In a groundbreaking development, the Tisch MS Research Center of New York has announced the publication of its FDA-approved Phase II stem cell treatment study results in the prestigious journal Stem Cell Research & Therapy. The study, which is the first-ever Phase II placebo-controlled, double-blind stem cell trial in the U.S. to investigate stem cell-derived treatments for disabled patients with progressive multiple sclerosi, has shown significant improvements in multiple areas of function for this patient population.
The study focused on 54 patients with Progressive MS, the most disabling form of the disease, who do not have active lesions or ongoing relapses, leaving them without viable treatment options. Although the study did not meet its primary endpoint, it demonstrated improvements in walking speed, bladder function, and gray matter volume for patients with significant disability.
Among the key findings, patients who received stem cell treatments and required walking assistance saw a 3.7% increase in their walking speed for the timed 25-foot walk test, while the placebo group experienced a 54% decrease. Additionally, 76% of patients who received stem cell treatments showed improved bladder function after one year, compared to only 27% in the placebo group. The study also found that stem cells could preserve gray matter volume in patients with less advanced decline.
Dr. Saud A. Sadiq, Director and Chief Research Scientist of the Tisch Center, emphasized the significance of these findings, stating, "These findings represent a significant breakthrough for progressive MS patients, who experience the most disabling symptoms and for whom there are no other treatment options available. Thus, these results have important quality-of-life implications for this group of patients."
The study also identified two new biomarkers that may provide a surrogate measure of treatment response. Patients who received stem cell treatments showed decreased levels of the protein CCL2, a potential indicator of inflammatory microglial cells, and an increase in the protein MMP9, which plays a regenerative role in the central nervous system, compared to the control group.
Dr. Violaine Harris, a principal investigator at the Tisch Center and lead author of the study, added, "The study's findings suggest that stem cells could be effective not only for treating MS, but also for reversing patient disability after significant progression. We look forward to building on these findings in future research, particularly in terms of increasing the dosage of stem cells and measuring the response of the two new biomarkers identified in our research."
The Phase II trial, which started in 2018, was a randomized, double-blind, placebo-controlled clinical study with a compassionate cross-over design. Patients received six injections of either autologous MSC-NPs or saline injections every two months. The study's Phase I results, published in 2018, were the first to show reversal of established disability in MS from stem cells, and a follow-up study demonstrated sustained clinical improvement in a subset of patients with no long-term side effects.
The Tisch MS Research Center of New York, the world's largest MS research center, is committed to finding the cause and cure for multiple sclerosis. The Center's multidisciplinary research is designed to understand all aspects of MS, from cellular and molecular experimentation to clinical trials focused on preventing or reversing disability, with the ultimate goal of treating and curing the disease. The publication of these Phase II results in Stem Cell Research & Therapy marks a significant milestone in the Center's ongoing efforts to develop effective stem cell-based treatments for progressive MS patients.
