Regeneration Biomedical, Inc. announced encouraging data from its Phase 1 clinical trial of Wnt-activated, autologous, expanded, adipose-derived stem cells (RB-ADSCs) for Alzheimer's Disease (AD). The study, involving direct administration of RB-ADSCs into the brain's lateral ventricles, demonstrated the treatment's safety and potential efficacy in reducing key Alzheimer's biomarkers. The data were presented at the 17th Clinical Trials on Alzheimer’s Disease (CTAD) conference in Madrid.
Phase 1 Trial Details
The open-label, single-arm study enrolled nine subjects with mild-to-moderate AD, using a 3 + 3 single dose escalation design. The primary goal was to evaluate the safety of RB-ADSC infusion, with secondary endpoints including clinical assessments and biomarker analysis. Participants will be followed for 52 months post-treatment. The treatment involves harvesting adipose-derived stem cells from the patient, followed by in vitro expansion and Wnt activation before reintroduction into the patient's brain via an Ommaya reservoir.
Key Findings
The treatment was well-tolerated, with minor adverse events reported, such as bruising post-liposuction and mild incisional pain after Ommaya reservoir implantation. The injection process itself, lasting approximately eight minutes without anesthesia, was also well-tolerated. Cerebrospinal fluid (CSF) analysis at 12 weeks showed a decrease in p-Tau levels from a median of 60.2 pg/ml to 36.8 pg/ml. Amyloid PET scan centiloid scores also decreased from a median of 138.5 to 100.5 at 12 weeks. Furthermore, MMSE scores improved in two of the three evaluable patients, from a pre-injection median of 17 to 19.5 at week 12.
Expert Commentary
"Results from the first dose cohort increase our confidence in the safety of our RB-ADSC product candidate in Alzheimer’s disease patients," said Christopher Duma, M.D., F.A.C.S., president, founder of RBI and presenter of the data. "In addition, improvements in Alzheimer’s disease biomarkers and an improving trend in a cognitive measure at up to 12 weeks are consistent with our hypothesis that infusion of Wnt-activated stem cells into the brain may initiate a cessation or reversal of at least some of the pathological processes underlying this devastating disease."
Future Directions
Regeneration Biomedical has completed enrollment of the second dose cohort and is progressing with the third and final dose cohort. The company anticipates presenting the full data set at future medical conferences. This Phase 1 trial (NCT05667649) lays the groundwork for potential Phase 2 studies to further evaluate the efficacy of RB-ADSCs in treating Alzheimer's disease.
