A New Drug Could Save This Boy’s Life — But He Can’t Get it

Meet Ryan Dunne, a Colorado 9-year-old who loves to read, paint, play with his friends and dance. In a few years, he will be confined to a wheelchair.

When Ryan was 3, he was diagnosed with Duchenne Muscular Dystrophy, a genetic disorder that causes progressive muscular degeneration. “Ryan’s muscles have basically been breaking down since the day he was born,” says Ryan’s father Chris Dunne. Kids with DMD (it mostly affects boys) usually end up in wheelchairs by age 11 and, thanks to the loss of lung and heart functions, don’t often live past early adulthood. “Every day that goes by I watch my son get weaker and weaker,” says Dunne. “He can’t climb stairs anymore, he can’t play sports with his friends anymore or ride the school bus.” Indeed, it is a heartbreaking condition with very little hope for the future — until now.

For the first time ever, there’s a promising new DMD drug under investigation. In early trials Sarepta Pharmaceuticals found that the drug — called eteplirsen — halted the progression of DMD and had no adverse side effects. It also showed increased effectiveness the longer patients were on it. The standard of care for DMD has always been steroid treatment, which Ryan has been on for years with little improvement (“and the side effects are awful,” says his dad). With the promise of eteplirsen, DMD families were finally given a glimmer of hope. But the U.S. Food and Drug Administration called an expedited approval request for the drug “premature.” The trials have been halted and, with it, the Dunne family’s shot at a better life for their son.

“We recognize the huge unmet medical need in Duchenne muscular dystrophy, the devastating nature of the disease … and the great urgency to make new treatments available,” the FDA said in a letter to families who’ve contacted them about eteplirsen. It goes on to state that more research is needed and that the results of the original trials aren’t open to the public, so they can’t comment further. “Please know that FDA has reached no conclusions about the possibility of using accelerated approval for any new drug for the treatment of Duchenne muscular dystrophy, and for eteplirsen in particular.” So the FDA is not necessarily shutting it down, but here’s the thing: They’ve been sitting on the eteplirsen research now for 16 months. Getting a new drug approved through the normal channels can take years and these kids don’t have years. “There are kids who are walking now who won’t be able to walk by that time,” says Dunne. “There are kids in wheelchairs who will be dead.” Time is literally running out.


“Who knows if it works long term but who knows if it doesn’t?” he says. And there is no alternative. “I could see if there was something else you could do, somewhere else we could go, but there isn’t,” he says. “This is our only hope. My son is going to die unless he gets this medicine.” Even if it buys the family a few years to keep Ryan healthy until something better comes along, wouldn’t it be worth it? Bottom line: This drug gave hope to a hopeless situation and now it’s been taken away.

As someone who is alive today because of a miracle drug that was fast-tracked by the FDA, Ryan’s story struck a chord with me. It is gut-wrenching to think there is a treatment out there that could help these kids but it’s being kept from them. Like my cancer, DMD is considered an orphan disease, meaning it doesn’t affect a large percentage of the population. Drug companies are rarely interested in developing drugs for these diseases because they won’t translate to dollar signs. The fact that there was a company out there willing to try but whose efforts are being thwarted is infuriating and unfair.
 
The Dunnes and other DMD families have reached out to Sarepta Pharmaceuticals, to the FDA and now they’re petitioning to get the White House involved. If they get 100,000 signatures by March 28, their issue will be addressed by government officials. You better believe I’ll be signing that petition. I hope you will too.

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