Crypto News: FDA considers a drug with mixed results

Hi, it's Angelica in Boston. Families are pleading with the FDA to approve a gene therapy for a debilitating genetic disease. But first…

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Sheila Ungerer started noticing something was amiss with her son Will when he was 3 years old. He couldn't jump with both feet, which she thought might just be a developmental delay.

But within minutes of an exam, a doctor told Ungerer and her husband that their son had a more serious problem. A genetic test confirmed the diagnosis: Duchenne muscular dystrophy, a disease that primarily affects boys and causes their muscles to weaken over time. Most patients die in their 20s.

The diagnosis was an "unexpectedly horrible bit of news," Ungerer says. A few years later, then 5-year-old Will received the opportunity to enroll in a clinical trial of a gene therapy from Sarepta Therapeutics. The one-time treatment promises to help patients produce a protein similar to the one they lack.

While the Ungerers didn't find out for sure that Will received the therapy for a few years, almost immediately they felt pretty sure he got the real thing. Within weeks, he was sitting upside down on the couch and running up to the top of a hill before rolling down it. Now 9, Will swims on a team and plays tag at recess. He can jump with two feet now, though he won't play soccer. But that's OK.

Ungerer recently shared Will's story at a meeting of independent experts advising the FDA on whether to approve the gene therapy. Will's story and others about boys who received Sarepta's drug helped convince the panel to narrowly recommend approving the treatment despite concerns the data doesn't exactly match those anecdotes.

In the trial Will participated in, Sarepta's therapy didn't clearly beat the placebo. FDA reviewers also worry the protein the therapy elicits isn't the same as the natural protein. Since patients may have only one shot at receiving gene therapy, the stakes are especially high.

The FDA is in a tough spot. Patients and families are desperate for new treatments, even imperfect ones.

"From my perspective, it's like, approve it," Ungerer says. "Let it go and let them do it and let it help people in the way that it helps Will because it's worth it."

Sarepta is currently running a larger trial that aims to answer definitively whether the therapy benefits patients. Results won't be available until later this year, and the FDA is expected to rule on Sarepta's application for an accelerated approval this month.

The agency appears poised to clear the therapy only for children between 4 and 5 years old who saw the best response in the trials, a move that could help the FDA strike a balance. Either way, people on both sides of the debate will probably be dissatisfied. — Angelica Peebles

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