BOSTON (CBS) – A medical breakthrough in Boston with far reaching implications offers a young boy a chance to see better. It’s the first time an FDA approved gene therapy has been used to fight an inherited disease. And after groundbreaking surgery at Mass Eye and Ear, both patient and doctor are beaming, with hope.
“This is just the beginning. I’m the first one. There’s going to be more,” says 13-year-old Jack Hogan from New Jersey who has had serious vision problems his whole life. “I have trouble seeing at night. Probably around 6 o’clock, I’ll go inside and I can’t see outside at all.”
Jack also has no peripheral vision. But Tuesday at Mass Eye and Ear in Boston, Jack was the first person outside of clinical trials to receive gene therapy that’s been 20 years in the making. The goal was to replace a gene that Jack is missing.
“This gene therapy was developed to take the synthetic copy of the RPE 65 gene, and put it into the retina,” says Dr. Jason Comander, who performed the surgery. By inserting the synthetic gene, the hope is to stop the progress of Jack’s rare vision loss and actually improve his sight.
The delicate surgery involves taking the synthetic gene which is placed in a virus, and putting it underneath Jack’s retina. “We put tiny little ports into the white part of the eye. Through a tiny, little cannula, the tip of which is the size of a human hair, we inject these three drops of liquid,” says Dr. Comander. “This drug can really be a miracle for a lot of these patients.”
For Jack, success could mean not being left behind. “My classmates were like, ‘oh look at that, oh look at this,’ and I didn’t see anything, so it just got me really upset,” Jack explains.
“I just hope that he’s included now in things, and he’s able to have fun and enjoy life like the rest of the regular 13-year-olds,” says Jack’s mom, Jeanette Hogan.
Jack had surgery on his left eye Tuesday. The right eye is scheduled for next week. He should know in about a month if it works.
This new drug is a big boost for gene therapy, but it comes at a great cost. The price tag is $850,000. Insurance is paying for Jack’s treatment.