Methotrexate and other medicines for rhodopsin-related retinitis pigmentosa
Pharmacological studies of RHO-associated retinitis pigmentosa
This project tests whether methotrexate and similar repurposed drugs can protect vision in people with rhodopsin-linked autosomal dominant retinitis pigmentosa.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-11295659 on NIH RePORTER |
What this research studies
The team is focusing on repurposing methotrexate, a drug already used for cancer and rheumatoid arthritis, as a treatment for RHO-adRP. They will study how methotrexate acts on AMPK and adenosine signaling pathways, compare drug levels in the eye after different delivery routes, and use retinal explant assays to screen other approved drugs that might help. The work builds on preclinical studies and a recent clinical trial that reported improved retinal function after intravitreal methotrexate. The goal is to find safer long-term dosing and additional repurposed medicines that could slow or stop vision loss.
Who could benefit from this research
Good fit: People with genetically confirmed rhodopsin-associated autosomal dominant retinitis pigmentosa, especially those with progressive vision loss, would be the ideal candidates for related clinical work.
Not a fit: People with other genetic causes of retinal degeneration, those with very advanced retinal scarring that cannot be rescued, or those who cannot safely receive methotrexate may not benefit from these interventions.
Why it matters
Potential benefit: If successful, this work could produce a safe, long-term medicine or delivery approach that slows or prevents vision loss in people with RHO-associated adRP.
How similar studies have performed: Early laboratory studies and a recent small clinical trial reported that intravitreal methotrexate improved retinal function, but larger confirmatory trials are still needed.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Chen, Yuanyuan — University of Pittsburgh at Pittsburgh
- Study coordinator: Chen, Yuanyuan
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.