Medicines to lower the prion protein (PrP)
Mechanism of Action of Prion Protein-Lowering Small Molecules
This work looks at small drug-like compounds that reduce levels of the prion protein to help people at risk for or living with prion disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Broad Institute, INC. NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-11306652 on NIH RePORTER |
What this research studies
Researchers discovered two small molecules that substantially lower the prion protein (PrP) in cells and showed they are selective among many proteins. They will use cell biology and genetic tools to figure out exactly how these compounds reduce PrP and confirm which molecular targets are involved. The team will also test whether the molecules can lower PrP in living models and study their effects on protein regulation and safety. If these steps succeed, the findings could guide the development of pill-like treatments that reach the brain more easily than large biologics.
Who could benefit from this research
Good fit: People living with prion disease, individuals with known genetic risk variants for prion disease, or volunteers willing to provide biological samples for related research would be most relevant to this work.
Not a fit: People with unrelated neurological conditions or those not eligible or willing to provide samples or participate in future clinical steps are unlikely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this research could lead to drug-like treatments that lower brain PrP and potentially slow or prevent fatal prion disease.
How similar studies have performed: Lowering PrP with antisense oligonucleotides has provided proof-of-concept in models, but using small, drug-like molecules to do this is a newer and less-tested approach.
Where this research is happening
Cambridge, United States
- Broad Institute, INC. — Cambridge, United States (Active)
Researchers
- Principal investigator: Vallabh, Sonia Minikel — Broad Institute, INC.
- Study coordinator: Vallabh, Sonia Minikel
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.