Gene therapy could restore sight for hereditary blindness

Posted at 5:12 PM, Oct 12, 2017

It's being called a breakthrough treatment that might let some people who are blind see again. Thursday, an FDA panel recommended approval of the first gene therapy for a form of hereditary blindness.     

Almost every day, you'll find Richard Faubion and his dog Parka walking the paths in City Park and enjoying the views. Even though for Faubian, they're a bit different.

Faubion has Retinitis Pigmentosa, or RP. It's a hereditary eye disease that causes tunnel vision and makes it difficult to see at night.

"I was 21 years old when I found out that I had the disease," Faubion recalls.

After his diagnosis, this sports lover says things in his childhood, started to add up. "I remember playing shortstop when I'd lose balls under my feet and wonder why I didn't see it right away," Faubion remembers.  

And as his disease progressed and he had to stop doing many of the things he loved.

"I was angry and mad and you know why is this happening to me kind of thing," Faubion says.  

Faubion adapted, embracing his new way of living, and working with an organization to raise money for research to find treatments for degenerative eye diseases.

"I felt probably nothing would ever come along in my lifetime or it was too late," Faubion says. "But no, its come along and I'm very excited about that."  

The FDA is considering a gene therapy treatment that has been proven in trials to improve vision in some patients with hereditary blindness. It works by replacing a defective gene that affects vision. If approved, this will be the first gene therapy in the U.S. for an inherited disease.  

"If they will approve that then there are hundreds of patients that would be ready to have their vision restored today," Faubion says.  

Faubion is excited about the possibility of gene therapy being used to restore his sight. But in the meantime, he's living life to the fullest.

"You can do just about anything you want to do," Faubion says.

"The only limitation is between your ears, your mind, your determination to do it or not. If you want to do it you can do it."

The FDA isn't required to follow the panel's recommendation, but these deliberations are usually the final step in the decision making process. The FDA has until mid-January to decide.