Gene Therapy Shows Promise for a Form of Blindness, but Is It a Cure? – The Wall Street Journal

A clinical trial of a gene therapy for an inherited condition leading to vision loss made an unexpected finding: Patients treated in one eye experienced improvement in the other eye as well.

Researchers gave the experimental therapy to 37 people with Leber hereditary optic neuropathy, or LHON, a rare genetic disease that can lead to irreversible vision loss, according to a paper published Wednesday in Science Translational Medicine. The disease, which primarily affects young men, can cause sudden loss of central vision in one eye, usually followed by the other eye within a short period of time.

Patients in the study received a single injection of the gene therapy in one eye and a placebo, a sham injection, in the other, with the idea that the drugs effectiveness could be determined by comparing vision changes in the two eyes. Instead, after 96 weeks of follow up, the researchers found that 78%, or 29 patients, had an improvement of vision in both eyes.

The eye turns out to be a really good place to do gene therapy, said Mark Pennesi, chief of the Paul H. Casey ophthalmic genetics division at Oregon Health & Science Universitys Casey Eye Institute, who wasnt involved in the study. Dr. Pennesi said 11 gene-therapy trials for eye disorders are under way at OHSU and more are set to start soon.

Growing numbers of gene-therapy trials have been launched since the Food and Drug Administration approved Luxturna in 2017 to treat another form of inherited vision loss. Developed by Spark Therapeutics, Luxturna uses an injection in the eyes to deliver a normal copy of the RPE65 gene directly to retinal cells. These cells produce a protein that converts light into an electrical signal in the retina and restores vision.

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Gene Therapy Shows Promise for a Form of Blindness, but Is It a Cure? - The Wall Street Journal