Stem Cell Transplantation in Multiple Sclerosis

A Potentially Promising But Risky MS Therapy

What is Stem Cell Transplant in Multiple Sclerosis?
What is Stem Cell Transplant in Multiple Slclerosis?. David Mack/Getty Images

Stem cell transplantation is an experimental and emerging field of MS research, and so far scientific results have been promising. That being said, experts are cautious, as this type of therapy is taxing on the body, and requires a careful weighing of the pros versus cons.

What Is Stem Cell Transplantation in MS?

In the past, stem cell transplants were reserved for those with certain types of cancer of the blood or bone marrow, like leukemia, lymphoma, or multiple myeloma.

But over the last several years, stem cell transplant has been investigated in treating other immune-related medical conditions, including multiple sclerosis.

A stem cell transplant for treating MS starts off with a procedure called stem cell harvesting. This procedure entails removing a person's stem cells from their own bloodstream (accessed through a vein) or their own bone marrow (accessed through a needle in the pelvis).

The formal medical term for this is an autologous hematopoietic cell transplant—autologous meaning a person's own stem cells are used and hematopoetic meaning blood-cell forming.

Once the stem cells are collected and stored, a person will have their immune system either suppressed (called a non-myeloablative transplant) or wiped out (called a myeloablative transplant). This suppression or wiping out is obtained through chemotherapy drugs and immune-depleting antibodies.

The difference between immune system suppression and ablation (wiping out) is that wiping out requires higher, more toxic doses of chemotherapy, as compared to suppression, which requires lower, less toxic doses.

After this phase is complete, a person will rest for a couple days before undergoing stem cell infusion—a procedure in which stem cells are given back to the person through a vein.

Once infused, these stems cells travel from the blood into the bone marrow where they reproduce and build up a novel immune system—the idea being that this new and improved immune system will be healthy and will not attack the myelin sheath in the brain and spinal cord.

The Science Behind Stem Cell Transplantation in MS

In a 2016 Canadian study in Lancet, 12 adult participants with relapsing-remitting multiple sclerosis and 12 patients with secondary-progressive MS underwent stem cell transplantation. These participants had aggressive disease and an overall very poor prognosis, meaning they had multiple early MS relapses with sustained disability, despite MS treatment.

Results of this study were promising though. Of those 24 participants, 17 (70 percent) had no MS disease activity three years after transplantation. No MS disease activity meant:

  • no new MS relapses
  • no new MS lesions on MRI
  • no evidence of disease progression

In addition, at 7.5 years after the transplant, 40 percent of the participants had improvement in their MS-related disability.

In fact, some of the participants had remarkable recoveries including:

  • resolution of nystagmus and ataxia
  • return to work or school
  • getting married or engaged
  • having children using banked or donated egg/sperm

This all being said, it's important to note that of the 24 participants, one did die due to an infection, a complication related to stem cell transplantation. Another participant also developed severe liver-related problems and was hospitalized for an extended period of time. Also, there were a number of side effects related to the transplantation, like neutropenic fever and chemotherapy-related toxicities.

More Stem Cell Transplant Research in MS

In a 2015 study in JAMA, 123 participants with relapsing-remitting MS and 28 participants with secondary-progressive MS underwent stem cell transplantation. The participants were followed on average for 2.5 years. Unlike the prior study, the immune systems in these patients were suppressed, as opposed to being wiped out prior to stem cell infusion—called a non-myeloablative stem cell transplant.

Results suggested both a decline in the number of relapses and the number of gadolinium-enhanced MS lesions on all post-stem cell transplant MRIs. Brain MRIs had been completed at 6 months after transplant and then annually.

Also, there was an improvement by one or more points in the EDSS score in 50 percent of the participants at 2 years and 64 percent of the participants at 4 years. The EDSS score measures MS severity and progression to being disabled. But this improvement was only seen in those with relapsing-remitting MS (not in those with secondary-progressive MS), and in those who had MS for 10 years or less.

This study had significantly less worrisome side effects—no deaths or serious infections. This is likely attributed to the suppression versus wiping out of the immune system before the stem cells were infused.

The Bottom Line

While this is exciting research, experts are still cautious. These trials are small and lack control groups. Larger and more long-term studies are needed to really understand the benefit and safety of stem cell transplantation in treating MS. Also, the health risks of stem cell transplantation are very real. So finding ways to reduce these risks is a current and pressing challenge.

In addition, even if stem cell transplantation is approved for MS, it may be a therapy that is reserved for those with more severe MS who have failed other therapies – not necessarily that it won't work for those with milder disease, but just that the risks may not be worth it.


American Cancer Society. Stem Cell Transplant for Cancer. Accessed June 19th 2016.

Atkins et al. Immunoablation and autologous haemopoetic stem-cell transplantaton for aggressive multiple sclerosis: a multicentre single-group phase 2 trial. Lancet. 2016. 2016 Jun 8. pii: S0140-6736(16)30169-6.

Burt RK et al. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA. 2015 Jan20;313(3):275-84.

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