How healthy blood stem cells might help in Alzheimer's
Understanding the mechanism of rescue of Alzheimers disease by hematopoietic stem cell transplantation
['FUNDING_R01'] · UNIVERSITY OF CALIFORNIA, SAN DIEGO · NIH-11295487
Researchers are using a mouse model of Alzheimer's to test whether transplanting healthy blood-forming stem cells can prevent memory loss and brain damage.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA, SAN DIEGO (nih funded) |
| Locations | 1 site (LA JOLLA, UNITED STATES) |
| Trial ID | NIH-11295487 on ClinicalTrials.gov |
What this research studies
Scientists work with a well-established mouse model that carries human Alzheimer-related genes and give the animals healthy hematopoietic (blood-forming) stem cells. They track memory and behavior, measure amyloid plaque levels, monitor inflammation and blood–brain barrier integrity, and follow how transplanted cells take on microglia-like roles in the brain. The team uses genomic tools such as ATAC-seq and cellular tracing to understand which cell types and gene programs are responsible for the strong benefit seen in earlier experiments. Their goal is to pin down the mechanism so the approach can be translated toward human therapies in the future.
Who could benefit from this research
Good fit: People with early-stage Alzheimer's disease or those at high genetic risk (for example with APP/PSEN1 mutations) would be the most likely future candidates for related clinical trials.
Not a fit: Patients with very advanced Alzheimer's, other types of dementia, or those medically ineligible for stem cell transplantation are unlikely to benefit directly from this line of research in the short term.
Why it matters
Potential benefit: If the mechanism is confirmed, this work could point to new stem-cell–based or microglia-targeting therapies that slow or prevent Alzheimer's progression.
How similar studies have performed: Previous work in the same mouse model showed dramatic rescue of memory and pathology after transplanting healthy HSPCs, but this approach has not yet been proven safe or effective in people.
Where this research is happening
LA JOLLA, UNITED STATES
- UNIVERSITY OF CALIFORNIA, SAN DIEGO — LA JOLLA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: CHERQUI, STEPHANIE — UNIVERSITY OF CALIFORNIA, SAN DIEGO
- Study coordinator: CHERQUI, STEPHANIE
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.