progressive MS. The goal of either approach is a reset of the immune system – with less inflammation and less damage. 

Most CAR-T cell therapies studied in MS are autologous, meaning they use the persons own T cells. Another type of CAR-T cell therapy is known as allogenic – using cells from others. While allogenic CAR-T cells are more readily available, they are not the person’s own cells and thus may produce more side effects than autologous CAR-T cells. There are many CAR-T cell approaches under study in various conditions and below are two types under investigation in MS: 

  • CD-19 CAR-T cell therapy – In this type of CAR-T cell therapy, researchers amplify cytotoxic T-cell activity so that they can rapidly identify cells that have a surface marker known as CD-19. These CD-19 cells are known contributors to MS damage. Most research to date has been with this type of CAR-T cell therapy. 
  • CAR-Treg cell therapy – In this type of CAR-T cell therapy, researchers engineer regulatory T-cells with a CAR that directs the cells to sites of inflammation where they can suppress the immune response and reduce inflammation. This is an attractive approach as the inflammatory cells are suppressed, but cells are not destroyed, potentially reducing risks and side effects. 

How Far Along Is the Research in MS?

Beginning in 2012, CAR-T cell therapy has been studied in the animal model of MS known as EAE. From there, a small number of people with MS (2 in one investigation and 4 people in another) have been treated with CD-19 CAR-T cell therapy. Results did show the CAR-T activity in the CNS and other mixed results with 3 people showing new inflammation and 3 with some evidence of stability. The treatment was  tolerated fairly well. Based upon these early individual patient results there are numerous Phase 1 and Phase 2 studies investigating CAR-T cell therapy safety and dosing in people with progressive MS. These are occurring in the US, Canada, Germany, Israel, and China. In addition, there is a trial for long term follow-up of people with various autoimmune conditions who have received at least one infusion of a CAR-T cell therapy. More information can be found at clinicaltrials.gov. 

Summary

CAR-T cell therapy is an innovative approach to treating MS, particularly progressive forms of the disease with the potential to target chronic inflammation within the CNS. Clinical investigations are in early phases, and if safety and efficacy are consistently found through systematically conducted clinical trials, the treatment could be available in the 2030’s.  While it is very early in the clinical trial process, it is not too early to discuss the treatment and the clinical trials with your MS provider. 

References

Li, Y. R., Chen, Y., & Yang, L. (2026). Redefining multiple sclerosis with CAR-T cell therapy. Molecular therapy : the journal of the American Society of Gene Therapy, 34(3), 1326–1339. https://doi.org/10.1016/j.ymthe.2025.12.022 

Kumawat, N., Sharma, A., & Upadhayay, S. (2026). Reprogramming immunity: the promise and challenges of CAR T cell therapy in multiple sclerosis. Inflammopharmacology, 10.1007/s10787-026-02295-2. 

Bruno, A., & Greco, R. (2025). Anti-B-cell CAR-T therapy in multiple sclerosis is simply a more profound immunoablation? No. Multiple sclerosis (Houndmills, Basingstoke, England), 31(10), 1163–1165.