Houston-area clinical research organization Hope Biosciences Research Foundation (HBRF) today shares positive top-line results of a Phase II clinical trial to evaluate Hope Biosciences’ allogeneic adipose‑derived mesenchymal stem cell therapy (HB-adMSCs) for patients with early to moderate Parkinson’s Disease (PD), a condition currently considered incurable that affects approximately one million in the U.S., with a projected global patient population of 25 million by 2050.

The trial successfully met its primary endpoint, demonstrating statistically significant improvements in motor function for the treatment group compared to the placebo group. The trial (NCT04995081) is a balanced randomized, double-blind, single center study that enrolled 60 participants, 30 in the treatment group and 30 in the placebo, mandating six intravenous infusions of 200 million stem cells over the course of 32 weeks. End of study was 52 weeks. Primary endpoints include clinically significant changes in motor function, using both the patient-reported Motor Experiences of Daily Living (MDS-UPDRS Part II) and clinician-rated Motor Function (MDS-UPDRS Part III).

At end of study, the clinician-rated MDS-UPDRS Part III demonstrated a progressive and clinically meaningful treatment effect. Improvements in the treatment group increased over successive infusions, with effect sizes growing over time (Cohen’s d: Infusion 4 = 0.34, Infusion 5 = 0.40, Infusion 6 = 0.87). By the sixth and final infusion, the treatment group achieved a mean change from baseline of −9.82 points versus −0.50 in placebo (RMA adjusted mean difference −9.32; 95% CI [−15.11, −3.54]; p=0.0023), substantially exceeding the MCID of −3.25. Bayesian analyses reinforced these findings, confirming statistical and clinical relevance. Treatment was safe and tolerable in both groups.

“The timing of patient improvements is emerging as a key takeaway,” says Donna Chang, President, HBRF. “Early infusions showed small improvements in motor function. By the sixth infusion, however, the treatment group showed the largest cumulative improvement. At end of study, we saw a decline in scores from that high point. Taken together, this means that improvements in motor function are possible through treatment with this cellular therapeutic, and that consistent, repeated treatment may be the most promising path forward for sustained enhancement in motor function for individuals living with Parkinson’s Disease.”

Chang also cites validation of protocol design as an achievement, noting divergent results between the clinician rating and the patient-reported MDS-UPDRS Part II.

“The subjective nature of patient reporting is something that must be balanced in trial design through inclusion of more objective data-gathering mechanisms that are vital for evaluating therapeutic benefit,” expounds Chang. “Taking all the data together, in this trial there is a clear treatment effect. We look forward to close out meetings with FDA, and a hopefully fruitful conversation about how we as a community of researchers can continue to balance patient- and clinician-reported outcomes.”

HBRF, a 501(c)(3) nonprofit organization, has to date successfully completed six FDA-authorized protocols in PD, including a global-first Intermediate Sized Expanded Access protocol for patients aged 76 years and older. This also constitutes HBRF’s second clinical trial using allogeneic cellular therapeutics. Both have completed safely, marking another step toward wider adoption of allogeneic therapies, which dramatically widen access due to reduced manufacturing costs and ability to serve individuals whose health conditions prohibit banking their own stem cells.

“We have had the privilege of serving a significant number of men and women living with Parkinson’s, with extremely diverse disease stories,” continues Chang. “For this trial, we are eager not only for results of the detailed analysis currently underway, but also to look across the research suite for trends that may inform treatment pathways for Parkinson’s in the future. With these encouraging Phase II results in hand, we are hopeful to advance to a Phase III confirmatory trial that could bring us closer to a meaningful new therapeutic option for patients.”

Learn more at hopebio.org.

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Improvements in motor function are possible through treatment with this cellular therapeutic, and that consistent, repeated treatment may be the most promising path forward for sustained enhancement in motor function for individuals living with Parkinson's