Targeting harmful misfolded proteins in Alzheimer's and related dementias
Chemical Control of Misfolded Protein Fate
Researchers are developing chemical tools that seek out and remove toxic misfolded proteins to help people with Alzheimer's, Parkinson's, and related diseases.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11009690 on NIH RePORTER |
What this research studies
This project is creating small molecules that can selectively bind and eliminate misfolded, aggregated proteins that build up in brain and systemic diseases. The team is adapting targeted protein degradation methods from cancer research to work in cells and animal models of Alzheimer's, frontotemporal dementia, and other proteinopathies. They previously used this approach to study tau protein and will expand the methods to more disease proteins while improving speed and usability. The goal is to understand how aggregates harm cells and to lay groundwork for future patient-directed therapies.
Who could benefit from this research
Good fit: Adults diagnosed with Alzheimer's disease, frontotemporal dementia, Parkinson's disease, Huntington's disease, or other protein-aggregate disorders who are willing to donate samples or consider participation in future clinical studies would be the most relevant participants.
Not a fit: People without protein-aggregate disorders, those needing immediate clinical treatment, or those unable to travel to research centers are unlikely to receive direct benefit from this preclinical research.
Why it matters
Potential benefit: If successful, this work could reduce toxic protein aggregates and help slow or prevent progression of Alzheimer's and other protein-aggregation diseases.
How similar studies have performed: Targeted protein degradation has produced successful cancer therapies and early laboratory studies on tau show promise, but applying these chemical tools broadly to brain proteinopathies is largely novel.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Ferguson, Fleur Marcia — University of California, San Diego
- Study coordinator: Ferguson, Fleur Marcia
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.