Stem Cell Research: Alzheimer's

Stem cells have a lot of potential, and scientists are starting to explore whether they could help people with Alzheimer’s. Some early results look promising, but it’s still early days. The research is far from clear-cut! Here’s a look at what we know so far from the studies that have been done in humans.

If you want to know more about the treatment process for Alzheimer’s, our article here goes into more depth. Or set up a conversation with our team here. They can guide you in deciding if it’s the right fit, help you choose the best clinic & region, and even assist you in securing some great discounts!

Current Alzheimer’s Trials in 2025

HUC-MSC-sEV-001: Nasal Drops for Alzheimer’s

(Full study is here)

What’s Being Tested?
A nasal drop treatment made from human umbilical cord-derived mesenchymal stem cell small extracellular vesicles (hUC-MSC-sEV-001). It’s being tested as a drug intervention.

Purpose:
To evaluate the safety and early signs of effectiveness of this stem cell-derived nasal therapy for neurodegenerative diseases, including:
– Alzheimer’s Disease
-Parkinson’s Disease
– Lewy Body Dementia

Location
Xuanwu Hospital, Capital Medical University, Beijing, China

Timeline:
Start Date: November 2024
Primary Completion: August 2027
Study Completion: August 2028
Status: Not yet recruiting (as of latest update on Sept 25, 2024)

Outcomes Being Measured:

Primary Outcome:
Change in Alzheimer’s Disease Composite Score (ADCOMS)

Secondary Outcomes:
Barthel Index (daily living)
MoCA (cognitive ability)
GDS (depression)
Sleep quality
Olfactory function
Brain MRI changes

Design Overview:
Single group
No control group (NA allocation)
Interventional
No results posted yet

Miami University Trial

(Clinical Trial ID: NCT06781333, here’s the full article)

Who’s Running It:
Dr. Bernard Baumel at the University of Miami is leading this study.

Participants:
8 people with moderate to severe Alzheimer’s who are experiencing behavioral symptoms like agitation, aggression, or anxiety.

What They’re Testing:
The trial is exploring whether a single IV infusion of 25 million mesenchymal stem cells (hMSCs) can help reduce these challenging symptoms. The stem cells are not taken from the patient, but come from a healthy donor. Basically they’re using allogeneic stem cells.

Why It Matters:
Behavioral symptoms like agitation are among the most difficult aspects of Alzheimer’s, both for patients and caregivers. This study is testing whether donor-derived stem cells can safely improve these symptoms without relying solely on medications.

What They’re Measuring:

Changes in behavior and mood, using a tool called the Neuropsychiatric Inventory
Whether patients need less antipsychotic medication
Side effects or safety issues from the treatment

Timeline:

The study is expected to begin in May 2025 and finish by June 2026
It is not yet recruiting participants

Texas University Trial

(Clinical Trial ID: NCT06775964. Full Article here.)

Who’s Running It:
The trial is being conducted at The University of Texas Health Science Center at Houston.

Participants:
12 adults between 60 and 80 years old who are in the early stages of Alzheimer’s or at high risk of developing it.

What They’re Testing

This study is testing whether stem cells taken from a person’s own fat tissue (autologous adipose-derived mesenchymal stem cells, or adMSCs) can help reduce brain inflammation and slow down memory loss.

Participants will undergo a fat biopsy to collect stem cells.
They’ll receive 4 intravenous (IV) infusions of their own stem cells over a 13-week period.

Each infusion contains about 200 million cells in a saline solution.

What They’re Measuring

Primary Outcomes:

Changes in brain inflammation, tracked using PET scans and biomarkers like TSPO (a signal of activated brain immune cells).

Inflammatory markers in spinal fluid (CSF).

