Regeneration Biomedical Doses First Patient in a First-in-Human Phase 1 Clinical Trial of Stem Cell Therapy Delivered Directly Into the Brain of Patients With Alzheimer’s Disease

Regeneration Biomedical, Inc. recently announced the first patient has been dosed in an FDA-cleared, first-in-human Phase 1 clinical trial of autologous, Wnt-Activated Adipose-Derived Stem Cells (RB-ADSC) for patients with mild-to-moderate Alzheimer’s disease (AD).

“Alzheimer’s disease remains highly prevalent, and a tremendous burden on patients, their caregivers and the broader healthcare system,” said Christopher Duma, MD, FACS, President, founder of RBI and inventor of the process. “Despite recent advances, current treatment options are wholly inadequate, leaving a major unmet medical need. Stem cells have represented a novel approach to treatment, but evidence of efficacy has been elusive because systemically administered cells are unable to bypass the blood brain barrier (BBB) and enter the brain. Our RB-ADSC product candidate is designed to overcome the BBB by delivering potentially efficacious stem cells directly to the brain. In vivo studies demonstrated that stem cells injected in this fashion do locate into the ventricles and parenchyma. In addition, an IRB-approved pilot study of an earlier version of our stem cell product in patients with advanced neurodegenerative diseases generated promising results that were consistent with our hypothesis, suggesting that infusion of stem cells directly into the brain may have utility in AD and potentially other neurodegenerative diseases. We are looking forward to the results of this first-in-mankind clinical trial.”

This FDA-cleared, Phase 1 open-label, single-arm study will enroll nine subjects over one year randomized into a 3 + 3 dose escalation design to evaluate the safety of autologous RB-ADSC infused into the lateral ventricles of the brain in subjects with mild-to-moderate AD, and to determine a recommended dose for a potential Phase 2 clinical trial. Secondary endpoints include AD clinical assessments and biochemical and anatomical biomarkers. Each participant will be followed for up to 12 months after treatment. More information on the clinical trial can be found at NCT05667649.

“We worked closely with the FDA in pre-IND meetings to create a development path for both our novel investigational product and our method for infusion, which is covered under an issued US patent,” said Bill Miller, Chief Executive Officer of RBI. “This clinical trial is a milestone achievement not just for us but for the field of stem cell therapies, and it gives us hope for the patients who suffer from neurodegenerative conditions for which there are no current cures.”

RB-ADSC are Wnt-activated adipose-derived stem cells obtained from a patient’s own adipose tissue. After collection, the stem cells are cultured and expanded in vitro, selected for Wnt expression, which is a signaling protein known to stimulate stem cells to communicate with other stem cells, and then reintroduced into the same patient via an Ommaya reservoir implanted under the scalp, which has direct access to one of the lateral ventricles located in the brain. Research in two animal models has demonstrated that stem cells infused into the ventricular system distribute themselves into brain parenchyma. In addition, no inflammation, obstruction of cerebral spinal fluid circulation or other safety signals were observed.

“The hope is that our cells will ‘turn on’ the stem cells that are sitting dormant in all of our brains, to initiate repair and replacement of damaged neurons,” said Dr. Duma.

Gustavo Alva, MD, principal investigator of the trial, added “The first patient in this trial was dosed in March 2024, and no side effects or adverse events have been reported to date. Enrollment is ongoing and we look forward to completing the trial and sharing its results. In Phase 2, we plan to use the same cells for other diseases with unmet or poorly met needs such as multiple sclerosis, Parkinson’s disease, traumatic brain injury and amyotrophic lateral sclerosis.”

Regeneration Biomedical, Inc. leverages world-class capabilities in stem cell therapy development and clinical application to develop autologous stem cell treatments for neurodegenerative diseases. Our proprietary therapy is derived from a patient’s own fat cells, which is a first-in-class Adipose-Derived Stem Cell (ADSC) population. We also employ a proprietary isolation-expansion-enrichment manufacturing protocol to synthesize the therapy and inject it directly into the brain. For more information, visit www.regenerationbiomedical.com.