Last week, the Food and Drug Administration approved the first gene therapy to treat a specific form of inherited blindness called Leber’s congenital amaurosis. Luxturna is the first gene therapy to treat any inherited disease at all. It has been hailed as a scientific breakthrough, but it is going to cost potentially $1 million per patient. The price tag has provoked hard questions about the value of the ability to see. Luxturna’s effects may also be partial and temporary. The company that makes Luxturna, Spark Therapeutics, has yet to set an official cost, but on the November earnings call, CEO Jeff Marrazzo, suggested the high cost is justified in part by the earnings patients and their caretakers could gain. Seventy percent of working-age Americans who are blind are unemployed, he said, citing a National Federation of the Blind, and Marrazzo said patients would gain a lot from Luxturna. But the National Federation of the Blind took issue with being cited this way, and said that Spark was “relying on erroneous and harmful notions about the capacity of blind people to live the lives we want.” Chris Danielsen, a spokesperson for the organization, said that the high unemployment rate is because of “society’s low expectations for the blind.” Stacy Cervenka was born blind due to an undeveloped optic nerve. She told The Atlantic that blind people are not just sitting around waiting for treatment, but that “blind people have lives that are as busy and chaotic and full as any sighted people.” She is not opposed to gene therapy to treat blindness, but emphasized the trade-offs. The therapy comes with risks (they are injecting into the eyeballs), and it takes time to travel to a hospital that offers it. She would only consider it if her vision would be good enough to drive. For some people, especially those who have been blind since birth, being able to see can actually be a “bizarre, disruptive experience.”
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