Combination drug approach to slow vision loss in Stargardt disease
Official title: Polypharmacological approach to treatment of Stargardt disease
Funded at Columbia University Health Sciences · NIH-11303329 · Data updated 2026-11-12
This project tests a combo-drug strategy to lower toxic eye pigments and help protect vision for people with Stargardt disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | March 1, 2023 |
| Project end | February 28, 2027 |
| Funding year | 2026 |
| Award (this year) | $617,264 |
| NIH project number | 5R01EY034461-04 |
| Project ID | NIH-11303329 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with genetically confirmed ABCA4-related Stargardt disease, especially those with early or progressive macular degeneration, would be the intended candidates for future trials.
✕Less likely to help
Patients with unrelated causes of vision loss, non-ABCA4 macular diseases, or very late-stage retinal degeneration with extensive cell loss are unlikely to benefit.
What this research studies
From a patient's perspective, the team is developing medicines that reduce buildup of harmful bisretinoid pigments that damage retinal cells in ABCA4-related Stargardt disease. Because blocking the key enzyme RPE65 alone can cause severe retinal toxicity, researchers plan to use a polypharmacological approach that gently targets multiple steps in the visual cycle to avoid that toxicity. They will screen and refine candidate compounds in lab and animal models of Stargardt disease to find options that lower toxic pigments without harming the retina. Promising leads would be advanced toward safety testing and possible future clinical trials.
Why it matters
Potential benefit: If successful, this work could slow progression of vision loss in people with Stargardt disease by reducing toxic pigment buildup in the retina.
How similar studies have gone: Previous animal studies showed that blocking RPE65 reduced harmful bisretinoids but caused severe retinal toxicity, so this combined-target approach is a relatively novel strategy.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: Petrukhin, Konstantin — Columbia University Health Sciences
- Study coordinator: Petrukhin, Konstantin
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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.