Investigating biomarkers related to prion disease and its treatment
Official title: Biomarkers and Mechanisms of PrP Misfolding, Mutation, and Deficiency
Funded at Broad Institute, INC. · NIH-11057683 · Data updated 2026-11-12
This study is looking for signs in blood and spinal fluid that can help us understand how prion disease progresses and responds to treatments, so we can find better ways to detect and treat this serious condition in people at risk.
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 start | April 1, 2024 |
| Project end | March 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $585,539 |
| NIH project number | 5R01NS132022-02 |
| Project ID | NIH-11057683 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals with a genetic predisposition to prion disease and those in the early stages of the disease.
✕Less likely to help
Patients with advanced prion disease or those without any genetic risk factors for the disease may not benefit from this research.
What this research studies
This research focuses on understanding prion disease, a fatal neurodegenerative condition, by identifying molecular biomarkers that indicate the disease's progression and response to treatment. The study will analyze cerebrospinal fluid and plasma samples from healthy individuals at risk and from infected animal models to track changes in prion protein (PrP) misfolding. By discovering these biomarkers, the research aims to improve early detection and treatment strategies for prion disease, potentially leading to better patient outcomes. The findings could help determine the safety and effectiveness of new therapies as they advance toward clinical use.
Why it matters
Potential benefit: If successful, this research could lead to earlier diagnosis and more effective treatments for prion disease, improving survival and quality of life for affected patients.
How similar studies have gone: Previous research has shown promise in using biomarkers for other neurodegenerative diseases, suggesting that this approach could be effective for prion disease as well.
Where this research is happening
-
Broad Institute, INC.
Cambridge, United States Active
Researchers
- Principal investigator: Minikel, Eric Vallabh — Broad Institute, INC.
- Study coordinator: Minikel, Eric Vallabh
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.