New approaches to treat fibrous dysplasia
Novel Strategies for Understanding and Treating Fibrous Dysplasia
Developing drugs and lab-based methods to stop abnormal bone growth in people with fibrous dysplasia, including those with McCune‑Albright Syndrome.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11323947 on NIH RePORTER |
What this research studies
This project uses patient-derived samples and lab-grown human stem cells together with genetically engineered mouse models to study why fibrous dysplasia causes abnormal bone. Researchers apply advanced genetic tools and an artificial-intelligence screening method to find compounds that block the overactive Gsα/cAMP and Wnt signals driving the disease. Promising candidates will be tested in cells and mice to see if bone lesions can be reduced or reversed. The long-term goal is to move the best approaches toward tests in people and new treatments for FD.
Who could benefit from this research
Good fit: People with fibrous dysplasia or McCune‑Albright Syndrome who are willing to donate tissue or may participate in future clinical testing would be the primary candidates.
Not a fit: People without fibrous dysplasia or MAS, or those needing immediate clinical care today, are unlikely to gain direct benefit from this primarily preclinical work.
Why it matters
Potential benefit: Could produce targeted medicines that shrink or repair FD bone lesions and improve pain, function, and appearance.
How similar studies have performed: Prior animal work showed that stopping excess Gs signaling can dramatically reverse FD-like lesions, but turning that finding into safe, effective human drugs remains unproven.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Hsiao, Edward C — University of California, San Francisco
- Study coordinator: Hsiao, Edward C
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.