Stem cell treatment results from the U.S. Food and Drug Administration-approved (FDA) Phase II study have been published by the Tisch MS Research Center of New York, the world’s largest multiple sclerosis (MS) research center, in the esteemed journal Stem Cell Research & Therapy. This groundbreaking study, focused on patients with Progressive MS, represents 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 disease who do not have active lesions or ongoing relapses, thus lacking viable treatment options.
Although the study did not meet its primary endpoint, it demonstrated significant improvements in several critical areas for patients with considerable disabilities, including walking speed, bladder function, and preservation of gray matter volume. Among patients requiring walking assistance, those who received stem cell treatments exhibited a 3.7% increase in their walking speed for the timed 25-foot walk test. In contrast, the placebo group saw a 54% decrease in walking speed. These improvements were consistent with results from a six-minute walking test administered to participants.
After one year of treatment, 76% of patients receiving stem cell treatments reported improved bladder function, compared to just 27% in the placebo group. Patients with gray matter volume above the 50th percentile who received stem cell treatments experienced preservation of gray matter volume. Conversely, the placebo group saw a decrease, suggesting that stem cells may help maintain gray matter volume in patients with less advanced decline.
New Biomarkers Identified in Stem Cell Study for Progressive Multiple Sclerosis
The study also identified two new biomarkers that could serve as surrogate measures of treatment response. Patients receiving stem cell treatments showed decreased levels of the protein CCL2, an indicator of inflammatory microglial cells, and increased levels of the protein MMP9, which plays a regenerative role in the central nervous system (CNS), compared to the control group.
“These findings represent a significant breakthrough for progressive multiple sclerosis 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,” said Dr. Saud A. Sadiq, Director and Chief Research Scientist of the Tisch Center. “This is the first time we’ve seen a treatment result in multiple areas of function for progressive multiple sclerosis patients. Furthermore, the identification of two new biomarkers provides another potentially valuable way for us to measure the efficacy of stem cell treatments moving forward.”
The Phase II trial, initiated in 2018, was a randomized, double-blind, placebo-controlled clinical study involving 54 patients. It featured a compassionate crossover design, where patients who received stem cell treatment in the first year were given the placebo in the second year, and vice versa. Patients received six injections of either autologous MSC-NPs (mesenchymal stem cell-derived neural progenitors) or saline every two months.
Highlighting Stem Cell Potential to Reverse Disability in Multiple Sclerosis
Dr. Violaine Harris, a principal investigator at the Tisch Center and lead author of the study, emphasized the potential of stem cells not only for treating multiple sclerosis but also for reversing disability after significant progression. “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,” she stated. “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 biomarkrs identified in our research.”
The Tisch Center’s earlier Phase I study, published in 2018, was the first to show the reversal of established disability in multiple sclerosis using stem cells. A follow-up study of Phase I patients revealed that a subset experienced sustained clinical improvement with no long-term side effects. Building on the promising results of the Phase II trial, the research team at the Tisch Center plans to explore higher dosages of stem cells and further investigate the newly identified biomarkers to enhance the understanding and efficacy of stem cell treatments for Progressive MS.
In conclusion, the results of the FDA-approved Phase II study represent a milestone in the treatment of progressive multiple sclerosis, offering hope for patients with severe disabilities. The findings suggest that stem cell therapy could improve multiple aspects of patient function and provide a new avenue for treating this challenging condition. The ongoing and future research at the Tisch MS Research Center aims to refine and expand these initial successes, potentially transforming the landscape of multiple sclerosis treatment.
Resource: BioMed Central, May 23, 2024
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