The Jett Foundation Fighting Duchenne Muscular Dystrophy

WSJ – Boys Not Statistics

July 8, 2016

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Boys, Not Statistics
A drug for muscular dystrophy works better than we reported.
Jack Willis, center, Nolan Willis, right, and Max LeClaire, bottom, attended the opening for Sarepta Therapeutics global headquarters in Cambridge on June 2, 2014.
Jack Willis, center, Nolan Willis, right, and Max LeClaire, bottom, attended the opening for Sarepta Therapeutics global headquarters in Cambridge on June 2, 2014. PHOTO: BOSTON GLOBE/GETTY IMAGES
July 7, 2016 7:27 p.m. ET

Last week we asked the Food and Drug Administration what’s holding up a first-in-class treatment for boys with a rare and lethal form of muscular dystrophy. Still no answer on that. But we understated the promising clinical results from the drug maker, Sarepta Therapeutics, and our error is a chance to note that the statistic captures only part of the therapy’s efficacy.

Ten out of 12 boys with Duchenne muscular dystrophy walk after four years of treatment on eteplirsen, which produces a protein called dystrophin. (We mistakenly said it was eight of 10.) A trial with more than 83% of patients walking longer than the disease’s progression would predict is more than marginal progress. It’s a breakthrough.

The two boys who lost the ability to walk—Jack and Nolan Willis, twins who now use wheelchairs—spoke in the public comment portion of an April FDA committee meeting. “We are not failures because we stopped walking,” they said. The boys testified that they can still brush their teeth, pick up books—abilities that typically would have disappeared sooner. Their hearts and lungs have been performing normally, and that will help them fight the condition longer. (See their testimony here)

Then there’s a blog post last week from Christine McSherry, executive director of the nonprofit Jett Foundation. Ms. McSherry wrote about a conversation with a friend, whose son is in a trial for a different iteration of the Duchenne drug. That mother reported that her son had recently lifted his arms to hug her, an ability he had lost. A restored ability is unheard of in Duchenne.

The point here is not to dispense with science: Eteplirsen deserves approval on the merits of its clinical results; patients also collected hard data on falls, walking after a fracture and more. But not every FDA analysis will include what matters to a boy staring down a 25-year life span.

At the April FDA meeting, several of the eteplirsen boys wore orange T-shirts that said “n=1”—meaning each is a sample size of one. That’s a way of saying what should be obvious: They are not subjects in an experiment, but patients who deserve access to a safe drug that will improve their lives in material ways that a bureaucracy can never measure.

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