The Wilmer Eye Institute, Johns Hopkins Medicine has received two awards totaling more than $20 million from the National Eye Institute at the National Institutes of Health, both of which will be used for a six-year clinical trial investigating a potential treatment for the most common inherited retinal disease, retinitis pigmentosa (RP). The condition causes retinal cells to break down, resulting in gradual and permanent vision loss. Currently, RP has no treatments.

Researchers hope to change the landscape of retinitis pigmentosa care with these awards, which they will use to look at whether the drug N-acetyl cysteine (NAC) may slow or halt vision loss from the condition when compared to a placebo.

Previously approved by the Food and Drug Administration to treat acetaminophen overdose, NAC is an antioxidant drug that reduces oxidative stress, which is a molecular mechanism in the body thought to play a role in retinitis pigmentosa. Previous animal studies, including those conducted by the Peter Campochiaro laboratory at Wilmer, showed that high doses of NAC reduced cell damage caused by oxidative stress, while simultaneously reducing vision loss.