Wednesday, April 24, 2024
Regeneration Biomedical, Inc. (RBI), a company focused on developing stem cell therapies for neurodegenerative diseases, has reached a significant milestone by dosing the first patient in a Phase 1 clinical trial approved by the FDA. This trial aims to evaluate the use of autologous, Wnt-Activated Adipose-Derived Stem Cells (RB-ADSC) in individuals with mild-to-moderate Alzheimer's disease (AD).
Christopher Duma, M.D., F.A.C.S., president and founder of RBI, highlighted the urgent need for effective treatments for AD, a condition that imposes a heavy burden on patients, caregivers, and the healthcare system. Traditional treatment options have been inadequate, prompting exploration into novel therapies like stem cell treatment. However, the effectiveness of stem cell therapy has been limited due to challenges in bypassing the blood-brain barrier (BBB). RB-ADSC is designed to address this issue by delivering stem cells directly to the brain. Preclinical studies have shown promising results, suggesting that stem cells administered in this manner can reach targeted areas in the brain.
The Phase 1 trial, an open-label, single-arm study, aims to enroll nine subjects over one year to assess the safety of RB-ADSC infusion into the brain's lateral ventricles in individuals with mild-to-moderate AD. Secondary objectives include evaluating AD clinical assessments and biomarkers. Participants will be monitored for up to 12 months post-treatment. Collaboration with the FDA during pre-IND meetings has facilitated the development of both the investigational product and the infusion method, protected under an issued U.S. patent.
RB-ADSC, derived from a patient's own adipose tissue and activated with Wnt signaling, hold promise as a potential therapeutic option. These stem cells are cultured and expanded in vitro before being reintroduced into the patient via an Ommaya reservoir implanted under the scalp, providing direct access to the brain's lateral ventricles. Animal studies have shown successful distribution of infused stem cells into the brain parenchyma without adverse effects on cerebral spinal fluid circulation or inflammation.
The ultimate goal of this therapy is to activate dormant stem cells within the brain, stimulating repair and replacement of damaged neurons. Dr. Duma and his team remain optimistic about the trial's progress, with the first patient dosed in March 2024 and no reported adverse events to date. Enrollment continues, with plans to expand the use of RB-ADSC in Phase 2 trials for conditions such as multiple sclerosis, Parkinson's disease, traumatic brain injury, and amyotrophic lateral sclerosis (ALS), addressing critical unmet medical needs in these areas.
Source: globenewswire.com
