Families Ask FDA To Fast-Track Duchenne Drug
TREVOSE, Pa. (CBS) -- Parents of children with Duchenne muscular dystrophy are fighting for their sick children, pleading with the FDA to approve the first drug to treat the disease. Some FDA reviewers have doubts that the drug is effective, so a family from Trevose is joining dozens of others, going to Washington, D.C. to plead their case.
Every year, Duchenne muscular dystrophy steals more of Jake Wesley's strength. A simple trip up the stairs is a long process.
"This is the one thing that the FDA doesn't see, the human side," said his father, Keith Wesley, as he helped lift Jake into a harness to go up the stairs.
The fatal genetic disorder prevents Jake's body from producing a key protein.
"It makes me weaker and less able to do things that people can normally do," Jake said.
The disease can also cause scoliosis. Jake recently needed painful surgery to reinforce his spine.
"If you can save one family and one child from having to go through that, it's worth it," Keith said.
Keith sees promise in an experimental drug called eteplirsen. Its maker, Sarepta, says in small exploratory studies, the drug helped Duchenne patients create the missing protein and slow the progression of the disease. Keith and Jake know a patient who is receiving the drug a clinical trial.
"His friend is actually able to lift his hand over his head now, so the drug does work," Keith said.
But Jake hasn't been able to take the drug because it's not approved. So Keith and other families are traveling to Washington to make their case in person for fast-track approval in front of an FDA advisory committee.
"I'm gonna be by my dad's side as he speaks," Jake said.
The FDA has been reluctant to approve the drug so far, saying there's not enough evidence it works. But Keith says, if there's ever a time for the FDA to give dying children some hope, it's now.
"Congress gave you the tools. Use them," Keith said.
The hearing in Washington was scheduled for Friday, but because of the upcoming storm, it has been delayed for at least several weeks. Keith and other parents will have to wait even longer for their chance to plead their case to the FDA.
