Methotrexate and other medicines for rhodopsin-related retinitis pigmentosa
Official title: Pharmacological studies of RHO-associated retinitis pigmentosa
Funded at University of Pittsburgh at Pittsburgh · NIH-11295659 · Data updated 2026-11-12
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 start | August 1, 2020 |
| Project end | February 28, 2030 |
| Funding year | 2026 |
| Award (this year) | $525,957 |
| NIH project number | 2R01EY030991-06 |
| Project ID | NIH-11295659 on NIH RePORTER |
Who could benefit from this research
✓Could help
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.
✕Less likely to help
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.
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.
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 gone: 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
-
University of Pittsburgh at Pittsburgh
Pittsburgh, United States Active
Researchers
- Principal investigator: Chen, Yuanyuan — University of Pittsburgh at Pittsburgh
- Study coordinator: Chen, Yuanyuan
Related research
Other NIH-funded projects on the same topics.
-
Inflammation in MERTK-related retinitis pigmentosa
This project aims to understand whether losing the MERTK gene causes retinal inflammation that leads to vision loss in people with MERTK-related retinitis pigmentosa.
-
Enhancing cone survival in RP through targeted cell-specific CRISPR therapy
A patient-friendly look at a new approach to protect cone cells in retinitis pigmentosa using precise gene editing to support vision.
-
Oral, once-daily MC16 medicine for retinitis pigmentosa
A gene-mutation-agnostic pill (MC16) is being studied to help people with retinitis pigmentosa by addressing a common cellular problem in the eye.
-
Role of IPM Proteoglycans in Retinal Health and Inherited Blindness
This grant studies how specific retinal extracellular matrix proteins (IMPG1 and IMPG2) affect eye health and contribute to retinitis pigmentosa, with potential implications for people who have or are at risk for inherited blindness.
-
NAC Attack: a late-stage trial for preserving vision in RP patients
A large, multi-site study is testing whether taking a daily NAC pill can help people with retinitis pigmentosa keep more of their vision.
-
Non-invasive biomarker for mitochondria health in retinitis pigmentosa
Aims to test a new, non-invasive OCT-based biomarker to explain and predict how mitochondria function affects cone cells in people with retinitis pigmentosa.
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.