Stem Cell Therapy for Alzheimer’s Granted FDA Fast Track as It Advances to Phase 2

Key details

• A large unmet need: an estimated 1 million people in the UK are living with dementia, and there are currently no treatments available on the NHS that can slow, stop, or prevent the condition.[1]
• An autologous approach: a US company, Regeneration Biomedical (RBI), is developing a therapy that uses a patient’s own stem cells, taken from their body fat, prepared in the laboratory and delivered directly into the brain.[2][3]
• Fast Track granted: the US Food and Drug Administration (FDA) has given the therapy Fast Track designation, a status intended to support the development of treatments for serious conditions with an unmet medical need. [2][4]
• Still early-stage: a small first-in-human Phase 1 study has been completed and a larger, placebo-controlled Phase 2 trial is now under way; the early results are encouraging, although they remain preliminary and are being further evaluated in an ongoing randomised trial.[5][6]

What is Alzheimer’s disease, and what treatments are available?

Alzheimer’s disease is the most common cause of dementia. It is a progressive condition that gradually affects memory, thinking, and the ability to carry out everyday tasks. In the UK, around 1 million people are estimated to be living with dementia, a figure projected to rise to 1.4 million by 2040.[1]

Treatment options remain limited. According to the Alzheimer’s Society, there are currently no treatments available on the NHS that can slow the progression of Alzheimer’s disease. Two newer treatments for early Alzheimer’s, lecanemab and donanemab, were licensed in the UK but were not approved for NHS use. [1] Most existing options manage symptoms rather than repairing the damage caused by dementia, which is why researchers continue to investigate approaches that could help address underlying disease processes.

What is the new therapy, and how does it work?

The therapy, known as RB-ADSC, is autologous, meaning it uses the patient’s own cells rather than a donor’s. A small amount of fat (50cc of lipoaspirate) is collected by liposuction, and the stem cells within it are purified, grown in the laboratory, and selected for a particular signalling property (Wnt expression) thought to be linked to repair, before being prepared for treatment. [5][7][8] These adipose-derived stem cells are a type of mesenchymal stem cell (MSC), a versatile family of cells studied across regenerative medicine for their ability to modulate inflammation and support tissue repair. [9][10]

To reach the brain, surgeons place a small device called an Ommaya reservoir under the scalp, with a fine catheter leading into one of the brain’s fluid-filled spaces (a ventricle). The prepared cells are delivered directly through this device. [5] The aim is to deliver the cells directly to the brain or bloodstream where they may support existing brain cells and the brain’s own repair processes. [11]

What did the first trial find?

The first-in-human Phase 1 study enrolled nine people with mild-to-moderate Alzheimer’s, with detailed results reported for the first seven and presented at the Clinical Trials on Alzheimer’s Disease (CTAD) conference in late 2025. The cells were given at three increasing doses. [5][7]

The main purpose at this stage was to test safety, and the approach was generally well tolerated. The only notable side effects were minor discomfort from the liposuction and the small operation to place the reservoir; no adverse events were reported from the injection of cells into the brain itself, even up to 25 weeks afterwards. [5] The researchers also reported early signals of possible benefit, including reductions in a spinal-fluid marker of Alzheimer’s and improvements in a memory and thinking test in most of the treated participants. [5]

These signals are encouraging, but they should be interpreted with caution: they come from a very small study with no placebo (comparison) group, so they cannot yet show whether the therapy truly works. That is the question the next, larger trial is designed to answer. [6]

What does FDA Fast Track mean?

Fast Track is a status the FDA grants to therapies for serious conditions that aim to fill an unmet medical need. It is designed to support a treatment’s development and allow more frequent contact with the regulator, so promising therapies can be assessed more quickly. [4] Importantly, Fast Track is not the same as approval: it does not mean a therapy has been shown to be safe and effective, only that it addresses a serious condition where better options are needed. [4] In June 2026, RBI announced that its Alzheimer’s programme had received FDA Fast Track designation. [2]

What happens next?

The programme has moved into a larger Phase 2 trial, which plans to enrol around 115 adults with mild-to-moderate Alzheimer’s. [6] Unlike the first study, this trial is randomised and placebo-controlled, comparing two different doses against a dummy treatment, with the cells given repeatedly (up to six times) through the implanted reservoir over about ten months. [6] The study looks at memory, thinking, and everyday function, alongside brain scans and spinal-fluid markers. [6] The company has said it hopes to explore the same approach in other conditions of the brain and nervous system, such as chronic traumatic encephalopathy (CTE) and Multiple Sclerosis, over time. [2][12]

What this means for families considering stem cell storage

Research like this is part of a wider move towards regenerative medicine that uses a person’s own (autologous) stem cells. [11]

The cells used in this trial are mesenchymal stem cells, a versatile family of cells found in several tissues in the body. [9][10] Umbilical cord tissue is a particularly rich and well-studied source of mesenchymal stem cells, and, unlike fat or bone marrow, it can only be collected at birth, where it can be collected for processing and storage. [13][14][15]That is one of the reasons some families choose to store their baby’s umbilical cord blood and cord tissue, preserving a sample of their child’s own stem cells.

