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Stem cell treatments for Multiple Sclerosis patients Is it successful and safe
Stem cell therapy for MS splits into two very different things, and only one of them has real human outcome data.
The one with data is aHSCT: chemo wipes out the immune system, then your own stem cells are returned. In a pooled 764 patients, 68% had no disease activity at 5 years and 23.3% had disability progression [1]. Treatment-related death was 5.3% overall but 0.3% after 2005 and 0% with low-intensity regimens [1]. A registry review puts non-relapse mortality around 1% in 2015-2020 and NEDA at 66-93% [3]. That is a real effect, but it is a punishing procedure, and the evidence is mostly single-arm, not randomized against the best drugs [2][3].
| Therapy | Best human evidence | Control group? |
|---|---|---|
| aHSCT | 68% NEDA at 5 yr, 764 pts [1] | Mostly no |
| aHSCT (registry) | NEDA 66-93%, NRM ~1% [3] | No |
| MSC intrathecal | 22 pts, 27% new lesions at 12 mo [4] | No |
| MSC intravenous | 10 pts, no control [4] | No |
The mesenchymal stem cell (MSC) infusions sold in clinics are a different story. The human trials are tiny, open-label, and uncontrolled: 22 patients here [4], 10 there [4], 15 in another [4]. Without a control group, improvements cannot be attributed to the cells [4]. The mouse data show benefit [4], but mouse MS is not human MS.
My call: aHSCT is a real but aggressive option for aggressive MS, best done in a trial or specialist center; clinic MSC infusions are unproven and should not be bought as treatment. Confidence: moderate on aHSCT, low on MSCs.
Sources used 4
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Autologous hematopoietic stem cell transplantation for MS
This study evaluates the safety and efficacy of autologous hematopoietic stem cell transplantation (aHSCT) for multiple sclerosis (MS), suggesting it may be safer and as effective as current disease-modifying drugs.
DOI: 10.1212/WNL.0000000000003995 -
Progress and prospects for the use and the understanding of the mode of action of autologous hematopoietic stem cell transplantation in the treatment of multiple sclerosis
An expert commentary reviewing the progress, mechanisms, regimens, and clinical implications of autologous hematopoietic stem cell transplantation (AHSCT) for multiple sclerosis, highlighting potential suppression of CNS inflammation and possible disability reversal alongside th…
DOI: 10.1080/1744666X.2017.1297232 -
Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT)
Autologous and allogeneic HSCT, plus emerging cellular therapies, are surveyed to summarize indications, outcomes, risks, and future directions in autoimmune diseases.
DOI: 10.1038/s41409-022-01702-w -
Cell-Based Therapeutic Strategies in Multiple Sclerosis
Mesenchymal stem cell transplantation is safe and tolerable in multiple sclerosis, with early phase I/II trials showing possible functional and MRI benefits but no established efficacy.
DOI: 10.17925/enr.2014.09.01.37