A new gene therapy is under review by the FDA that if approved, could lead to new treatments for inherited retinal eye diseases. Research for the new treatment was presented today and reported on by Eureka Alert.
Patients in the study had a condition called Leber congenital amaurosis (LCA), which begins in infancy and progresses slowly, eventually causing complete blindness. This new, first-of-its-kind gene therapy is currently under review by the U.S. Food and Drug Administration for potential approval this year. There are currently no treatments available for inherited retinal diseases.
Ophthalmologist Stephen R. Russell, M.D., of the University of Iowa, is one of the lead researchers for this pioneering treatment. Data from the first randomized, controlled, phase 3 study showed that 27 of 29 treated patients (93 percent) experienced meaningful improvements in their vision, enough that they could navigate a maze in low to moderate light. They also showed improvement in light sensitivity and peripheral vision, which are two visual deficits these patients experience.
An approval from the FDA on this form of therapy could expedite a considerable amount of the research and process already being done for related genetic eye diseases.
Clinical trials of gene replacement therapy for LCA caused by mutations in the RPE65 are now underway at Children’s Hospital of Philadelphia, Universities of Pennsylvania and Florida, and Moorfields Eye Hospital at the University College of London. Children and young adults who were nearly blind have had dramatic improvements in their vision after treatment.
The treatments, according to researchers, do not bring back full normal vision, but they can help allow for considerably more independence from other traditional vision support systems like seeing-eye dogs and canes. One of the clear avenues in treatments for many diseases in through gene therapy and the future is upon us.