Stem Cells Treatments Show Promise in MS

Violaine Harris, Ph.D., a senior research scientist with Tisch MS Research Center of New York, discusses new research that shows stem cell treatments can improve disability in patients with MS.

Stem cell treatments may result in substantial improvement in muscle strength in patients with multiple sclerosis (MS), according to new research.

In a preliminary analysis of the data, researchers at the Tisch MS Research Center of New York found that stem cells are the first treatments that improve neurological outcomes in secondary progressive MS (SPMS) and primary progressive MS (PPMS) patients, Tisch MS Research Center of New York, said in a news release.

Following the stem cell treatments, upper limb dexterity and walking speed improved for 36% of patients, while 30% realized improved muscle strength.

Managed Healthcare Executive® spoke with Violaine Harris, Ph.D., a senior research scientist with Tisch MS Research Center of New York who is also closely involved with the stem cell treatment study. Harris not only discussed the stem cell treatment research, but also other promising MS discoveries.

MHE: Please discuss the significance of the Center’s interim stem cell treatment trials, and the next steps in this research.

Harris: We are conducting a phase 2 double-blind, placebo-controlled clinical trial testing the efficacy of autologous mesenchymal stem cell neural progenitors (MSC-NPs) administered into the spinal fluid (intrathecally) of patients with SPMS and PPMS. While we did observe encouraging trends of improvement in patients treated with stem cells, it must be emphasized that a full data analysis with comparison with the placebo arm remains to be completed to determine whether trends in efficacy are statistically significant. We are currently completing the final stages of the trial and anticipate a full data analysis completed by the end of this year.

Based on the preliminary analysis, we are starting discussions with the FDA to explore whether a dose escalation trial is warranted for the more disabled patients who did not respond to stem cell treatment. Our goal is to optimize cell therapy for progressive MS patients, which may require higher doses in some patients in order to achieve clinical benefit. In line with the goal of treatment optimization, we are particularly excited about our biomarker research, which would allow us to predict which patients are most likely to respond to cell therapy based on their biomarker profile.

MHE: Describe the progress in stem cell research globally and the implications for treating MS. Are there already commercial applications?

Harris: Translation of stem cell technology to patients with MS has been frustratingly slow. Regarding mesenchymal stem cell-based therapies, multiple early-stage clinical studies have been performed with encouraging data but small sample sizes. Very few phase 2 studies have been conducted, so we really need more data from larger trials to show efficacy. Good phase 2 data is complicated by the fact that MS is a heterogenous disease, and we lack good clinical outcomes or surrogate markers for progressive MS.

I am less familiar with the commercial space for cell therapy in MS, but it is most likely hindered by the high cost of treatment, lack of standardization, and regulatory hurdles associated with biological licensing. Altogether, this slows the pace of translation, despite the very exciting stem cell research that continues to be conducted around the world. Given the rapidly-advancing stem cell research and associated technology, I assume more and more companies will start to enter this field to address this unmet need in MS.

MHE: Which other MS treatments and research are you excited about?

Harris: Recent clinical success with anti-CD20 monoclonal antibody therapy (ocrelizumab, rituximab) as a highly effective treatment for MS has shifted the focus of MS research to CD20-expressing cells. CD20 is mostly present in B-cells; however, a population of CD20-containing T-cells has been acknowledged for some time, and these are present in higher numbers in MS patients.

New research seems to suggest that the interaction between B-cells and T-cells is required for the origin of CD20+ T-cells, which along with the antigen-presenting activity of B-cells, play an important role in MS disease. Understanding what population of CD20-containing cells are being targeted by anti-CD20 therapy and how they contribute to MS is essential to better characterize disease onset and progression.

MHE: Please explain the connection between Epstein-Barr virus and MS.

Harris: Understanding the cause of MS has been a central and unanswered question in MS research. We have long suspected that Epstein-Barr virus (EBV) plays a critical role in the development of MS, and a recent study from Harvard adds substantial data to support the EBV hypothesis.

What we still don’t know is the mechanism that connects EBV infection, which happens in most people, to the development of MS, which only happens in some people. People with MS may have a genetic predisposition along with other environmental triggers — including low vitamin D — but we have yet to connect the dots between EBV and MS.

There is renewed interest in developing a vaccine against EBV, which would block EBV infection and hypothetically prevent MS. This line of research is challenging, but critically important to finding a cure for this disease.