FDA-Approved Phase II Stem Cell Treatment Trial Results Presented at AAN

Tuesday, April 25, 2023

FDA-Approved Phase II Stem Cell Treatment Trial Shows Significant and Diverse Improvements for Multiple Sclerosis (MS) Patients


The study, from the Tisch MS Research Center of New York, is the first to link stem cells with a broad array of improvements in multiple areas


Progressive MS patients demonstrated better outcomes in walking speeds, gray matter volume, bladder function, and biomarker indicators


The Tisch MS Research Center of New York, the world’s largest independent research center focused on MS, today announced the Phase II results of its FDA-approved stem cell treatment study. The research is a continuation of a Phase I trial, which was the first ever in the U.S. to receive FDA approval to investigate the injection of stem cells into the cerebrospinal fluid of MS patients. The Phase II trial showed improvements across multiple areas in progressive MS patients with significant disability.

“The findings of this study suggest that stem cells could have major implications for treating MS and reversing patient disability even after significant progression”

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Among patients who require walking assistance, those who received stem cell treatments saw a significantly higher percentage of improvement in both a timed 25-foot walk test and a 6-minute walking test than those in the control group. These results suggest that stem cell treatments could be correlated with walking improvement, a finding that could have major implications for quality of life for progressive MS patients struggling with motor function.

The study also found a positive increase in gray matter volume among patients who began the trial with normalized gray matter above the 50th percentile. This indicates that stem cells could restore neuronal cells and potentially reverse cognitive decline in patients with less advanced disease progression. Additionally, patients who received the stem cell treatments showed improved bladder function, with 69% of patients showing improved post-void residual volume.

The stem cell treatments also resulted in notable biomarker changes in patients’ cerebrospinal fluid. Those who received the stem cell treatments showed decreased levels of the protein CCL2 (which is associated with inflammatory diseases like MS), as well as an increase in the protein MMP9 (a potential indicator of an increase in reparative microglial cells) compared to the control group. As a result, these biomarkers may provide a valuable mechanism to measure treatment response in future studies.

“These findings are unprecedented in the realm of researching stem cells as a treatment pathway for progressive MS patients. They represent the first time we have seen a regenerative treatment improve patient outcomes in multiple areas, providing strong evidence of the potential efficacy of stem cells in patients with significant and varying disabilities,” said Saud A. Sadiq, Director and Chief Research Scientist of the Tisch Center. “We are particularly encouraged by the improvements in walking speed among patients who require assistance, which suggests that stem cells can make a potentially life-altering difference for these patients. Furthermore, the biomarker changes observed in the study show biochemical evidence of the reparative nature of stem cell treatments and can provide a valuable metric for us to further study their impact.”

In 2014, the Phase I results of the Tisch Center’s stem cell treatment study were the first ever to show reversal of established disability in MS from stem cells. A follow-up study of those patients from Phase 1 showed that a subset of the patients sustained clinical improvement and that there were no long-term side effects for any of the participants.

The Phase II trial began in 2018 and was a randomized, double-blind, placebo-controlled clinical study with 54 patients. The study had a compassionate cross-over design, in which patients who received the stem cell treatment in the first year received the placebo in the second year (and vice versa). Patients received six injections (of either autologous MSC-NPs or saline injections) every two months.

As a next step, the Center is exploring an investigation of the effects of increasing the stem cell dosage that patients receive.

“The findings of this study suggest that stem cells could have major implications for treating MS and reversing patient disability even after significant progression,” added Dr. Violaine Harris, a principal investigator at the Tisch Center and lead author of the study. “We look forward to building on these findings in future research and potentially studying the effects of increasing the dosage of stem cells administered to patients.”


About the Tisch MS Research Center of New York

The mission of the Tisch Multiple Sclerosis Research Center of New York is to conduct groundbreaking medical research to ensure unparalleled care and positive outcomes for MS patients. Its integrated relationship with the International MS Management Practice (IMSMP) accelerates the pace at which research discoveries translate from lab bench to bedside. The Center aims to identify the cause of MS, understand disease mechanisms, optimize therapies, and repair the damage caused by MS while offering patients access to the best and most advanced treatments possible.

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