Lay summary by Matthew Chakraverty, reviewed by Dr Dr Naomi Hartopp and a Dementia lay panel
Background
Alzheimer’s disease (AD) is a widespread type of dementia caused by damage to brain cells. Current medicines can reduce symptoms of AD but do not prevent the disease from getting worse over time. As such much research is focused on new possible treatments that work differently.
Stem cells are cells that can become different cell types depending on the part of the body they are in. Previous research suggests stem cells taken from the blood and given back to patients can help protect the brain, allow it to heal and limit new damage. Additionally, naturally occurring chemicals in the body called growth factors can aid in healing the brain in AD. Therefore, a treatment which combines stem cells and certain growth factors could be a new dementia treatment.
Why is the study important?
Current research on the use of stem cells to treat dementia has been conducted on animals. For a treatment to be prescribed to patients it must first be tested in humans to confirm it works and is safe to use. This study therefore tested stem cell therapy in a small group of volunteers before it sees widespread use.
What did the authors do and how did they do it?
38 volunteers diagnosed with Alzheimer’s, who had no other major health issues and enough capacity to provide informed consent for the study were split into two groups. The first group treated with stem cells and growth factors. The second group was given existing medications for Alzheimer’s. Groups were assigned randomly, with patients knowing which group they were in but researchers analysing the results of the study not knowing. This is called ‘blinding’ and is done to prevent researchers from subconsciously reporting what they expect to see.
Before any treatment began all participants were given a score on how bad their dementia was based on interviews and questionnaires. Brain scans were used to detect the level of Amyloid Beta (a protein involved in causing Alzheimer’s) and damage to the brain. Levels of proteins in the blood which indicate damage to the brain were recorded.
Blood samples were taken from each volunteer in the first group. Stem cells were separated from the blood. These cells are then processed and mixed with the growth factors before being given back to the patient through injection. After a few months they were asked to repeat the initial testing and scans they went through before treatment to see if there were any changes. The second group went through the same process but did not receive the new treatment so their results could be used as a point of comparison.
What are the results?
All patients completed the treatment with no major side effects, only some noting tiredness and a temperature.
The treatment groups’ dementia score changed from 18.2 to 22.3 over 6 months. A low score represents worse symptoms, so this was an improvement. The other group saw no improvement on their initial score of 17.8. Other scoring systems used showed similar results.
Scans showed less amyloid and increased brain activity in key areas linked to memory after treatment. There was a reduction in size in the area involved in memories, but this reduction was smaller in the treatment group. However, the authors to warn that the results of the scans could be unreliable.
Blood levels for proteins involved in healing the brain were higher, whilst those associated with harm were lower in those taking the new medication.
What do the findings mean going forward for people with the disease?
The study shows that combined stem cell and growth factor therapy seems to work better than current medicines at reducing the symptoms of Alzheimer’s and is likely to be safe. However, the study is small scale and for it to be approved it will need multiple trials and long term follow up of patients. Ultimately the study describes a potential new treatment for Alzheimer’s which is likely to progress to larger clinical trials.
This study can be found at
https://doi.org/10.1186/s12974-025-03545-w
Paper title
Therapeutic Efficacy of Autologous Blood-Derived Stem Cells with Growth Factors in Moderate to Severe Alzheimer’s Disease: A Clinical Trial.
Lead author
Woong Jin Lee, Gyung Whan Kim
Publication details including date of publication
Lee WJ, Cho K, Lee D, Lee S, Kim GW. Therapeutic Efficacy of Autologous Blood-Derived Stem Cells with Growth Factors in Moderate to Severe Alzheimer’s Disease: A Clinical Trial. Mol Neurobiol. 2025 Dec 10;63(1):277. doi: 10.1007/s12035-025-05583-0. PMID: 41366593; PMCID: PMC12689718. Date of publication : 10th December 2025