NIH Develops Eye Drops That Delay Vision Loss in Animal Models

Researchers at the National Institutes of Health have developed experimental eye drops that significantly delay vision loss in animal models of degenerative eye diseases, including retinitis pigmentosa. The treatment uses small peptides derived from a natural human protein and may represent a non-invasive way to slow the progression of blindness in people affected by inherited retinal disorders.

The eye drops are based on a protein known as pigment epithelium-derived factor (PEDF), which is found in the eye and known for its ability to protect retinal cells. Because the full PEDF protein is too large and functionally complex for use in treatment, researchers created smaller peptide fragments that could travel through the eye to reach the retina. Two formulations—named 17-mer and H105A—were developed and tested in mice. Both versions were well tolerated and reached the retina within an hour of application.

When tested in mice genetically predisposed to rapid retinal degeneration, daily eye drop treatment with the H105A peptide preserved up to 75% of photoreceptors. These mice also maintained strong retinal responses to light, compared to untreated mice which experienced near-total photoreceptor loss within a week. The results suggest the peptide treatment slows retinal cell death and preserves vision.

To evaluate long-term potential, researchers followed eye drop treatment with gene therapy and found vision was preserved for at least another six months. They also tested the treatment on human retinal tissue models developed in the lab, where it prevented cell death caused by induced stress. These findings indicate that the peptide-based eye drops may be suitable for future human trials and could complement gene therapy approaches by maintaining retinal health until gene treatments become available.

Article by multiple RFHC contributors, based upon information from a press release issued by the National Eye Institute (NEI), part of the National Institutes of Health (NIH).

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