Secondary Outcomes:

Safety: number of side effects
Changes in memory and thinking using tests like MMSE and RBANS
Changes in Alzheimer’s-related proteins in spinal fluid (like Tau, Amyloid-β, and Nf-L)
Brain metabolism changes via FDG-PET scans
Daily functioning, measured using the Lawton IADL scale
Immune markers in blood and spinal fluid

Timeline:

Start Date: February 2025
Primary Completion: December 2026
Full Study Completion: January 2027

Recruitment Status: Not yet recruiting

Why This Matters

This is one of the first human trials using a person’s own fat-derived stem cells (autologous adMSCs) to specifically target neuroinflammation in the early stages of Alzheimer’s. Most previous studies have used donor-derived cells or focused on later-stage disease.

Targeting inflammation in the brain is a promising direction because it’s increasingly seen as a major driver of Alzheimer’s progression, not just a side effect.

Regeneration BioMedical Trial

(Clinical Trial ID: NCT05667649, the full study is here)

Who’s Running It:
This study is sponsored by Regeneration Biomedical, Inc., a clinical-stage biotech company developing autologous stem cell therapies for neurodegenerative diseases.

Participants:
The trial is enrolling 9 adults with mild to moderate Alzheimer’s disease whose symptoms are not being adequately addressed by existing treatments.

What They’re Testing
This study is testing a new stem cell treatment called RB-ADSC, using stem cells taken from a person’s own fat.

Here’s how it works:

Doctors take a small sample of fat from the patient.
In the lab, they grow and select special stem cells that may help reduce brain inflammation and support healing in the brain.
These cells are then injected directly into the brain through a small, soft device that’s placed just under the scalp. This device gives doctors a safe way to deliver the treatment straight into the brain, skipping the usual barriers that protect it.

Because this is an early-phase trial, they’re also testing different doses to find out what’s safe and effective for future studies.

Doses Tested:

Low Dose: 2 million cells
Medium Dose: 5 million cells
High Dose: 10 million cells

What They’re looking at

Main Goal:
To make sure the treatment is safe, by checking for side effects, running lab tests, and doing physical checkups for about 6–7 months.

Other Things They’re Measuring:

Memory and thinking abilities, using common tests like the MMSE and ADAS-Cog
Brain size and structure, using MRI scans
Alzheimer’s-related proteins in spinal fluid (like tau and amyloid-beta)

Brain activity, using special PET scans that show changes before and after treatment

What They’ve seen So Far (as of May 2025)

80% of patients had lower levels of p-Tau, a protein linked to Alzheimer’s damage
60% improved on their memory test (MMSE)
80% improved on their thinking skills test (ADAS-Cog)

And importantly, no serious side effects were reported in the first 23 weeks after treatment

Longeveron’s Laromestrocel Trial

(Clinical Trial ID: NCT05233774. Results were posted here)

Who’s Running It:
This study was led by Longeveron, a biotech company focused on regenerative cell therapies. It was conducted across 10 sites in the U.S., including centers in Florida, Texas, and California.

Participants:
The trial included 50 adults between 60 and 85 years old who had been diagnosed with mild Alzheimer’s disease.

What they Tested

The treatment is called Lomecel-B (laromestrocel). A type of stem cell therapy made from donor bone marrow (these are not the patient’s own cells). These special stem cells are known for their ability to reduce inflammation and support blood vessel and brain health.

Participants were randomly placed into four groups:

Group 1: Placebo (no stem cells), given 4 times
Group 2: One infusion of 25 million stem cells, then 3 placebos
Group 3: Four infusions of 25 million cells
Group 4: Four infusions of 100 million cells

All treatments were given through a standard IV drip, once a month for four months.

What They Measured

Main Goal:
Make sure the treatment was safe, looking for side effects or problems on brain scans.

Other Goals:

Track changes in memory and thinking, using tools like MMSE and ADAS-Cog
Measure brain shrinkage (especially in the hippocampus) using MRI
Look at inflammation in the brain through advanced imaging

See if cognitive improvements matched physical brain changes

What They Found

Safe: There were no serious side effects, and no brain issues like swelling or microbleeds on MRI (a common problem in other Alzheimer’s drugs)
Cognitive improvement:
- 60–80% of patients improved on memory and thinking tests
Slowed brain shrinkage:
- Whole brain shrank 48% less compared to placebo
- Hippocampus (a key memory area) shrank 62% less
Less brain inflammation was seen on imaging
Cognitive improvements matched physical improvements in the brain

Why It Matters

This is one of the most advanced stem cell trials for Alzheimer’s to date. It shows that laromestrocel (Lomecel-B) is safe and may help slow brain degeneration and improve memory without the side effects seen in other treatments like antibody drugs. A larger Phase 3 trial may be next to confirm these results.

