Regeneration Bio commences trial of Alzheimer’s stem cell therapy delivered directly into the brain

Company hopes to ‘turn on’ dormant stem cells in the brain to initiate repair and replacement of damaged neurons.

Stem cell therapy company Regeneration Biomedical revealed has dosed the first patient in a Phase 1 clinical trial to evaluate the safety and efficacy of its therapy for patients with mild-to-moderate Alzheimer’s disease. Stem cells have long been thought to have regenerative potential in the brain, but therapies have been hindered by the blood-brain barrier, so the company is aiming to overcome this challenge by taking a more direct approach.  

Regeneration Bio’s stem cell therapy, known as RB-ADSC, uses autologous, Wnt-activated adipose-derived stem cells, which means they are derived from a patient’s own fat cells. Crucially, the therapy bypasses the blood-brain barrier and delivers stem cells directly into the brain via an “Ommaya reservoir” – a plastic device implanted under the scalp. The method targets the lateral ventricles of the brain, facilitating the distribution of stem cells into the functional tissue of the brain.

“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 Christopher Duma, founder of Regeneration Biomedical.

RB-ADSC undergoes a specialized manufacturing process to enhance its therapeutic potential. Cultured and expanded in vitro, the stem cells are selected for Wnt expression, a signaling protein that stimulates communication between stem cells. The optimized therapy is then reintroduced directly into the patient’s brain via the implanted reservoir.

Preclinical studies in animal models have shown promising results, with stem cells effectively distributing into the brain parenchyma without adverse effects on cerebral spinal fluid circulation. These findings, combined with positive outcomes from earlier pilot studies, provide the foundation for commencing the Phase 1 trial.

“Our RB-ADSC product candidate is designed to overcome the blood brain barrier by delivering potentially efficacious stem cells directly to the brain,” added Duma. “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.”

The Phase 1 trial, cleared by the FDA, will enroll nine subjects over a year, employing a dose escalation design to assess safety and determine an optimal dosage for further studies. Secondary endpoints include clinical assessments and biomarker analysis to gauge treatment efficacy.

“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, CEO of Regeneration Biomedical. “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.”

“The first patient in this trial was dosed in March 2024, and no side effects or adverse events have been reported to date,” said Dr Gustavo Alva, the trial’s principal investigator. “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.”

Photograph: Image-Source/Envato