Federal panel to consider gene therapy for blindness
A girl saw her mother’s face for the first time. A boy tore through the aisles of Target, marveling at toys he never knew existed. A teen walked onto a stage and watched the stunned expressions of celebrity judges as he wowed “America’s Got Talent.”
Caroline, Cole, Christian. All had mere glimmers of vision and were destined to lose even that because of an inherited eye disease with no treatment or cure.
Until now.
On Thursday, U.S. Food and Drug Administration advisers will consider whether to recommend approval of a gene therapy that improved vision for these three youths and some others with hereditary blindness.
It would be the first gene therapy in the U.S. for an inherited disease, and the first in which a corrective gene is given directly to a patient. Only one gene therapy is sold in the U.S. now, a cancer treatment approved in August that engineers patients’ blood cells in the lab.
A hearing like no other
Children, parents, doctors and scientists will tell the FDA panel what it’s like to lack and then gain one of our most primal senses.
Cole Carper, an 11-year-old boy who got the therapy when he was 8, describes how sight changed what he knew of the world. When he returned to his home in Little Rock, Arkansas, after treatment, “I looked up and said, ‘What are those light things?’ And my mom said, ‘Those are stars.’”
His sister, 13-year-old Caroline Carper, treated when she was 10, said that afterward, “I saw snow falling and rain falling. I was completely surprised. I thought of water on the ground or snow on the ground. I never thought of it falling,” because the sky was something she couldn’t see, along with other things like her mother’s smile.
The treatment, Luxturna, is made by Philadelphia-based Spark Therapeutics. It does not give 20-20 vision or work for everyone, but a company-funded study found it improved vision for nearly all of those given it and seemed safe. The company’s Nasdaq ticker symbol is ONCE, for how often it hopes the therapy is needed.
“It’s exciting” and in some cases might be a cure, although how long the benefits last isn’t known, said Dr. Paul Yang, an eye specialist at Oregon Health & Science University who is testing gene therapies for other companies. “There’s nothing else for these kids.”