Investigational Stem Cell Therapy Aims to Stabilize Alzheimer’s Disease Progression
A University of Miami Miller School of Medicine clinical trial is testing whether a single infusion of mesenchymal stem cells, added to standard anti-amyloid therapy, can stabilize cognitive decline in patients with early Alzheimer’s disease.
A new investigator-initiated clinical trial at the University of Miami Miller School of Medicine is exploring whether a single infusion of mesenchymal stem cells can stabilize cognitive decline in patients with mild cognitive impairment or early Alzheimer’s disease.
The trial, led by Barry Baumel, M.D., an associate professor of neurology and director of memory disorders clinical trials at the University of Miami Miller School of Medicine, builds on both emerging standard-of-care therapies and earlier stem cell research in which Dr. Baumel participated, including a published phase I clinical trial investigating Lomecel‑B, a mesenchymal stem cell–based therapy.
“What I want to see, in this group that’s being treated to slow their disease, is if we can stabilize so it stops getting worse,” Dr. Baumel said.
Combining Stem Cell Therapy With Standard Alzheimer’s Treatments
The current study targets patients already receiving anti-amyloid monoclonal antibody therapies (Leqembi or Kisunla) that are widely used in early Alzheimer’s disease. These drugs remove amyloid plaque and slow disease progression but do not halt or reverse it.
“We have data that shows us how people do on these drugs,” Dr. Baumel said. “It’s like a ski slope. Over time, the slope changes. It’s still a ski slope, but it’s not as steep a ski slope, if you take the medications. You end up going down the hill slower.”
The trial adds a one-time intravenous infusion of human mesenchymal stem cells (hMSCs) to see if they can further improve outcomes. Participants will be monitored longitudinally following the stem cell infusion, with outcomes focused on whether cognitive decline stabilizes or improves.
The study intentionally delays the stem cell treatment until patients have already been on monoclonal antibody therapy for six months, minimizing confounding safety signals from overlapping treatments.
Who Qualifies for the Alzheimer’s Clinical Trial
The trial focuses on individuals in the early stages of Alzheimer’s disease.
Eligible participants must:
• Be between 55 and 90 years old
• Have a diagnosis of mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s disease
• Demonstrate a Mini-Mental State Examination (MMSE) score between 20 and 26
• Retain sufficient cognitive capacity to provide consent independently
• Have a designated study partner to help assess daily functioning
How Stem Cells May Reduce Brain Inflammation in Alzheimer’s Disease
The study is grounded in a growing understanding that Alzheimer’s disease involves more than amyloid accumulation alone. Neuroinflammation is increasingly recognized as a key driver of disease progression.
“There’s a lot of brain inflammation in people with Alzheimer’s disease,” Dr. Baumel said. “We know removing the amyloid reduces inflammation, and that may be why it slows the progression.”
Mesenchymal stem cells may offer a complementary approach because of their immunomodulatory and regenerative properties. They can migrate to sites of injury, release anti-inflammatory molecules and promote tissue repair.
“Stem cells are also very potent anti-inflammatories,” Dr. Baumel said. “So that may add to the anti-inflammatory effect of removing the amyloid. Our theory is that adding another anti-inflammatory medication, a potent one like stem cells, can really be helpful.”
This dual mechanism of reducing amyloid burden and modulating inflammation underpins the trial’s hypothesis.
Building on Early Clinical Evidence for Mesenchymal Stem Cells
Dr. Baumel’s current work is informed by a previously published phase I clinical trial, co-authored by him, that evalated Lomecel-B in patients with mild Alzheimer’s disease.
In that study, patients received a single, intravenous infusion of stem cells or placebo in a randomized, double-blind design. The trial’s endpoint was safety, and that was successfully met, with no treatment-related serious adverse events.
Exploratory findings suggested potential clinical benefit. Patients receiving lower-dose stem cell infusions showed improved or stabilized cognitive performance compared to placebo, along with favorable changes in biomarkers related to inflammation and vascular function. Fluid-based and imaging outcomes also indicated that stem cell therapy may influence multiple aspects of Alzheimer’s pathology, including neuroinflammation and neurovascular health.
The current trial expands those findings by combining stem cell therapy with standard anti-amyloid treatment.
The Role of Private Funding in Advancing Research
Dr. Baumel’s study is funded through philanthropic contributions, and he stressed how integral the generosity of organizations like the Ohrstrom Foundation and the William Gross Family Foundation, as well as several grateful patients, is to scientific discovery.
“These groups allow us to do our work, on behalf of the patients we’re trying to help,” Dr. Baumel said. “Without them this research might not happen.”
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Tags: Alzheimer's disease, brain health, clinical trials, cognitive decline, Department of Neurology, Dr. Barry Baumel, mesenchymal stem cells, neurology, stem cells