Small-molecule drugs that target synaptojanin 1 for Alzheimer's
Official title: Novel Disease-modifying Small Molecules for Treatment of Alzheimer's Disease”
Funded at Minneapolis VA Medical Center · NIH-11264765 · Data updated 2026-11-12
This work is trying new small-molecule drugs to slow Alzheimer's by lowering activity of a brain protein called synaptojanin 1, which is linked to memory loss and APOE ε4-related problems.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Minneapolis VA Medical Center NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | January 1, 2023 |
| Project end | December 31, 2026 |
| Funding year | 2026 |
| NIH project number | 5I01BX005934-04 |
| Project ID | NIH-11264765 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with early or mild Alzheimer’s disease, especially those who carry the APOE ε4 gene, would be the most likely candidates for future clinical trials.
✕Less likely to help
People with advanced, severe dementia or cognitive decline due to non‑Alzheimer causes are unlikely to benefit in the near term from this preclinical work.
What this research studies
Researchers at the Minneapolis VA are designing and testing small molecules that reduce synaptojanin 1 (synj1), a protein tied to synapse loss and Alzheimer pathology. The team uses laboratory models, biochemical assays, and analyses of human brain tissue to see if lowering synj1 protects synapses and helps clear amyloid beta. Promising compounds will be optimized for safety and brain delivery as steps toward an investigational new drug (IND) application. The project focuses on pathways relevant to APOE ε4-related dementia with the goal of moving candidates into future human trials.
Why it matters
Potential benefit: If successful, this work could lead to oral drugs that slow or stop Alzheimer’s-related brain damage and cognitive decline.
How similar studies have gone: Genetic reduction of synaptojanin 1 has improved cognition and reduced pathology in animal models, but small-molecule therapies targeting synj1 are new and unproven in humans.
Where this research is happening
-
Minneapolis VA Medical Center
Minneapolis, United States Active
Researchers
- Principal investigator: Cai, Dongming — Minneapolis VA Medical Center
- Study coordinator: Cai, Dongming
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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.