Houston-based Hope Biosciences Research Foundation (HBRF) has announced positive top-line results from its Phase II clinical trial evaluating adipose-derived autologous mesenchymal stem cell therapy (HB-adMSCs) for mild to moderate relapsing-remitting multiple sclerosis (MS). The trial's findings suggest a potential new approach for managing this currently incurable disease, which affects over 2 million people worldwide.
The randomized, double-blind, single-center study (NCT05116540) enrolled 24 participants, with 12 receiving HB-adMSCs and 12 receiving a placebo. Participants in the treatment group received six intravenous infusions of 200 million stem cells over 32 weeks, totaling 1.2 billion cells. The primary endpoint was the MS Quality of Life-54 Instrument, a comprehensive measure assessing various aspects of quality of life.
Significant Improvements in Quality of Life
The HB-adMSC group demonstrated a statistically significant improvement from baseline in their Physical Health Composite Scores (p<0.0001) compared to the placebo group (p<0.4856). The effect size between the groups was large (Cohen’s d=1.23), with a significant overall treatment difference (p=0.0002). Mental Health Composite Scores also showed significant improvement in the HB-adMSC group (p<0.0042) compared to the placebo group (p<0.5724), with a substantial effect size (Cohen’s d=0.85) and a statistically significant overall treatment difference (p=0.016).
Safety and Tolerability
The treatment was reported to be safe and tolerable in both the treatment and placebo groups, indicating that the stem cell therapy did not pose significant risks to the participants.
Implications and Future Directions
"The results of this trial are groundbreaking for multiple sclerosis. They clearly demonstrate that high doses of fresh HB-adMSCs delivered on a regular schedule can result in consistent efficacy in a highly complex and variable condition like MS," said Donna Chang, President of HBRF. She also expressed optimism that this positive response could translate to other autoimmune diseases in the future.
These findings offer a promising outlook for individuals with relapsing-remitting MS, potentially providing a novel therapeutic option to improve their quality of life. Further detailed analysis of the trial data is currently underway to explore the full scope of the treatment's effects.
