Medicines to correct the ApoE4 protein linked to Alzheimer's
Discovery and optimization of ApoE4 correctors for the treatment of Alzheimer's Disease
['FUNDING_R01'] · SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE · NIH-11136909
Testing small molecules that aim to make the high-risk ApoE4 protein act more like the safer ApoE3 form to help people with or at risk for Alzheimer's.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE (nih funded) |
| Locations | 1 site (LA JOLLA, UNITED STATES) |
| Trial ID | NIH-11136909 on ClinicalTrials.gov |
What this research studies
Researchers are searching for small drug-like compounds that bind the ApoE4 protein and change its shape so it behaves more like the lower-risk ApoE3 form. They will produce and purify human ApoE proteins and use laboratory tests such as protein thermal shift assays and analyses of human brain tissue lysates to find compounds that stabilize ApoE4. Promising molecules will be optimized and tested further in humanized animal models and additional preclinical experiments to improve stability and function.
Who could benefit from this research
Good fit: Ideal candidates are people who carry one or two copies of the APOE ε4 allele or individuals with early Alzheimer's linked to APOE4.
Not a fit: People who do not carry the APOE4 allele or whose dementia is driven by other causes are less likely to benefit from ApoE4-specific correctors.
Why it matters
Potential benefit: If successful, this approach could reduce Alzheimer's risk or slow disease progression for people who carry the APOE4 gene.
How similar studies have performed: Previous laboratory and preclinical efforts have shown some promising signs that ApoE4 can be stabilized, but no ApoE4-correcting drug has been proven effective in clinical use.
Where this research is happening
LA JOLLA, UNITED STATES
- SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE — LA JOLLA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: JACKSON, MICHAEL — SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE
- Study coordinator: JACKSON, MICHAEL
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome