Violaine Harris, Ph.D., principal investigator at the Tisch MS Research Center in New York, discusses new research that shows stem cell treatments can improve disability in MS patients.

According to new research, stem cell treatments could lead to substantial improvements in muscle strength in patients with multiple sclerosis (MS).

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

Following stem cell treatments, upper limb dexterity and walking speed improved for 36% of patients, while 30% achieved an improvement in muscle strength.

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

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

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

Based on the preliminary analysis, we are entering into discussions with the FDA to determine whether a trial of dose escalation is warranted for the most disabled patients who have not responded to stem cell treatment. Our goal is to optimize cell therapy for patients with progressive MS, which may require higher doses in some patients 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.

SME: Describe the progress of stem cell research globally and the implications for the treatment of MS. Are there commercial applications already?

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

I know less about the cell therapy commercial space in MS, but it is most likely hampered by the high cost of treatment, lack of standardization, and regulatory hurdles associated with biologic licensing. Overall, this slows the pace of translation, despite the exciting stem cell research that continues to be conducted around the world. Given the rapid development of stem cell research and related technology, I suspect that more and more companies will start to enter this field to address this unmet need in MS.

SME: What other MS treatments and research are you passionate about?

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

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

SME: Please explain the link between the Epstein-Barr virus and MS.

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

What we still don’t know is the mechanism that links EBV infection, which occurs in most people, to the development of MS, which only occurs in some people. People with MS may have a genetic predisposition as well as other environmental triggers – including low vitamin D – but we haven’t yet made the connection between EBV and MS.

There is renewed interest in the development of an EBV vaccine, 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.