While the treatment showed promising results overall, the study did not report whether higher doses led to better outcomes, so it’s still unclear if more cells provide more benefit.

Previous Reviews, Trials & Studies (Post 2020)

Feizi et al’ Review on Cell Therapies 2023

Study Title:
A systematic review of clinical efficacy and safety of cell-based therapies in Alzheimer’s disease
Published: June 2023
Authors: Feizi et al.
PMID: 39258164
Article Link: Here

What They Looked At:
Researchers analyzed all published human clinical studies (up to June 2023) that tested cell-based therapies like stem cells as a treatment for Alzheimer’s disease.

Out of 1,014 studies, only 5 clinical trials met the criteria for inclusion.

These studies involved 70 total participants (59 treated with stem cells and 11 given a placebo)

What Types of Stem Cells were used?

Across the 5 studies, treatments included:

Adipose-derived stem cells (from fat) injected into the brain’s ventricles
Umbilical cord–derived MSCs, delivered either by IV or directly into the hippocampus and precuneus

Lomecel-B (bone marrow–derived MSCs), delivered by IV infusion

Key Findings

Safety:
All treatments were considered safe and well tolerated. No major side effects or safety concerns were reported, regardless of the type of stem cell or delivery method.

Effectiveness (Early Signs):
Some studies reported improvements in cognitive test scores and brain biomarkers, suggesting possible benefits.

Limitations

Small sample sizes (only 70 people total)
Three studies had no control group (which limits how reliable the results are)
Different methods, doses, and cell types were used, making it hard to compare outcomes
Some concerns about bias in how results were measured

Review Conclusion

Stem cell therapy for Alzheimer’s appears safe and shows early signs of benefit, but there’s not enough strong evidence yet.

Larger, better-designed trials are needed before doctors can make clear recommendations.

Nasal Spray Study Overview: 2022

Who’s Running It
This trial was led by Dr. Jianqing Xie and a neurology team at Tianjin Huanhu Hospital and the Tianjin Neurological Institute in China. (Full article is here.)

Methodology
This was a Phase I/II open-label clinical trial testing a new therapy for Alzheimer’s disease using exosomes derived from allogeneic human adipose-derived mesenchymal stromal cells (ahaMSCs-Exos).

Exosomes are small vesicles secreted by cells that carry proteins, RNA, and other molecules. They can travel through bodily fluids and potentially cross into the brain to support cell repair and reduce inflammation.

Key Points

Safety

No adverse events or serious adverse events were reported.
Participants tolerated the intranasal administration well, even those with elevated IgE levels.

Vital signs and lab tests (liver, kidney, etc.) remained stable and normal throughout the trial.

Participants

9 patients completed treatment: 5 with mild AD, 4 with moderate AD.
Participants were divided into 3 dosage groups:
- Low: 2×10⁸ particles
- Medium: 4×10⁸ particles
- High: 8×10⁸ particles
Treatment: 2 nasal sprays per week for 12 weeks, followed by 9 months of observation.

Efficacy

Medium-dose group showed the most promising results:
Low and high doses were less consistent or showed no significant cognitive improvement.
Amyloid and tau buildup didn’t change much overall, though one high-dose patient showed a notable reduction in amyloid levels.

Mechanism

Exosomes carried over 1,400 proteins and 277 microRNAs, including:

– Neurotrophic and anti-inflammatory factors
– Molecules linked to neurogenesis, synaptic plasticity & cell protection
These molecules are believed to support brain repair and cognitive function.

