After Nerve-Wracking Eye Surgery, the World Comes into Focus for Early Gene Therapy Recipient

After Nerve-Wracking Eye Surgery, the World Comes into Focus for Early Gene Therapy Recipient (STAT)

(This is a story about the first recipient of Luxturna, which is the first gene therapy for a genetic disorder approved by the FDA. UF Professor William Hauswirth’s work with AAV, in conjunction with inventors from University of Pennsylvania and Cornell University, is the technology behind Luxturna, marketed by Spark Therapeutics.)

The machine looked like a giant eyeball. There was a hole where the pupil should have been, and the technician told Jack Hogan to stick his head inside. As the white dome began to flash with light, electrical messages began zinging up from his retina to his brain — and every flicker of voltage was picked up by the electrode that had been stuck onto his cornea.
“It hurts,” Jack said, his voice echoing around the Giant Eyeball Machine.

About eight weeks earlier, in March, his eyes had been just as hollow. He’d been the first person to get an $850,000 therapy called Luxturna since it had hit the market. It was intended to replace a mutant gene in Jack’s retinal cells that impaired his vision. After the surgery, with his eyes temporarily drained of liquid and pumped full of air, he’d had to lie back for six hours, staring at the ceiling, so that the medication would pool in the right part of his retina. His mother had held an iPad above him, streaming basketball games and music until her arms got tired. Sometimes, she fed him crackers. She could only hope that the drug had worked.

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