Among blind people, says Kim Charlson, asking if you’d prefer to see always starts a lively debate: “Every opinion is going to be different.” Charlson, who lost her sight at age 11 and now is president of the American Council of the Blind, says she would hold out for full color vision. Others might settle for seeing in blurry black and white. And yet other blind people might have no desire to see at all.
For a small number of blind people, this hypothetical question recently become a real one.
Last week, the Food and Drug Administration approved Luxturna, the first gene therapy to treat a specific form of inherited blindness called Leber’s congenital amaurosis. In fact, it’s the first gene therapy to treat any inherited disease at all. The news has been universally hailed as a scientific breakthrough. But its stratospheric cost—potentially $1 million per patient—has provoked hard questions about the value of the ability to see, especially if its effects are only partial and temporary, as may be the case with Luxturna.
Spark Therapeutics, the company that makes Luxturna, has yet to set an official cost. But the $1 million figure comes from a November earnings call, where the company’s CEO, Jeff Marrazzo, suggested the high cost is justified in part by the earnings patients and their caretakers could gain. He cited the National Federation of the Blind to note that 70 percent of working-age Americans who are blind are unemployed. “This economic reality of unemployment reminds us of a wonderful real-life example of the value of Luxturna,” said Marrazzo. One woman in the Luxturna trial regained enough vision to get her very first job at age 38.