Hope Biosciences has linked its stem cell therapy candidate to improved motor function in Parkinson’s disease patients in a phase 2 trial.
The single-site study randomized 60 people with early to moderate Parkinson’s to receive six infusions of Hope’s allogeneic adipose‑derived mesenchymal stem cell therapy or placebo across 32 weeks. Interest in using the stem cells in Parkinson’s is based on evidence that they contribute to neuroprotection and neuroregeneration, differentiate into neural progenitor cells and migrate to sites of neurodegeneration.
ClinicalTrials.gov lists MDS-UPDRS Part II—a patient-reported motor function scale—as the primary efficacy endpoint. The other 16 primary endpoints cover safety and tolerability. Presenting the data, Hope said MDS-UPDRS Part II and the clinician-rated MDS-UPDRS Part III were primary endpoints.
The press release focuses on Part III. By the final infusion, the treatment group had a mean reduction on the clinician-rated scale of 9.82 points versus 0.5 for placebo. Hope said a 3.25-point reduction is the minimal clinically important difference. Part III scores fell across the six infusions, only for the trend to reverse after the final dose.
“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,” Donna Chang, president of the study’s sponsor the Hope Biosciences Research Foundation, said in a statement.
Chang’s optimism is based on the clinician-rated Part III results. The press release lacks data from the Part II assessment, but Chang noted “divergent results” between the clinician-rated and patient-reported scales. Chang presented the divergent findings as validation of the study design rather than a question mark over the intervention’s efficacy.
“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,” Chang said. “Taking all the data together, in this trial there is a clear treatment effect.”
Hope is preparing for close-out meetings with the FDA. Chang said the team is “hopeful to advance to a phase 3 confirmatory trial” of the stem cell therapy candidate.