A new study found that hematopoietic stem cell transplantation was more effective than disease-modifying therapy for patients with relapsing-remitting multiple sclerosis. In a randomized clinical trial that included 110 patients with relapsing-remitting MS, treatment with nonmyeloablative HSCT compared with disease-modifying therapy resulted in a significantly prolonged time to disease progression.

HSCT represents a potentially useful approach to slow or prevent progressive disability in relapsing-remitting MS. Researchers at the Northwestern University Feinberg School of Medicine, in Chicago, compared the effect of nonmyeloablative HSCT against disease-modifying therapy on disease progression.

Between September 2005 and July 2016, a total of 110 patients with relapsing-remitting MS, with at least two relapses while receiving DMT in the prior year, and an Expanded Disability Status Scale score of 2.0 to 6.0 were randomized at 4 U.S., European, and South American centers. 

Patients were randomized to receive HSCT along with cyclophosphamide and antithymocyte globulin, or DMT of higher efficacy or a different class than DMT taken during the previous year. The primary end point was disease progression, defined as an EDSS score increase after at least one year of 1.0 point or more on two evaluations six months apart, with differences in time to progression estimated as hazard ratios. The researchers found that disease progression occurred in three patients in the HSCT group and 34 patients in the DMT group. 

The study of patients with relapsing-remitting MS, nonmyeloablative HSCT, compared with DMT, resulted in prolonged time to disease progression. The researchers said further research is needed to replicate these findings and to assess long-term outcomes and safety.

According to Dr. Ben Thrower, MS Focus senior medical advisor, hematopoietic stem cell transplantation remains a hot topic in MS. HSCT is a stem cell therapy that is very different from mesenchymal stem cells which are being researched for neural repair. With HSCT the person with MS is having their immune system rebooted in a way that should stop the attack on myelin and axons. The interest and research in HSCT reflect a larger trend towards using more effective therapies earlier in MS. So, what have we learned about HSCT so far?

• It is a very effective therapy for the right person with MS.
• The right person appears to be someone with active RRMS below the age of 50.
• The therapy appears much less effective in SPMS or PPMS.
• The safety of HSCT is improving in recent years.

This study compares the effectiveness of HSCT to more traditional disease-modifying therapies in people with active RRMS. The researchers found that HSCT was more effective in stopping relapses, preventing new lesions on MRI and slowing progression of disability. The majority of patients had no evidence of disease progression after HSCT. Just importantly, HSCT had a good safety profile.

According to Dr. Thrower, an especially interesting part in the study was how their patients on natalizumab (Tysabri) did. The researchers found that a significant number of their Tysabri patients were still having relapses or new lesions on MRI. This has not been our experience in the real world. The majority of Shepard Center patients with RRMS on Tysabri or Ocrevus are doing very well. This study was not able to comment on Ocrevus as the drug was not approved in time.

Dr. Thrower said, “We may be moving towards a time when HSCT is a first line treatment option for people with RRMS. Clearly, it fits with the shift towards using more effective therapies earlier in the course of MS.” 

The study was published in JAMA online.