For families affected by Alzheimer’s disease, the search for effective treatments can feel like a race against time.
While some drugs aim to manage symptoms, two groundbreaking clinical trials have revealed that stem cell therapies may address the underlying causes of the disease by reducing brain inflammation, clearing harmful proteins, and even improving cognitive function.
These new approaches offer a different path forward, focusing on healing and regeneration rather than just slowing the decline. The results from these recent studies provide a new sense of hope for the millions of people living with Alzheimer’s.
Two Promising Stem Cell Therapies Emerge
Recent findings from two major clinical trials presented at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference have generated significant excitement.
Both studies highlight the potential of stem cells to offer a multi-faceted approach to treatment, a departure from the single-target strategy of many existing drugs.
1. Direct-to-Brain Autologous Stem Cells (Regeneration Biomedical)
A first-in-human Phase 1 trial by Regeneration Biomedical tested a therapy where a patient’s own stem cells, enhanced to express the Wnt protein, were injected directly into the brain. This method aims to activate dormant stem cells already present in the brain to promote regeneration. [1]
The results were highly encouraging. After 12 weeks, 83% of participants showed improved cognitive scores on the ADAS-Cog assessment, a standard measure of cognitive function. The therapy also reduced levels of phosphorylated tau, a protein that forms tangles in the brain, and improved amyloid PET scan scores in 66% of patients. [1]
Most importantly, the treatment was found to be safe and well-tolerated. The direct-to-brain injection, administered without anesthetic, caused no adverse events, and other side effects were minor. The study is now advancing to a larger Phase 2 trial. [1]
2. Mesenchymal Stem Cells – Laromestrocel (Longeveron)
In a separate Phase 2a trial called CLEAR MIND, researchers investigated laromestrocel, a therapy using mesenchymal stem cells (MSCs) derived from bone marrow. This approach focuses on reducing neuroinflammation, a key factor in Alzheimer’s progression. [2]
MRI analysis revealed that laromestrocel significantly reduced brain inflammation, particularly in the hippocampus, the brain’s memory center.
The therapy also slowed the rate of brain volume loss by 49% and reduced left hippocampal volume loss by an impressive 84% in one treatment group. [2]
Patients treated with laromestrocel also showed statistically significant improvements in cognitive function, as measured by the Montreal Cognitive Assessment (MOCA) and the Mini-Mental State Exam (MMSE).
Like the Regeneration Biomedical trial, laromestrocel was found to be safe, with no cases of amyloid-related imaging abnormalities (ARIA), a side effect sometimes seen with amyloid-targeting drugs. [2]
How Do Stem Cells Work in Alzheimer’s Disease?
Stem cell therapies offer a fundamentally different approach to treating Alzheimer’s. Instead of targeting a single problem like amyloid plaques, they work through multiple mechanisms to create a healthier brain environment.
| Mechanism of Action | Description |
|---|---|
| Anti-Inflammatory Effects | Stem cells release anti-inflammatory molecules that calm the chronic inflammation in the brain, which is known to accelerate neuron damage. [3] |
| Trophic Support | They secrete growth factors that protect existing neurons from damage and support their survival and function. [4] |
| Cellular Regeneration | Some therapies, like the direct-to-brain approach, aim to stimulate the brain’s own dormant stem cells to create new, healthy neurons. [1] |
| Amyloid & Tau Reduction | Studies show that stem cells can help clear amyloid-beta plaques and reduce the hyperphosphorylation of tau proteins. [1] [5] |
| Improved Blood Flow | MSCs can promote the formation of new blood vessels, improving circulation and the delivery of oxygen and nutrients to the brain. [4] |
This multi-pronged approach may explain why these therapies are showing benefits in both biomarkers and cognitive function. As Joshua Hare, MD, of Longeveron noted, laromestrocel “offers the potential to address the underlying pathology of Alzheimer’s disease.” [2]
A Safer Alternative to Amyloid-Targeting Drugs?
One of the most significant advantages of the stem cell therapies studied is their safety profile. Recent amyloid-targeting drugs have been associated with side effects like amyloid-related imaging abnormalities (ARIA), which can involve brain swelling or bleeding. [2]
In both the Regeneration Biomedical and Longeveron trials, no cases of ARIA were reported. This suggests that stem cell therapies could offer a safer alternative for people with Alzheimer’s, particularly those at higher risk for such complications.
The Road Ahead
The promising results from these early- and mid-stage trials mark a major step forward, but more research is needed. Both therapies are now advancing to larger, more definitive Phase 2 and Phase 3 studies to confirm their safety and effectiveness in a broader population.
If these trials are successful, stem cell therapy could represent a paradigm shift in how we treat Alzheimer’s disease—moving from managing symptoms to actively healing the brain. For the millions of individuals and families affected by this condition, these developments offer a new and powerful reason for hope.
References
[1] NeurologyLive. (2025, December 4). Autologous Stem Cell Approach Shows Promising Early Phase 1 Data in Alzheimer Disease. https://www.neurologylive.com/view/autologous-stem-cell-approach-promising-early-phase-1-data-alzheimer
[2] NeurologyLive. (2025, December 2). MRI Analysis Shows Stem Cell Therapy Laromestrocel Reduced Neuroinflammation in Mild Alzheimer Disease. https://www.neurologylive.com/view/mri-analysis-shows-stem-cell-therapy-laromestrocel-reduced-neuroinflammation-mild-ad
[3] Michigan Medicine. (2024, April 25). Stem cells improve memory, reduce inflammation in Alzheimer’s mouse brains. https://www.michiganmedicine.org/health-lab/stem-cells-improve-memory-reduce-inflammation-alzheimers-mouse-brains
[4] DVC Stem. (n.d.). Stem Cell Therapy for Alzheimer’s: Findings and Treatment. https://www.dvcstem.com/post/stem-cell-therapy-for-alzheimers
[5] Liu, X. Y., et al. (2020). Stem cell therapy for Alzheimer’s disease. PMC. https://pmc.ncbi.nlm.nih.gov/articles/PMC7477654/
