The National Institutes of Health (NIH) has conducted studies in animals indicating that the drug reserpine, approved in 1955 for managing high blood pressure, may have potential in treating retinitis pigmentosa. This rare inherited disease begins affecting vision from a young age, and currently, there are no available therapies for it. The findings from NIH’s research on rats were published in the journal eLife.
"The discovery of reserpine's effectiveness may greatly speed therapeutics for retinitis pigmentosa and many other inherited retinal dystrophies," remarked Anand Swaroop, Ph.D., the study's lead investigator and senior investigator at NIH's National Eye Institute. He noted that reserpine's neuroprotective impact does not depend on a particular gene mutation.
Inherited retinal dystrophies, including retinitis pigmentosa, cause retinal degeneration, leading to vision loss. Depending on the genetic cause, the condition's onset and progression can vary. Some genetic defects are dominant and require just one mutated gene copy for vision loss to occur, whereas others are recessive, requiring mutations in both gene copies.
While gene therapies show promise, they are costly, time-consuming to develop, and specific to certain genes. The latest NIH study suggests reserpine may help with photoreceptor cell survival, the key cells in converting light to visual signals. Previous research by the Swaroop Lab in 2023 also suggested reserpine's role in managing vision loss associated with LCA10, another type of retinal dystrophy.
The NIH research adopted a rat model with a genetic mutation common among Irish Americans with retinitis pigmentosa. It found reserpine helped preserve rod photoreceptors, vital for low-light vision, particularly in female rats. This sex-specific benefit was unexpected, and Swaroop commented, "We can only speculate about these sex-specific differences. However, future research would benefit from teasing out these differences and understanding them."
Reserpine is no longer widely used for blood pressure due to side effects, but it may still have potential in treating certain retinal conditions due to its ease of eye delivery.
The research received support from the NEI Intramural Research Program. NEI leads the federal effort in researching visual systems and eye diseases.
For more information, visit the National Eye Institute at https://www.nei.nih.gov or the National Institutes of Health at www.nih.gov.