New ways to understand and treat fibrous dysplasia (Albright syndrome)
Novel Strategies for Understanding and Treating Fibrous Dysplasia
Looking for medicines that reduce abnormal bone growth in people with fibrous dysplasia and 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-11528909 on NIH RePORTER |
What this research studies
This project focuses on fibrous dysplasia (FD), a condition that causes abnormal bone growth often seen in McCune‑Albright syndrome. Researchers are using patient-derived stem cells (iPSCs), skeletal stem/progenitor cells, genetically engineered mice, and advanced genetic tools to learn why bone cells behave abnormally. An artificial-intelligence computer screen will search for compounds that target the mutant Gsα protein and lower the cAMP and Wnt signals driving the disease. Promising compounds will be tested in cells and mouse models to identify candidates for future work in people.
Who could benefit from this research
Good fit: People diagnosed with fibrous dysplasia or McCune‑Albright syndrome, especially those willing to provide tissue or cell samples or to be considered for future clinical studies, are the most relevant candidates.
Not a fit: People without FD or GNAS-related bone disease, or those with long‑standing deformities unlikely to be reversed, are less likely to benefit directly from this project.
Why it matters
Potential benefit: If successful, this work could produce drugs that reduce or reverse FD bone lesions, improve bone healing, and lessen pain or deformity.
How similar studies have performed: Earlier mouse experiments showed that blocking excess Gs pathway activity can reverse FD-like lesions, so this builds on promising preclinical results though human treatments remain 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.