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

Tuesday, April 15, 2025

NIH studies in animals show reserpine protects retinal-neurons necessary for vision, especially in females

New studies in rats suggest the drug reserpine, approved in 1955 for high blood pressure, might treat the blinding disease retinitis pigmentosa. No therapy exists for this rare inherited disease, which starts affecting vision from childhood. A report on the studies, conducted at the National Institutes of Health (NIH), published today in eLife.

“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. Reserpine’s neuroprotective effect is independent of any specific underlying gene mutation,” said the study’s lead investigator, Anand Swaroop, Ph.D., senior investigator at NIH’s National Eye Institute.

Inherited retinal dystrophies cause degeneration of the retina, the light-sensing 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 defects may be inherited as dominant, where a mutation in just one of the two copies of the gene (one each from the mother and father) is sufficient to cause vision loss. Other genetic defects 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 take a long time to develop, are gene specific, and are often quite expensive.

The preservation of rod photoreceptor structure is shown in these representative images involving female rats. Rod photoreceptors are immunostained with REEP6 (green). Rod photoreceptor inner segments are shorter in the control-treated rats (left) compared with those treated with reserpine (right).