Limitations & Next Steps

Small sample size and no control group
Participants varied in disease severity and age, which may have affected results
Future trials should:
- Use a randomized, placebo-controlled design
- Focus on mild AD or early intervention
Explore dose optimization and mechanisms of action

Why This Matters

This is the one of the first published human clinical trial testing MSC-derived exosomes for Alzheimer’s. Results suggest that medium-dose intranasal exosomes could help slow cognitive decline and maintain daily function with a strong safety profile.

It also provides early clinical evidence that this stem cell-based approach, especially without using whole cells could be a HUGE for treating neurodegenerative diseases like Alzheimer’s.

MediPost’s NeuroStem Trials: South Korea

Study Title: Clinical safety and efficacy of allogenic human adipose mesenchymal stromal cells-derived exosomes in patients with mild to moderate Alzheimer’s disease
Location: Samsung Medical Center, Seoul, South Korea
Sponsor: MEDIPOST
Published: 2021 (Here is the link)

Who Ran the Study?
This research was carried out by doctors and scientists at a top hospital in South Korea to test a new Alzheimer’s treatment using umbilical cord stem cells. These stem cells came from healthy donors and were turned into a treatment called NEUROSTEM®.

Who Took Part

9 people with mild to moderate Alzheimer’s disease
All were between 50 and 85 years old
Everyone had Alzheimer’s confirmed by memory tests and brain scans

What Was the Treatment?

The treatment involved injecting stem cells directly into the brain using a small device placed under the scalp (called an Ommaya reservoir).

Each person got:

3 injections total
One every 4 weeks

Two dosage levels were tested:

Low dose: 10 million cells per injection

High dose: 30 million cells per injection

Was it Safe?

Mostly, yes. But there were some short-term side effects:

Everyone got a fever after the injection
Some people felt sick, had headaches, chills, or muscle aches
These symptoms lasted less than 2 days
No one had long-term problems, and brain scans showed no serious issues

Bottom line: The treatment was generally safe, even though it caused brief fevers and discomfort.

Did it Help?

This trial wasn’t designed to prove whether the treatment works. Tt was focused on safety. But the doctors still looked for early signs of improvement:

Some Alzheimer’s-related proteins (like tau and amyloid) went down right after the injection, but only for a short time.
Memory test scores didn’t really improve.
Brain scans looked mostly the same after a year.

So while the stem cells may have had some short-term effects, they didn’t lead to big improvements in memory or thinking, at least not in this small group.

Why This Matters

This was one of the first studies to test whether stem cells from umbilical cords can be safely injected into the brain of people with Alzheimer’s.

Here’s why it’s important:

The approach was safe enough to keep testing
It showed that repeated brain injections are possible
It raised new questions, like whether higher or more frequent doses are needed to see real benefits

So, What Happened to NEUROSTEM®?

After the initial trial showed the treatment was safe, researchers followed patients for another 3 years to track long-term effects. But here’s the thing, no results have ever been published, and there’s been no press release or update since that follow-up study ended.

Even more telling? The treatment is no longer listed on MEDIPOST’s website.

That usually means one of two things:

The results weren’t exciting enough to move forward, or
The company quietly shifted focus to other therapies that showed more promise.

It doesn’t necessarily mean NEUROSTEM® failed or caused harm, just that it likely didn’t work well enough to justify a larger, expensive trial. Instead of announcing that publicly, the company seems to have just let it fade away.

So for now, NEUROSTEM® looks like a promising idea that didn’t quite pan out. At least not yet.

Conclusion

Stem cell research for Alzheimer’s is still in the early stages, but it’s happening mainly in South East Asia & parts of the US. The treatments use different types of stem cells, including ones from umbilical cords, bone marrow, and a person’s own fat.
Some are injected into the bloodstream, some directly into the brain, and others even delivered as nasal sprays. Doses vary a lot, from 10 million to 200 million cells.

So far, the big takeaway is that these therapies appear safe, with only mild, short-term side effects. Some patients have shown improvements in memory, brain scans, or Alzheimer’s-related proteins. But results vary, and the studies are small.

We don’t yet know which method works best, how often treatment is needed, or who it helps most.

If we look at just the research, more large, controlled trials are needed. But, there have been some amazing stories from people who have had treatment in clinical settings too.