Study finds that gene therapy for Leber Congenital Amaurosis (LCA) helps kids most

Scientists at OHSU Casey Eye Institute and Baylor College of Medicine's Cullen Eye Institute recently published findings from a clinical trial in the journal Ophthalmology, which showed that children had the greatest benefit from gene therapy for treatment of Leber Congenital Amaurosis (LCA), a rare genetic disorder that causes childhood blindness. 

The two-year study included eight adults and four children and showed that treatment for LCA delivered by injection in the poorer-seeing eye was safe, which was the primary goal of the clinical trial.  Sharpness of vision or visual field improved in nine out of 12 patients.

The study is the first to show improvement in visual field with the use of a novel analytical tool created by Richard Weleber, M.D., senior author and professor of ophthalmology in the OHSU Casey Eye Institute. Called Visual Field Modeling and Assessment, the tool is able to more accurately measure visual field, the total area in which objects can be seen (both central and side vision).

"I am very optimistic about these study findings," said Weleber. "Gene replacement therapy has proven to be the most promising method to halt progression of childhood blindness due to single gene defects. The eye is a perfect platform for gene therapy because we can treat one eye to see how it responds compared to the other eye."

"Treatments for childhood blindness have profound effects because these children have their whole lives ahead of them," said David Wilson, M.D., director of the OHSU Casey Eye Institute, chair of the Department of Ophthalmology in the OHSU School of Medicine and study co-author. "Demonstrating the effectiveness of gene therapy in the eye will have broad implications for the rest of medicine as well."