Regeneration Biomedical Shares Phase I Stem Cell Trial Data for Alzheimer's

NEWPORT BEACH, CA, USA I October 29, 2024 I Regeneration Biomedical, Inc. (RBI), a company engaged in developing autologous stem cell treatments for neurodegenerative diseases, has shared promising results from a Phase 1 clinical trial. The trial involved the administration of Wnt-activated, autologous, expanded, adipose-derived stem cells (RB-ADSCs) directly into the lateral ventricles of the brain in patients suffering from Alzheimer’s Disease (AD). The initial dose cohort was well tolerated by the patients, with notable reductions in levels of p-Tau and beta amyloid observed after 12 weeks. Additionally, results from the Mini-Mental State Examination (MMSE) indicated a trend toward improved cognition in two out of three subjects. These findings were presented at the 17th Clinical Trials on Alzheimer’s Disease (CTAD) conference in Madrid, held on October 29 and 30, 2024.

Christopher Duma, M.D., F.A.C.S., the president and founder of RBI, presented the data. He expressed increased confidence in the safety profile of the RB-ADSC product candidate for Alzheimer’s patients based on the observed results. The improvements in Alzheimer’s biomarkers and cognitive measures at 12 weeks supported the hypothesis that infusing Wnt-activated stem cells into the brain may help halt or reverse some pathological processes associated with Alzheimer’s Disease. The company has completed the enrollment for their second dose cohort and is now moving forward with the third and final dose cohort. Full data from this study is expected to be presented at upcoming medical conferences.

The Phase 1 study is an open-label, single-arm trial, designed to enroll nine subjects over approximately one year. It follows a 3 + 3 single dose escalation design to assess the safety of autologous RB-ADSCs infused into the lateral ventricles of the brain in subjects with mild-to-moderate AD. The trial aims to establish a recommended dose for a potential Phase 2 clinical trial. Secondary endpoints include clinical assessments related to AD, as well as biochemical and anatomical biomarker evaluations. Each participant will be monitored for up to 52 months post-treatment.

RB-ADSCs are derived from a patient's own adipose tissue, which undergoes a collection process followed by in vitro culture and expansion. The stem cells are selected for Wnt protein expression, a signaling protein that promotes communication between stem cells. These selected stem cells are then reintroduced into the patient via an Ommaya reservoir implanted under the scalp, providing direct access to the brain's lateral ventricles.

The treatment process, including the liposuction procedure to harvest adipose-derived stem cells, was generally well tolerated. Minor adverse events reported included slight bruising and discomfort post-liposuction, and mild pain following Ommaya reservoir implantation. The injection process, which took an average of eight minutes and did not require anesthesia, was also well tolerated, with no adverse events reported for at least 11 weeks post-injection.

Cerebrospinal fluid (CSF) analysis at 12 weeks in all three patients showed a decrease in p-Tau levels from a median of 60.2 pg/ml to a median of 36.8 pg/ml. Additionally, pre-injection amyloid PET scan centiloid scores decreased from a median of 138.5 to 100.5 at 12 weeks. MMSE scores showed improvement, with median scores rising from 17 pre-injection to 19.5 at week 12 in two out of three evaluable patients.

Regeneration Biomedical, Inc. focuses on developing autologous stem cell treatments for neurodegenerative diseases, leveraging world-class capabilities in stem cell therapy development. The proprietary therapy uses a patient's own adipose-derived stem cells, employing a unique isolation-expansion-enrichment protocol for synthesis and direct brain injection.