Repurposing a high blood pressure drug may prevent vision loss in blinding inherited diseases

Press release

Tuesday, April 15, 2025

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NIH animal studies show that reserpine protects retinal neurons needed for vision, particularly in females.

Preservation of rod photoreceptor structure is illustrated in these representative images involving female rats. Rod photoreceptors are immunolabeled with REEP6 (green). Rod photoreceptor inner segments are shorter in control-treated rats (left) than in reserpine-treated rats (right).

New studies in rats suggest that the drug reserpine, approved in 1955 for high blood pressure, could treat retinitis pigmentosa, a blinding disease. There is no treatment for this rare inherited condition, which begins to affect vision in childhood. A report on the studies, conducted at the National Institutes of Health (NIH), published today in eLife.

“The discovery of reserpine’s efficacy could significantly accelerate the treatment of retinitis pigmentosa and many other inherited retinal dystrophies, which can be caused by any of a thousand possible mutations affecting more than 100 genes. The neuroprotective effect of reserpine is independent of any specific underlying genetic mutation,” said study lead investigator Anand Swaroop, Ph.D., a senior investigator at the NIH’s National Eye Institute.

Inherited retinal dystrophies cause degeneration of the retina, the light-sensitive tissue at the back of the eye. Vision loss can be present at birth or develop later in early adulthood. Disease progression varies depending on the gene involved. Some genetic abnormalities can be inherited as dominant, where a mutation in only one of the two copies of the gene (one from the mother and one from the father) is enough to cause vision loss. Other genetic abnormalities are recessive, where both copies of a gene must carry a mutation to cause vision loss. Gene therapies to correct inherited retinal dystrophies are promising, but their development is lengthy, they are gene-specific, and they are often quite expensive.

These results constitute the latest evidence that reserpine improves the survival of photoreceptor cells, the light-detecting retinal neurons that die in retinitis pigmentosa and other retinal dystrophies. In 2023, Swaroop lab has demonstrated the potential of reserpine to prevent vision loss due to LCA10a retinal dystrophy caused by gene mutations CEP290 .

In their latest work, Swaroop's team tested reserpine in a rat model of a dominant form of retinitis pigmentosa caused by a mutation in the gene for the visual pigment, rhodopsin. This disease mutation is common in Irish Americans with retinitis pigmentosa. Compared to untreated rats, reserpine preserved the process by which photoreceptors convert light entering the eye into electrical signals sent to the brain to produce vision, called phototransduction, in retinal cells called rod photoreceptors. Rod photoreceptors enable vision in low light; cone photoreceptors enable color vision in bright light.

Unexpectedly, reserpine better protected rod photoreceptors in female rats than in male rats. The scientists also observed a significant preservation of cone photoreceptors in female rats than in male rats.

“We can only speculate on these sex-specific differences. However, future research would benefit from identifying and understanding these differences to lay the foundation for personalized approaches to treating retinal diseases,” Swaroop said.

Swaroop's lab is currently developing other, more potent reserpine-based drugs. The idea would be to use such options to treat late-onset or slowly progressive inherited retinal dystrophies, or simply to delay vision loss in aggressive forms of retinitis pigmentosa until more effective treatments are developed to reverse this vision loss.

Reserpine is no longer used to treat high blood pressure due to its side effects. The dose required to treat retinal degeneration, however, would be very low and administered directly into the eye. Reserpine is a small molecule therapy, making it easier to deliver to target tissues in the eye.

This work was supported by the NEI Intramural Research Program.

The NEI leads the federal government's research on the visual system and eye diseases. The NEI supports basic science and clinical programs to develop sight-saving treatments and address the unique needs of people with vision loss. For more information, visit https://www.nei.nih.gov.

About the National Institutes of Health (NIH): The NIH, the nation's medical research agency, comprises 27 institutes and centers and is a component of the U.S. Department of Health and Human Services. The NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research. It studies the causes, treatments, and cures for common and rare diseases. For more information about the NIH and its programs, visit www.nih.gov.

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