Boosting nerve-cell cleanup to fight prion and Alzheimer-related brain damage
Restoring neuronal degradative capacity as a therapeutic strategy to treat Prion Disease
This project boosts neurons' waste-disposal system to reduce toxic protein clumps in people with prion disease and related dementias.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scripps Research Institute, the NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11247516 on NIH RePORTER |
What this research studies
Researchers are studying how nerve cells in prion disease and some Alzheimer's-related dementias build up misfolded proteins inside enlarged, poorly functioning compartments. They plan to restore lysosomal and autophagy pathways that clear these protein aggregates, testing drug and molecular approaches in lab-grown neurons and animal models. The team will measure whether boosting degradative capacity reduces protein clumps and improves axonal transport and neuronal health in these models. Any human-sample or patient-related work would be coordinated through Scripps Research and would start with biomarkers or tissue studies rather than a direct treatment right away.
Who could benefit from this research
Good fit: People diagnosed with prion disease or Alzheimer-type dementias marked by protein aggregation would be the most relevant candidates for future therapies developed from this research.
Not a fit: Patients whose conditions are not driven by protein-aggregate pathology or those with very advanced, widespread brain damage may not receive direct benefit from this project in the near term.
Why it matters
Potential benefit: If successful, this work could lower toxic protein buildup and help prevent or slow nerve-cell damage in prion disease and possibly some forms of Alzheimer's dementia.
How similar studies have performed: Laboratory and animal studies have shown that activating autophagy can reduce protein aggregates and improve cell function, but this approach has not yet been proven as a therapy in humans.
Where this research is happening
La Jolla, United States
- Scripps Research Institute, the — La Jolla, United States (Active)
Researchers
- Principal investigator: Encalada, Sandra E — Scripps Research Institute, the
- Study coordinator: Encalada, Sandra E
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.