Trial for Regeneration Biomedical’s Alzheimer Disease Stem Cell Therapy Doses First Patient

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The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year.

The first patient has been dosed in Regeneration Biomedical’s phase 1 clinical trial (NCT05667649) for RB-ADSC, an autologous, Wnt-activated adipose-derived stem cell therapy, for the treatment of Alzheimer Disease (AD).1

The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year, to be treated in a 3+3 dose escalation manner. The trial will evaluate safety and seek to establish a recommended dose for a possible phase 2 trial, with AD clinical assessments and evaluations of biochemical and anatomical biomarkers serving as secondary outcome measures. The study is recruiting patients aged 45 to 80 years of age with mild-to-moderate AD, with plans to follow patients for up to 12 months posttreatment.

“The first patient in this trial was dosed in March 2024, and no side effects or adverse events have been reported to date,” Gustavo Alva, MD, the principal investigator of the trial, said in a statement.1 “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.”

RB-ADSC consists of stem cells derived from patient’s own adipose tissue via lipoaspirate that have been expanded ex vivo with selection for expression of the signaling protein Wnt. RB-ADSC is delivered to patients directly to the lateral ventricles of the brain through an Ommaya reservoir implanted in the scalp. Based on results from 2 animal models used in preclincal research, Regeneration Biomedical expects that the RB-ADSC cells will distribute into the brain parenchyma. The company noted that there was no inflammation, obstruction cerebral spinal fluid circulation, or any other safety signal seen in the animal models.

Key Takeaways

  • The first patient has been dosed in Regeneration Biomedical’s phase 1 clinical trial (NCT05667649) for RB-ADSC, an autologous, Wnt-activated adipose-derived stem cell therapy, for the treatment of Alzheimer Disease (AD).
  • The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year, to be treated in a 3+3 dose escalation manner.
  • RB-ADSC consists of stem cells derived from patient’s own adipose tissue via lipoaspirate that have been expanded ex vivo with selection for expression of the signaling protein Wnt. RB-ADSC is delivered to patients directly to the lateral ventricles of the brain through an Ommaya reservoir implanted in the scalp.

“AD remains highly prevalent, and a tremendous burden on patients, their caregivers and the broader healthcare system,” Christopher Duma, MD, FACS, the president, founder of Regeneration Biomedical. “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 institutional review board-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.”

Regeneration Biomedical is not the only company seeking to bring cell therapy to AD. Neurona Therapeutics’ allogeneic regenerative neural cell therapy, NRTX-1001, is currently being evaluated in first-in-human phase 1/2 clinical trial (NCT05135091) for the treatment of patients with mesial temporal lobe epilepsy.2 Although, Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, told CGTLive® in a December 2023 interview that the company eventually hopes to evaluate NRTX-1001 in patients with AD.

NKGen is also evaluating SNK01, an autologous natural killer cell therapy, in patients with AD in the phase 1 ASK-AD clinical trial (NCT04678453).3 The company reported at the Clinical Trials on Alzheimer’s Disease (CTAD) Annual Meeting, held October 24 to 27, 2023, in Boston, Massachusetts, that among 10 patients evaluated compared to their baseline at 1 week after receiving their final dose in the trial, 30% of patients demonstrated clinical improvement on Alzheimer’s disease composite score (ADCOMS), 60% of patients demonstrated a stable ADCOMS score, and one patient who previously had an ADCOMS score classified as moderate achieved an ADCOMS score classified as mild.

REFERENCES
1. Regeneration Biomedical Doses First Patient in a First-in-Human Phase I Clinical Trial of Stem Cell Therapy delivered directly into the brain of Patients with Alzheimer’s Disease. News release. Regeneration Biomedical, Inc. April 23, 2024. Accessed April 27, 2024. https://www.globenewswire.com/news-release/2024/04/23/2867508/0/en/Regeneration-Biomedical-Doses-First-Patient-in-a-First-in-Human-Phase-I-Clinical-Trial-of-Stem-Cell-Therapy-delivered-directly-into-the-brain-of-Patients-with-Alzheimer-s-Disease.html
2. Neurona Therapeutics presents new clinical data from first cohort in ongoing phase I/II open-label trial of NRTX-1001 cell therapy for drug-resistant mesial temporal lobe epilepsy (MTLE). News release. Neurona Therapeutics. December 1, 2023. Accessed April 27, 2024. https://www.neuronatherapeutics.com/news/press-releases/120123/
3. NKGen Biotech presented phase I clinical trial data at the 16th Annual Clinical Trials on Alzheimer’s Disease (CTAD) conference. News release. NKGen Biotech Inc. October 26, 2023. Accessed April 27, 2024. https://nkgenbiotech.com/nkgen-biotech-presented-phase-i-clinical-trial-data-at-the-16th-annual-clinical-trials-on-alzheimers-disease-ctad-conference/
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