Blood Pressure Drug Studied as Potential Therapy for Retinitis Pigmentosa

A decades-old blood pressure medication is being investigated for its potential to slow vision loss in inherited retinal diseases, according to recent animal studies led by researchers at the National Eye Institute (NEI). The team found that reserpine, a drug originally approved in the 1950s, may help preserve retinal cells in a rat model of retinitis pigmentosa (RP)—a rare genetic condition that causes progressive vision loss. The findings were published in eLife.

Animal Studies Suggest Retinal Protection

The research focused on rod photoreceptors, cells responsible for vision in low-light conditions. In rats carrying a mutation in the rhodopsin gene—a common cause of RP—reserpine treatment helped maintain the structural integrity and function of these cells. Specifically, the drug appeared to support phototransduction, the process by which light is converted into electrical signals in the retina and transmitted to the brain.

Interestingly, the protective effect was more pronounced in female rats, which also showed better preservation of cone photoreceptors—the cells that support color and daylight vision. The reason for this sex-specific difference is unclear and, according to the researchers, warrants further investigation.

A Potential Non-Gene-Specific Therapeutic Strategy

Reserpine’s mechanism of action appears to be independent of the specific genetic mutation, making it a candidate for use across a wide spectrum of inherited retinal diseases. Currently, no approved therapies exist for most forms of RP, and while gene therapy shows promise, it tends to be expensive, mutation-specific, and time-consuming to develop.

These findings build on earlier work from the same NIH team, which showed reserpine’s potential in Leber congenital amaurosis type 10 (LCA10)—a different retinal dystrophy caused by mutations in the CEP290 gene.

Because reserpine is a small molecule, it may be suitable for localized delivery to the eye at low doses, potentially reducing systemic side effects that previously limited its use in treating high blood pressure. The research team is also working to develop more potent, related compounds that could be used to delay disease progression in both slowly developing and aggressive forms of retinal degeneration.

Next Steps

While these results are promising, they are based on preclinical studies in animals, and further research is needed before reserpine or related compounds can be considered for human use. The study also underscores the importance of investigating sex-based differences in treatment response, which could shape future personalized approaches to managing inherited retinal disorders.

This work was supported by the NEI Intramural Research Program.