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Retinitis pigmentosa (RP), a group of inherited eye diseases that cause progressive blindness, currently has limited treatment options and no cure. However, a new study published in PLOS Biology suggests that two newly identified small-molecule compounds could help slow retinal degeneration. Researchers used a virtual screening approach to identify compounds that stabilize rhodopsin—a protein involved in vision—offering a potential strategy to delay vision loss in individuals affected by this condition.
RP often results from genetic mutations that cause rhodopsin to misfold, leading to the death of retinal cells. While current experimental treatments, such as synthetic vitamin A derivatives, exist, they have drawbacks, including toxicity and light sensitivity. In search of better alternatives, researchers at Case Western Reserve University conducted a virtual screening to identify compounds that could bind to and stabilize rhodopsin, improving its folding and overall stability.
The screening process identified two non-retinoid compounds capable of crossing both the blood-brain and blood-retina barriers. Laboratory tests showed that these compounds improved rhodopsin function in 36 out of 123 genetic subtypes of RP, including the most common form. Additionally, in a mouse model of RP, the compounds helped maintain photoreceptor cells and improved retinal function.
The findings represent a significant step forward in the search for RP treatments. By stabilizing rhodopsin, these compounds could slow the progression of retinal degeneration and potentially delay vision loss. Given that RP affects approximately 100,000 people in the United States alone, the potential impact of these findings is substantial.
The researchers emphasize that further studies are needed before human trials can begin. As the authors note, RP is a "progressive and currently untreatable blinding disease." This study highlights the promise of pharmacochaperone therapy, offering a potential new therapeutic strategy to prevent vision loss in individuals with RP.