New treatment approach for a rare bone disease using small molecules
Small molecules combination therapy using polypharmacology approach as a novel treatment paradigm for rare bone disease
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 ID | NIH-11005466 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are infants and children diagnosed with Progressive Osseous Heteroplasia.
Not a fit: Patients with other forms of ossification disorders or those not diagnosed with Progressive Osseous Heteroplasia may not benefit from this research.
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 performed: 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
Potomac, United States
- 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 below.