Repurposed Blood Pressure Drug Shows Promise in Preventing Inherited Vision Loss

A medication originally developed to control high blood pressure may offer new hope for individuals with inherited blinding disorders. In preclinical studies conducted by the National Institutes of Health (NIH), the drug reserpine was found to preserve retinal neurons essential for vision, especially in female rats. These findings suggest potential for reserpine as a treatment for retinitis pigmentosa, a rare genetic disease that causes progressive vision loss beginning in childhood.

Retinitis pigmentosa is one of many inherited retinal dystrophies that lead to degeneration of the retina—the light-sensitive tissue at the back of the eye. These disorders can be caused by mutations in over 100 different genes and can vary significantly in onset and progression. Existing treatment options remain limited, with gene therapy offering promise but facing challenges due to high costs and the need for mutation-specific solutions. The NIH study, published in eLife, reveals that reserpine’s protective effects on retinal cells are not tied to a specific gene mutation, making it a potentially broad-spectrum therapeutic option.

The study specifically focused on a rat model carrying a dominant mutation in the rhodopsin gene, which is known to cause a form of retinitis pigmentosa commonly seen in Irish Americans. The researchers found that reserpine preserved the function of rod photoreceptors—cells responsible for low-light vision—by maintaining their ability to perform phototransduction, the process of converting light into electrical signals sent to the brain. The treatment also showed preservation of cone photoreceptors, which are responsible for color vision in bright light. Interestingly, the protective effects were more pronounced in female rats, suggesting potential sex-specific responses that warrant further investigation.

Building on previous work showing reserpine’s benefit in another inherited retinal disease caused by mutations in the CEP290 gene, the NIH team plans to develop more potent reserpine-like drugs. These next-generation compounds could be used to delay vision loss in slowly progressing forms of retinal dystrophies or in aggressive cases of retinitis pigmentosa, potentially buying time until gene or regenerative therapies become available. Unlike its former use for hypertension, the doses required for vision protection would be much lower and delivered directly into the eye, reducing the risk of side effects. As a small molecule drug, reserpine also offers advantages in delivery and manufacturing scalability.

Article by multiple RFHC contributors, based upon information from a National Eye Institute (NEI) press release.

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