First FDA-Approved Phase II Stem Cell Treatment Shows Significant Improvements in Progressive MS

The Tisch MS Research Center of New York, recognized as the world’s largest multiple sclerosis (MS) research institution, has released the findings from its FDA-approved Phase II stem cell treatment study. The results, published in the journal Stem Cell Research & Therapy, concentrated on progressive MS patients, the most debilitating form of the disease. Although the primary endpoint of the study was not achieved, there were notable improvements in various domains such as walking speed, bladder function, and gray matter volume in patients with significant disabilities.

This pioneering research represents the first Phase II placebo-controlled, double-blind stem cell trial in the U.S. aimed at evaluating stem cell-derived treatments for disabled patients with progressive MS who do not have active lesions or ongoing relapses, conditions that often leave them without effective treatment options.

Key results from the study indicate that stem cell treatments could enhance the quality of life for progressive MS patients. Among the significant findings:

1. Improved Walking Speed: Patients requiring assistance to walk who received stem cell treatments exhibited a 3.7% increase in their walking speed during the timed 25-foot walk test, in stark contrast to a 54% decrease seen in the placebo group. These results were consistent with a six-minute walking test also administered to participants.

2. Enhanced Bladder Function: After one year of treatment, 76% of patients who received stem cell treatments reported improved bladder function, compared to just 27% in the placebo group.

3. Preservation of Gray Matter Volume: In patients with gray matter volume above the 50th percentile, the placebo group experienced a decrease in gray matter volume, unlike those who received stem cell treatments. This suggests that stem cell treatments may help preserve gray matter volume in patients with less advanced disease progression.

Additionally, the study identified two new biomarkers that could serve as surrogate measures for treatment response. Patients receiving stem cell treatments exhibited decreased levels of the protein CCL2, potentially indicating a reduction in inflammatory microglial cells, and an increase in the protein MMP9, which plays a regenerative role in the central nervous system.

Dr. Saud A. Sadiq, Director and Chief Research Scientist of the Tisch Center, highlighted the significance of these findings for progressive MS patients who suffer from the most disabling symptoms and have limited treatment options. The results suggest meaningful quality-of-life improvements for this patient group and the identification of new biomarkers offers a valuable method for assessing the efficacy of stem cell treatments in future studies.

The Phase II trial, which started in 2018, involved 54 patients and employed a randomized, double-blind, placebo-controlled design with a compassionate cross-over approach. This meant that patients who received the stem cell treatment in the first year received a placebo in the second year, and vice versa. Each patient received six injections, administered every two months, of either autologous mesenchymal stem cell-derived neural progenitors (MSC-NPs) or saline.

Dr. Violaine Harris, a principal investigator at the Tisch Center and the lead author of the study, emphasized the potential effectiveness of stem cells not only in treating MS but also in reversing disability after significant disease progression. The research team is eager to build on these findings, particularly by increasing the dosage of stem cells and further investigating the response of the newly identified biomarkers.

This study is part of a broader effort by the Tisch MS Research Center to find the cause and cure for multiple sclerosis. The Center’s ongoing research spans from basic cellular and molecular experimentation to clinical trials aimed at preventing or reversing disability in MS patients.

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