California-based Regeneration Biomedical has dosed the first patient has been dosed in a first-in-human trial of its autologous stem cell treatment, RB-ADSC, for patients with mild-to-moderate Alzheimer’s disease.

The Phase I trial (NCT05667649) of the candidate bypasses the blood-brain barrier (BBB) which most approved and investigated treatments pass through, with Regeneration’s stem cell treatment, RB-ADSC, being injected directly into the ventricular system.

RB-ADSCs are Wnt-activated adipose-derived stem cells obtained from a patient’s own adipose tissue. The cells are cultured and expanded in vitro, selected for Wnt expression, and then reintroduced into the patient’s brain.

The Phase I trial is an open-label, single-arm study which will enroll nine patients over one year. Patients will be randomised 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, as well as to determine a recommended dose for a potential Phase II clinical trial.

Secondary endpoints include AD clinical assessments and biochemical and anatomical biomarkers. Each patient will be followed for up to 12 months after treatment.

Founder of Regeneration Biomedical, Christopher Duma, said: 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 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.”

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Duma said the company hopes that the therapy will ‘turn on’ the stem cells that are sitting dormant in the brain to initiate repair and replacement of damaged neurons.

The company has plans to investigate the therapy in other neurodegenerative diseases at Phase II including multiple sclerosis, Parkinson’s disease, traumatic brain injury and amyotrophic lateral sclerosis.

Alzheimer’s landscape

Alzheimer’s disease is a chronic neurodegenerative disease that usually starts slowly in people aged over 65 and worsens over time. It is the most common cause of dementia, accounting for 60% to 70% of all cases. Estimates suggest that as many as 12.4 million patients in the US could have the disease by 2050.

Currently, the only disease-modifying treatments on the market are amyloid targeting treatments Leqembi (lecanemab) and Aduhelm (aducanemab), however, Biogen has decided to discontinue Aduhelm, returning the rights for the drug back to the original developer Neurimmune.

Eli Lilly was hoping that its amyloid targeting therapy donanemab would be on the market by now however it has faced a couple of set back by the US Food and Drug Administration (FDA), with the agency now wanting an Adcomm to further evaluate the ‘unique trial design’.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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