The most common form of multiple sclerosis is a relapsing-remitting MS. In relapsing-remitting multiple sclerosis, patients have relatively quiet, healthy times interrupted by disease flares. MS flares can cause many different neurological symptoms. The disease can interfere with any number of bodily processes, from walking to seeing, to urinating.
For many years, the only treatments available for relapsing-remitting MS were steroids or other powerful medications that had troubling side effects. Recently, disease-modifying drugs have become available that extend the times in between flares. Steroids are still used to treat disease flares, but disease-modifying drugs seem to make those flares occur less often.
One way that scientists judge the value of disease-modifying therapy for relapsing-remitting MS is to see how long it can prevent relapse. For example, researchers divide a group of patients into two groups, give each group a different treatment, and track to see the time until a relapse occurs.
Researchers took this same scientific approach to compare disease-modifying therapy to nonmyeloablative hematopoietic stem cell transplantation which is the infusion of stem cells without destroying the patient’s existing bone marrow and stem cells with chemotherapy. The results were published in the prestigious Journal of the American Medical Association (JAMA).
The scientists from hospitals and institutions around the world followed about 100 women with relapsing-remitting multiple sclerosis. Half received disease-modifying therapy, which is the current standard of care, while the other half received stem cell therapy.
During the first year of the study, the symptoms were more severe in patients who received disease-modifying therapy. This was expected since patients with this form of MS tend to get worse over time. Impressively, the group who received stem cell treatment had less severe symptoms than when they started a year earlier.
Three patients who received stem cell therapy experienced disease progression; however, a staggering 34 patients in the disease-modifying group had disease progression. In other words, far fewer RRMS patients in the stem cell therapy group had disease progression than those receiving standard disease-modifying treatments.
The authors of this groundbreaking study mention that “further research is needed to replicate these findings,” presumably in a double-blind trial. Nevertheless, this is not a small study (~100 patients) and patients were followed for a very long time (5 years). As such, the results provide strong, preliminary evidence that stem cell therapy was more effective than disease-modifying therapy for patients with
Reference: Burt, R., et al. (2019). Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis. JAMA. 2019;321(2):165-174.