New treatment approach for a rare bone disease using small molecules
Official title: Small molecules combination therapy using polypharmacology approach as a novel treatment paradigm for rare bone disease
Funded at Nostopharma, LLC · NIH-11005466 · Data updated 2026-11-12
This study is looking at a new way to help kids with Progressive Osseous Heteroplasia (POH) by using small doses of a special treatment that could reduce joint stiffness and stop extra bone growth, making it a friendlier option than other treatments.
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 | March 1, 2024 |
| Project end | July 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $44,394 |
| NIH project number | 3R43TR004574-01A1S1 |
| Project ID | NIH-11005466 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are infants and children diagnosed with Progressive Osseous Heteroplasia.
✕Less likely to help
Patients with other forms of ossification disorders or those not diagnosed with Progressive Osseous Heteroplasia may not benefit from this research.
What this research studies
This research focuses on Progressive Osseous Heteroplasia (POH), a rare genetic condition that causes abnormal bone growth in infants and children. The approach involves developing small, locally administered doses of Hedgehog signaling inhibitors to reduce joint stiffness and prevent further ossification. By targeting the underlying genetic mutations and signaling pathways, the research aims to provide a more effective treatment option with fewer side effects compared to systemic therapies. The goal is to demonstrate the feasibility of this novel treatment in a preclinical setting.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for children suffering from Progressive Osseous Heteroplasia, potentially improving their quality of life and mobility.
How similar studies have gone: While the specific approach of using small molecule combination therapy for POH is novel, similar strategies targeting Hedgehog signaling have shown promise in other conditions involving abnormal bone growth.
Where this research is happening
-
Nostopharma, LLC
Potomac, United States Active
Researchers
- Principal investigator: Jeremic, Jelena Gvozdenovic — Nostopharma, LLC
- Study coordinator: Jeremic, Jelena Gvozdenovic
Related research
Other NIH-funded projects on the same topics.
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Combining existing drugs to treat a rare bone disease
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.
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.