Toby Willis gave up on medical miracles long ago.
The Seattle man's eyesight started deteriorating when he was a kid and by the time he was in his early 40s, his visual world had dimmed to shadows.
Still, he refused to obsess over scientific advances. "I made it a point not to go down those rabbit holes, to be chasing that pipe dream," he said.
Nevertheless, the possibility that something might be on the horizon occasionally tickled at the back of his mind.
So Willis checked in with his eye doctor late last year, setting in motion a train of events that culminated in becoming the first adult to undergo the first gene therapy approved in the U.S. for treatment of an inherited disease.
The medication, called Luxturna, is injected in the eye and uses a harmless virus to deliver working versions of a gene to the cells of patients whose own genes are defective. It targets a single, rare mutation, but has raised hopes that after decades of disaster and disappointment, a wave of gene therapies for other disorders is on the way.
But Luxturna's staggering price tag of $425,000 per eye is raising questions about who can afford the newest medical technologies — and whether the results will justify the costs.
Willis already knows it won't be a miracle cure for him. The therapy is expected to work best in children with early diagnoses, before the disease causes irreversible damage. In Willis' case, doctors hope it will keep the 44-year-old software engineer's eyesight from getting worse and perhaps improve his ability to distinguish objects and navigate the world. Without the surgery, he would eventually lose his eyesight entirely.
