Three-pronged drug approach for inherited retinal degeneration
Triple threat therapeutics for inherited retinal degenerations
Researchers are trying two existing drugs to lower harmful lipids and protect eye cells in people with inherited retinal dystrophies like Stargardt and Batten disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11262935 on NIH RePORTER |
What this research studies
If you have an inherited retinal dystrophy, this project is developing a drug-based approach that could help many genetic forms of the disease at once. The team is testing two small-molecule drugs in mouse models: a bisphosphonate that reduces cholesterol and ceramide production, and an adiponectin-receptor agonist that boosts ceramide breakdown and mitochondrial health in the retinal pigment epithelium. The idea is a “triple threat” that lowers cholesterol and ceramide while protecting RPE mitochondria to prevent immune activation and photoreceptor loss. Results in animals will guide whether these drugs can be repurposed and moved toward human testing.
Who could benefit from this research
Good fit: Ideal candidates would be people with inherited retinal dystrophies such as Stargardt disease or Batten disease who are interested in treatments that target common disease pathways rather than a single gene.
Not a fit: Patients with non-degenerative eye conditions or vision loss caused by unrelated mechanisms are unlikely to benefit from this research in the short term.
Why it matters
Potential benefit: If successful, this approach could slow or prevent vision loss across many inherited retinal diseases by targeting shared lipid and mitochondrial problems rather than individual genes.
How similar studies have performed: Prior preclinical studies support targeting cholesterol, ceramide, and mitochondrial health in retinal disease models, but clinical benefit in people has not yet been demonstrated.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Lakkaraju, Aparna — University of California, San Francisco
- Study coordinator: Lakkaraju, Aparna
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.