I am very pleased to tell you that a Foundation-funded team at The Children’s Hospital of Philadelphia (CHOP) released positive results today regarding the treatment of the second eyes of three adult patients in an ongoing gene therapy trial for Leber congenital amaurosis (LCA). The results: After six months, it proved safe and improved light sensitivity, visual acuity and visual field.
So, what does this mean, exactly? Well, it’s the first time that patients in any retinal gene therapy clinical trials have received treatment in both eyes. And thanks to these successful results, the other nine patients in the CHOP study can now be invited to have their second eyes treated – which, I suspect, they’ve been looking forward to very much. This milestone, achieved by Drs. Jean Bennett and Albert Maguire (wife and husband, in case you didn’t know), is a big boost for the whole retinal gene therapy field, because it establishes a precedent for treating both eyes in human studies.
In fact, one other CHOP participant who has already received some second-eye treatment is Corey Haas, a boy whose first-eye success the Foundation reported on a few years ago. “Corey has had his second eye treated, and is doing well,” Dr. Bennett reports. “We are waiting to measure the results.”
All of this also bodes well for the eventual development of a treatment that can be made available to the public. And, by the way, there are four other LCA clinical trials out there. Collectively, more than 40 people have been successfully treated. In fact, researchers from the Universities of Pennsylvania and Florida recently published very nice three-year results for their clinical study.