Stem cell therapy for multiple sclerosis is an active research area, but no stem cell product is FDA-approved for the disease. The best-studied approach is a blood stem cell transplant, while mesenchymal cell studies remain early and inconsistent.
How approved and experimental cell products differ is covered in what is stem cell therapy, and this page looks at one neurological condition where the research is more advanced than most.
Regulatory status for multiple sclerosis
The FDA consumer alert on regenerative medicine products said in 2020 that the only FDA-approved stem cell products were blood-forming stem cells derived from umbilical cord blood, approved for disorders that affect the production of blood and not for other uses. On December 18, 2024, the FDA approved Ryoncil, a donor bone marrow-derived mesenchymal stromal cell therapy, for steroid-refractory acute graft-versus-host disease in children; the FDA’s list of approved cellular and gene therapy products is the current reference. The alert also says that none of the products it discusses have been approved to treat any neurological disorder, with multiple sclerosis listed first among the examples.
This does not mean there is no research. It means that any stem cell procedure offered for multiple sclerosis outside a regulated study is unapproved for that use, and that the evidence has to be judged on the published trials rather than on the way a procedure is described.
Why researchers study cell therapies for multiple sclerosis
A 2025 review in the journal Multiple Sclerosis and Related Disorders, summarised on PubMed, describes multiple sclerosis as a chronic autoimmune disease of the central nervous system that leads to neurodegeneration and disability. That description explains the two main research strategies. One tries to reset or replace the immune system that is driving the damage, and the other tries to use cells that may calm inflammation or support nerve tissue.
These strategies use very different cell types and carry very different levels of evidence, which is why a general phrase such as stem cell therapy can mislead. The ISSCR patient guide notes that the same stem cell treatment is unlikely to work for different diseases or conditions, and that cell type needs to match the science of the disease, a principle that applies strongly here.
- Only approved stem cell products are cord-blood-derived: Only approved stem cell products are cord-blood-derived
- Approved only for blood production disorders: Approved only for blood production disorders
- No approval for neurological disorders including MS: No approval for neurological disorders including MS
- AHSCT efficacy statement: AHSCT efficacy statement
- Safety improved, lower mortality: Safety improved, lower mortality
Blood stem cell transplantation
The approach with the most research behind it is autologous hematopoietic stem cell transplantation, often shortened to AHSCT. The patient’s own blood-forming stem cells are collected, the immune system is suppressed by chemotherapy, and the cells are returned to rebuild the blood and immune system. A review of recommendations from the National Multiple Sclerosis Society in JAMA Neurology reports that studies in relapsing multiple sclerosis have shown favorable results, while larger randomized trials are still needed to compare it with the most effective drugs, and that the safety of the procedure has improved considerably, with much lower mortality rates than reported earlier.
The same document is explicit about the limits. It says questions remain about the ideal protocol, particularly the best conditioning regimen, and that larger randomized clinical trials are needed to learn whether the transplant has advantages over the most effective disease-modifying drugs now available. It points to one such trial, BEAT-MS, as in progress at the time of writing.
The Society’s position is narrow rather than general. It states that the transplant may be a useful option for people with relapsing multiple sclerosis who show substantial breakthrough disease activity despite high-efficacy treatment, or who cannot take those drugs. It adds that the procedure should only be performed at centers with substantial experience and expertise. Those conditions are a long way from an open-ended offer to treat any form of the disease, and the choice between this and other options belongs in a specialist consultation.
Mesenchymal stem cell studies
Mesenchymal stem cells are a different matter. A 2024 updated systematic review and meta-analysis analysed 30 studies of these cells in people with multiple sclerosis. It reported that people given cells by an intrathecal route, meaning into the fluid around the spinal cord, had a noteworthy improvement in a standard disability score compared with a placebo group, while the intravenous group did not show significant changes. The annualised relapse rate did not decrease significantly.
The authors’ own conclusion is cautious: the safety potential is highlighted, but the effectiveness of mesenchymal cell therapy remains uncertain because of study limitations and ineffective outcome measures. The most common complications reported in both short-term and long-term follow-up were infection, back pain and gastrointestinal symptoms.
The 2025 systematic review mentioned above searched the literature to April 2025 and found 7574 studies, of which 34 met its inclusion criteria, covering both preclinical and clinical work. It says short-term safety profiles were favorable but that long-term efficacy and optimal administration protocols remain uncertain, and that variations in cell sources, administration methods and patient responses show why standardized protocols are needed.
Safety and trial status
Safety deserves the same attention as potential benefit. Transplantation that uses chemotherapy to suppress the immune system is a major medical procedure, which is part of why the Society recommends experienced centers and a shared database of recipients so that outcomes can be tracked. Cell injections given outside a trial raise a different problem, because there is no protocol, oversight or comparison group to show what happened.
The ISSCR guide also says that cells from a person’s own body are not automatically safe, because collection, processing and reintroduction can involve contamination and other risks, and that an experimental treatment offered for sale is not the same as a clinical trial. The practical test is whether a study is registered, ethically overseen and designed to answer a specific question, and whether the person offering it can show that.
What this means for readers
Taken together, the research describes one route with the most clinical research, with specific eligibility rules and a set of cell-based approaches that are still being worked out. Neither is the same as a clinic advertising a single injection for the disease. The ISSCR patient guide advises caution toward treatments offered without regulatory approval or outside a legitimate and registered clinical trial, and says that receiving one unproven treatment may make a person ineligible for future clinical trials or treatment options.
For multiple sclerosis in particular, there are already many FDA-approved disease-modifying therapies, as the National Multiple Sclerosis Society document itself notes. Decisions about which option to use, including whether a transplant or a clinical trial is appropriate, belong with a licensed neurologist who knows the reader’s disease course. Similar questions about status and evidence arise in other conditions, as in the discussions of stem cell therapy for Parkinsons disease and COPD stem cell therapy.
Frequently asked questions
Is stem cell therapy approved for multiple sclerosis?
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No. The FDA consumer alert says none of the widely promoted stem cell products have been approved to treat neurological disorders, and multiple sclerosis is one of the conditions it names.
What is AHSCT?
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It is a transplant of a person’s own blood-forming stem cells after immune-suppressing treatment. The National Multiple Sclerosis Society describes it as possibly useful for a defined group of people with relapsing disease and calls for larger randomized trials.
Are mesenchymal stem cells proven to work in multiple sclerosis?
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No. Reviews report mixed results and say that effectiveness remains uncertain, with differences in cell source and route of delivery between studies.
Who should decide whether a transplant or trial is appropriate?
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A licensed neurologist who knows the person’s type of multiple sclerosis, treatment history and current disease activity.
