Champaign man who's losing sight finally sees a ray of hope

Champaign man who's losing sight finally sees a ray of hope

CHAMPAIGN — The first red flag alerting Keegan Floyd that his vision was fading was a traffic accident.

It happened about a year-and-a-half ago at dusk, and he didn't see the other car, he said.

Floyd, 25, of Champaign learned he had inherited retinitis pigmentosa, an eye disease causing progressive vision loss and eventually, in some people, blindness.

At the time, doctors told him there wasn't a treatment.

"It was a lot to handle all at once," Floyd recalled. "Literally, they told me, 'You have this disease. There's no way to fix it. We can't cure it. We don't know how fast it will progress.'"

Floyd got the first hopeful news about his vision last month when a groundbreaking gene therapy with the brand name Luxturna was approved.

Luxturna is a one-time treatment targeting retinal disease caused by a rare gene mutation, and it's being rolled out with an eye-catching price tag of $850,000 for the necessary injections in both eyes.

The treatment is intended for people who have mutations in both copies of the RPE65 gene that's essential for normal vision, "and they're thinking it will only be applicable to 1,000 to 2,000 patients in the whole country," said Dr. Michael Tsipursky, an ophthalmologist and retinal specialist at Carle.

The RPE65 gene provides instructions to make a protein involved in the steps that convert light entering the eye to electrical signals transmitted to the brain.

"These patients have missing proteins that allow light to be converted to electrical stimulus," Tsipursky said. "The eye can pick up the light, but it doesn't know what to do with it."

The treatment is being injected with a small needle directly beneath the retina, Tsipursky said, and for it to work, patients must have some viable retinal tissue left.

People with inherited retinal disease resulting from mutations in both copies of the RPE65 gene nearly all progress to complete blindness, according to Luxturna's maker, Spark Therapeutics. It often comes with night blindness from decreased light sensitivity in childhood or early adulthood and involuntary eye movements and progresses to a loss of peripheral vision, developing tunnel vision and eventually sometimes the loss of central vision.

Floyd said he doesn't recall signs of vision loss during his childhood. But in just the past year-and-a-half, his vision has narrowed to the point that he no longer drives.

"I still have a valid driver's license, but it got to the point where I took it upon myself where it was better for me not to be on the road," he said.

Currently between jobs, Floyd said he can still see well enough to job-search online, but anything in small print is tough for him to read.

His night vision faded first, he said.

"I really struggle at night," Floyd said.

Since then, his peripheral vision and depth perception that allows for judging distances and seeing objects in three dimensions are "pretty shot," he said.

Floyd said neither of his parents have the same inherited vision loss, but it's turned up in two of his siblings.

Tsipursky said Carle has been screening potential patients who could benefit from Luxturna for about a year, and only one or two out of several dozen would be candidates.

Because the treatment is intended for such a small number of patients, it will initially be available in only about a half-dozen premier institutions that haven't yet been identified, he said.

"This is definitely not going to come to us in Champaign-Urbana for many, many years," he said.

While the price point for Luxturna is high, Tsipursky said the drug-maker is creating assistance programs. He believes most people who could benefit from the treatment will be able to access it.

"They're not going to be selling their houses," he said.

Some assistance programs announced earlier this month by Spark Therapeutics are plans to share risk with certain health insurers by paying rebates if the treatment doesn't work well enough and working with commercial insurance payers so that their specialty pharmacies would purchase the treatment rather than the medical providers administering it. The company also said it's in discussions with the Centers for Medicare and Medicaid Services on a proposal that would allow payers the option of spreading out payment for the treatment over multiple years.

"We believe that access to therapy is a shared responsibility among Spark Therapeutics, payers, health benefit providers, physicians and treatment centers," said Jeffrey Marrazzo, CEO of Spark Therapeutics. "We have been working with stakeholders across the health care sector to help ensure that appropriate patients have access to a product that challenges all of the current conventions of how patients are treated, how products are delivered and how payments are handled."

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