NIH Study Finds Repurposed Blood Pressure Drug May Help Prevent Inherited Vision Loss

Researchers at the National Institutes of Health (NIH) have discovered that a decades-old medication, once used to treat high blood pressure, may offer protection against vision loss caused by certain inherited retinal diseases. The drug, reserpine, was found to help preserve critical cells in the eye in preclinical models, offering hope for conditions like retinitis pigmentosa, a leading cause of inherited blindness.

The study, led by Dr. Anand Swaroop at the National Eye Institute (NEI), showed that reserpine could slow degeneration of rod photoreceptors—cells responsible for night and peripheral vision—in a rat model of autosomal dominant retinitis pigmentosa (adRP). This disease is typically caused by one of many gene mutations that progressively destroy photoreceptor cells in the retina.

“The discovery of reserpine’s effectiveness may greatly speed therapeutics for retinitis pigmentosa and many other inherited retinal dystrophies, which can be caused by one of more than a thousand possible mutations affecting more than 100 genes,” said Dr. Swaroop. “Reserpine’s neuroprotective effect is independent of any specific underlying gene mutation.”

One particularly notable finding was that reserpine provided significantly greater protection in female rats, where it preserved not only rods but also cone photoreceptors, which are responsible for color vision and clarity in daylight. While the reason for this sex-specific response is still unclear, the results point to a need for further investigation into hormonal or genetic factors that may influence treatment outcomes.

Reserpine, approved in 1955 as a blood pressure medication, fell out of favor due to side effects at higher doses. However, the low doses used in the current study, and the potential for direct delivery to the eye, suggest it may be safe and practical in a retinal context.

This research expands on previous work by the same team, which in 2023 found that reserpine could protect against vision loss in Leber congenital amaurosis type 10, another severe inherited eye disease. The current findings indicate that reserpine—or similar compounds—could serve as a broadly applicable treatment approach for a range of genetically diverse retinal conditions.

The study appears in the journal eLife and highlights the promise of drug repurposing as a faster, more cost-effective path to new therapies—especially for rare diseases with limited treatment options.

For further details, visit the full NIH release: https://www.nih.gov/news-events/news-releases/repurposing-blood-pressure-drug-may-prevent-vision-loss-inherited-blinding-diseases