FDA advisers endorse gene therapy to treat form of blindness
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.
“This is the first time that a genetic therapy has been used to treat an inherited disease in the United States,” Narula said. “This disorder we are talking about, LCA is a disorder that has no treatment. It has no cure.”
The FDA is not required to follow the group’s recommendation, but such deliberations are generally the final step in the agency’s decision-making process. The FDA has until mid-January to decide. If approved, it would be the first gene therapy in the U.S. for an inherited disease, offering hope to patients with a variety of related conditions.
The treatment, called Luxturna, 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.
“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.”
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.
Source:-Â https://www.cbsnews.com/news/fda-advisers-review-gene-therapy-to-treat-form-of-blindness/
