Patients in an ongoing Phase I clinical trial of a new stem cell therapy for Alzheimer’s disease (AD) are showing a reduction in proteins linked to disease progression and improvements in cognitive scoring. This is according to preliminary data from Regeneration Biomedical, a clinical-stage company that develops autologous stem cell therapies for neurodegenerative diseases, including AD.
Regeneration is presenting updated interim results from the Phase I trial of its treatment at the scientific annual meeting of the International Society for Cell & Gene Therapy (ISCT) taking place in New Orleans from May 7–10, 2025. The company’s abstract was also selected for a Host Region award at the meeting.
At the meeting, Christopher Duma, MD, president and founder of Regeneration Biomedical, will present preliminary data from the first five patients to receive the company’s Wnt-activated, autologous, expanded, adipose-derived stem cells (RB-ADSCs). The company shared data from two patients last year.
As the name implies, the stem cells are obtained from patients’ own adipose tissue, cultured and expanded in vitro, and then selected for Wnt expression. The treatment is designed to stimulate the activation and maintenance of stem cells in regenerative regions of the brain while reducing inflammation. Selected stem cells are injected directly into patients’ brains using a device implanted under the scalp. The device provides direct access to the brain’s lateral ventricles, thus bypassing the blood-brain barrier.
The trial is being conducted at Hoag Memorial Hospital Presbyterian in Newport Beach, CA. Regeneration is enrolling nine patients with mild-to-moderate Alzheimer’s disease and using a 3 + 3 single-dose escalation design to assess the safety of stem cells infused into the lateral ventricles of the brain. The study aims to determine a recommended dose for a potential Phase II trial. Secondary endpoints include Alzheimer’s disease clinical assessments, along with biochemical and anatomical biomarkers. Each participant will be followed for up to 12 months post-treatment.
According to some key findings from the trial, 80% of patients had reduced p-tau levels from a median of 60.2 pg/mL pre-injection (range: 46.9–76.1) to a median of 36.8 pg/mL (range: 15.0–66.6) at 12 weeks. The scientists also saw improvements in patients’ mini-mental state examination (MMSE) scores and their Alzheimer’s disease assessment scale—cognitive subscale (ADAS-Cog) scores. In terms of the MMSE scores, 60% of patients improved from a median score of 16 pre-injection (range: 14–19) to a median score of 18 (range: 12–20). For their ADAS-Cog scores, 80% of patients improved from a median of 53 pre-injection (range: 40–69) to a median score of 38 (range: 20–69).
Importantly, there were no immediate or delayed adverse effects reported at least 23 weeks post-injection.
Compared to current monoclonal antibody therapies, Gustavo Alva, MD, principal investigator of the trial at Hoag Hospital, noted that these results “suggest that regenerative therapies like RB-ADSCs may offer a superior safety profile and a more comprehensive approach with meaningful benefits for patients living with this devastating disease.”
Duma added that Regeneration is continuing to advance its clinical program for AD while also considering other targets for its stem cell therapies. “We are actively exploring next steps, including a Phase II trial, and see potential opportunities to investigate this approach in other neurodegenerative diseases in the future, pending further data and regulatory guidance,” he said.
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Regeneration is presenting updated interim results from the Phase I trial of its treatment at the scientific annual meeting of the International Society for Cell & Gene Therapy (ISCT) taking place in New Orleans from May 7–10, 2025. The company’s abstract was also selected for a Host Region award at the meeting.
At the meeting, Christopher Duma, MD, president and founder of Regeneration Biomedical, will present preliminary data from the first five patients to receive the company’s Wnt-activated, autologous, expanded, adipose-derived stem cells (RB-ADSCs). The company shared data from two patients last year.
As the name implies, the stem cells are obtained from patients’ own adipose tissue, cultured and expanded in vitro, and then selected for Wnt expression. The treatment is designed to stimulate the activation and maintenance of stem cells in regenerative regions of the brain while reducing inflammation. Selected stem cells are injected directly into patients’ brains using a device implanted under the scalp. The device provides direct access to the brain’s lateral ventricles, thus bypassing the blood-brain barrier.
The trial is being conducted at Hoag Memorial Hospital Presbyterian in Newport Beach, CA. Regeneration is enrolling nine patients with mild-to-moderate Alzheimer’s disease and using a 3 + 3 single-dose escalation design to assess the safety of stem cells infused into the lateral ventricles of the brain. The study aims to determine a recommended dose for a potential Phase II trial. Secondary endpoints include Alzheimer’s disease clinical assessments, along with biochemical and anatomical biomarkers. Each participant will be followed for up to 12 months post-treatment.
According to some key findings from the trial, 80% of patients had reduced p-tau levels from a median of 60.2 pg/mL pre-injection (range: 46.9–76.1) to a median of 36.8 pg/mL (range: 15.0–66.6) at 12 weeks. The scientists also saw improvements in patients’ mini-mental state examination (MMSE) scores and their Alzheimer’s disease assessment scale—cognitive subscale (ADAS-Cog) scores. In terms of the MMSE scores, 60% of patients improved from a median score of 16 pre-injection (range: 14–19) to a median score of 18 (range: 12–20). For their ADAS-Cog scores, 80% of patients improved from a median of 53 pre-injection (range: 40–69) to a median score of 38 (range: 20–69).
Importantly, there were no immediate or delayed adverse effects reported at least 23 weeks post-injection.
Compared to current monoclonal antibody therapies, Gustavo Alva, MD, principal investigator of the trial at Hoag Hospital, noted that these results “suggest that regenerative therapies like RB-ADSCs may offer a superior safety profile and a more comprehensive approach with meaningful benefits for patients living with this devastating disease.”
Duma added that Regeneration is continuing to advance its clinical program for AD while also considering other targets for its stem cell therapies. “We are actively exploring next steps, including a Phase II trial, and see potential opportunities to investigate this approach in other neurodegenerative diseases in the future, pending further data and regulatory guidance,” he said.
The post Regeneration Biomedical’s Cell Therapy Reduces Tau, Boosts Cognition in Alzheimer’s Cases appeared first on GEN - Genetic Engineering and Biotechnology News.