Regeneration Biomedical reports Phase I Alzheimer’s stem cell results and FDA Phase II clearance

By AI, Created 5:26 PM UTC, May 18, 2026, /AGP/ – Regeneration Biomedical says its direct-to-brain stem cell therapy produced biomarker and cognitive improvements in a small Phase I Alzheimer’s study, with effects lasting up to a year after one treatment. The FDA has cleared the company to begin a Phase II trial as it expands testing of the investigational therapy.

Why it matters: - Regeneration Biomedical’s early data suggest a one-time direct-to-brain stem cell treatment may affect multiple Alzheimer’s disease pathways at once. - The company says the Phase I findings were strong enough for FDA clearance to move into a Phase II study. - The results could matter because Alzheimer’s treatments that target only amyloid have had limited impact on broader disease biology.

What happened: - Regeneration Biomedical reported Phase I findings from an FDA-authorized trial of RB-ADSC, a Wnt-activated autologous adipose-derived stem cell therapy. - The study tested intracerebroventricular delivery, meaning the cells were injected into the brain’s ventricular system through an Ommaya reservoir. - The company presented the data at the International Society for Cell & Gene Therapy Annual Meeting in Dublin, May 6-9, 2026. - The trial enrolled six people ages 45-80 with FAST stage 4-5 Alzheimer’s disease. - Participants received either 2 million or 5 million RB-ADSCs after lipoaspiration and reservoir placement. - The FDA cleared Regeneration Biomedical to proceed into Phase II after reviewing the Phase I safety and biomarker findings.

The details: - Median CSF p-Tau fell 56.9% from baseline to 12 weeks. - Median CSF total tau fell 54.6% over the same period. - Median amyloid PET centiloid scores dropped 23.6% at 12 weeks. - Median ADAS-Cog-13 scores improved 13.8% at 12 weeks. - At 52 weeks, median CSF p-Tau remained down 65.4% from baseline. - At 52 weeks, median CSF total tau remained down 62.8% from baseline. - Median amyloid PET centiloid scores stayed below baseline at one year. - The company also reported favorable changes in CSF proteomic markers tied to neuroinflammation, neuronal injury, cellular stress signaling and extracellular histone biology. - Regeneration Biomedical said the biomarker shifts also included amyloid beta, histone-related neurodegenerative signaling markers and inflammatory proteomic signatures. - No treatment-related adverse events, ARIA events or serious adverse events were seen through 16 months of follow-up. - The therapy uses ex vivo-expanded autologous stem cells activated with a proprietary Wnt-signaling protocol. - Regeneration Biomedical says the direct CSF route is designed to bypass the blood-brain barrier and create broad CNS exposure.

Between the lines: - The company is emphasizing breadth, not just plaque reduction, as the main signal from the trial. - The data are being framed as evidence of possible regenerative and immunomodulatory effects, but the study was very small and early-stage. - The company’s comparison with antibody drugs such as Leqembi and Kisunla highlights a potential differentiation strategy, not a head-to-head trial result. - Because RB-ADSC is investigational, the findings do not prove the treatment will work in larger randomized studies.

What’s next: - Regeneration Biomedical is preparing a randomized, placebo-controlled Phase II trial in Alzheimer’s disease. - The company plans to study efficacy, durability, repeat dosing and broader proteomic responses in a larger population. - RBI also plans to explore the same direct-to-brain platform in chronic traumatic encephalopathy, amyotrophic lateral sclerosis, multiple sclerosis and Parkinson’s disease.

The bottom line: - Regeneration Biomedical’s small Phase I study points to possible multi-pathway Alzheimer’s effects from a single intracerebroventricular stem cell dose, and the FDA has now allowed the program to advance.