Combining existing drugs to treat a rare bone disease
Official title: Small molecules combination therapy using polypharmacology approach as a novel treatment paradigm for rare bone disease
Funded at Nostopharma, LLC · NIH-10759694 · Data updated 2026-11-12
This study is looking at a new way to treat Progressive osseous heteroplasia (POH), a rare bone disease, by using existing medications in a new combination to help reduce side effects and improve how your body forms bone, which could lead to a better quality of life for patients.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Nostopharma, LLC NIH-funded |
| Lab location | 1 site (Potomac, United States) |
| Project start | August 10, 2023 |
| Project end | July 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $318,796 |
| NIH project number | 1R43TR004574-01A1 |
| Project ID | NIH-10759694 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Progressive osseous heteroplasia or similar rare bone disorders.
✕Less likely to help
Patients with common bone diseases or those not diagnosed with Progressive osseous heteroplasia may not benefit from this research.
What this research studies
This research focuses on developing a new treatment for Progressive osseous heteroplasia (POH), a rare genetic bone disease with no current effective therapies. The approach involves repurposing existing small molecules and combining them to create a synergistic effect that can reduce dosage and toxicity. By targeting the Hedgehog signaling pathway, the research aims to alter the differentiation of mesenchymal progenitor cells, which are responsible for abnormal bone formation in soft tissues. Patients may benefit from a novel therapeutic option that could improve their quality of life.
Why it matters
Potential benefit: If successful, this research could provide a new, effective treatment option for patients suffering from Progressive osseous heteroplasia.
How similar studies have gone: While the approach of repurposing drugs for rare diseases is gaining traction, this specific combination therapy targeting the Hedgehog pathway is relatively novel and has not been extensively tested.
Where this research is happening
-
Nostopharma, LLC
Potomac, United States Active
Researchers
- Principal investigator: Jeremic, Jelena Gvozdenovic — Nostopharma, LLC
- Study coordinator: Jeremic, Jelena Gvozdenovic
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.