Storing your child’s stem cells is often described as a form of biological insurance: something you hope never to use, but may be valuable if the need ever arises. It preserves a matched sample that remains available should it ever be clinically appropriate. As research into stem cell therapies continues to grow, it is an option increasingly being considered by parents as part of long-term family planning, reflecting a growing focus on preserving potential access to future developments in regenerative medicine.

To learn more about umbilical cord blood and cord tissue stem cells and how you can preserve them, fill in the form below to request your free Cells4Life Welcome Pack.

References

[1] Alzheimer’s Society (2025). Facts for the media about dementia. alzheimers.org.uk

[2] FirstWord Pharma (2026). RBI Granted ‘Fast Track’ Designation by FDA, Validating Its Direct-to-Brain Stem Cell Therapy. firstwordpharma.com/story/7593961

[3] ClinicalTrials.gov. Autologous Activated Adipose-Derived Stem Cells (RB-ADSC) Injected Directly Into the Brain for Mild to Moderate Alzheimer’s Disease, NCT05667649. clinicaltrials.gov/study/NCT05667649

[4] U.S. Food and Drug Administration. Fast Track. fda.gov

[5] Hoffman, M. (2025). Autologous Stem Cell Therapy for Alzheimer Disease Advances to Phase 2 After Positive Early Findings. CGTLive / NeurologyLive (reporting Phase 1 data from CTAD 2025). cgtlive.com

[6] ClinicalTrials.gov. Phase 2a Study of Intracerebroventricular Injections of Autologous Adipose-Derived Stem Cells (RB-ADSCs) in Participants With Mild-Moderate Alzheimer’s Disease, NCT07205601. clinicaltrials.gov/study/NCT07205601

[7] NeurologyLive. Patient Dosing Commenced in Phase 1 Study of Regeneration’s Stem Cell Therapy for Alzheimer Disease. neurologylive.com

[8] Whyte, J. L., Smith, A. A., & Helms, J. A. (2012). Wnt signaling and injury repair. Cold Spring Harbor perspectives in biology, 4(8), a008078. https://doi.org/10.1101/cshperspect.a008078

[9] Duma, C., et al. (2025). Early results of a “First-in-Human” Phase 1 clinical study of intracerebroventricular injections of ex vivo expanded, autologous, Wnt-activated, adipose-derived stem cells in participants with mild to moderate Alzheimer’s Disease. PubMed Central, PMC12725320. ncbi.nlm.nih.gov/pmc/articles/PMC12725320

[10] Si, Z., Wang, X., Sun, C., Kang, Y., Xu, J., Wang, X., & Hui, Y. (2019). Adipose-derived stem cells: Sources, potency, and implications for regenerative therapies. Biomedicine & Pharmacotherapy, 114, 108765. https://doi.org/10.1016/j.biopha.2019.108765

[11] Alzforum. Autologous Adipose-Derived Stem Cells (therapeutics entry). alzforum.org

[12] Regeneration Biomedical (2025). RBI, Inc. to Present their Phase 1 First-in-Human, Direct-to-Brain Autologous Stem Cell Therapy Results at CTAD 2025 (news release). einpresswire.com

[13] Zeddou, M., Briquet, A., Relic, B., Josse, C., Malaise, M.G., Gothot, A., Lechanteur, C. and Beguin, Y. (2010), The umbilical cord matrix is a better source of mesenchymal stem cells (MSC) than the umbilical cord blood. Cell Biology International, 34: 693-701. https://doi.org/10.1042/CBI20090414

[14] Vanessa N. Raileanu, Jennifer Whiteley, Theresa Chow, Alexandra Kollara, Aisha Mohamed, Armand Keating, Ian M. Rogers, Banking Mesenchymal Stromal Cells from Umbilical Cord Tissue: Large Sample Size Analysis Reveals Consistency Between Donors, Stem Cells Translational Medicine, Volume 8, Issue 10, October 2019, Pages 1041–1054, https://doi.org/10.1002/sctm.19-0022

[15] Velarde, F., Castañeda, V., Morales, E., Ortega, M., Ocaña, E., Álvarez-Barreto, J., Grunauer, M., Eguiguren, L., & Caicedo, A. (2020). Use of Human Umbilical Cord and Its Byproducts in Tissue Regeneration. Frontiers in bioengineering and biotechnology, 8, 117. https://doi.org/10.3389/fbioe.2020.00117