DENVER (CBS4) - With passion and tears Jennifer Dunne pleads for answers and action from the Food and Drug Administration, which has held up the use of an experimental drug that has shown promise for children like her son, a 9-year-old with Duchenne Muscular Dystrophy, a 100 percent fatal disease.
"They need to move -- we have no more time, no more time," Dunne told CBS4.
Her son, Ryan, a third-grader, was diagnosed with DMD years ago. It is a genetic, muscle-wasting disease that hits young boys and is always fatal, usually by the time the patient reaches their mid-20s.
But two experimental medications have shown great promise in treating the disease. In 2013, Ryan was placed on one of the medications as part of a six-month trial study.
"When there was no hope, all of a sudden things were getting better," said Ryan's father, Chris.
"He walked further, had better stamina and energy and didn't fall into bed saying, 'I'm tired,' " said his mother. "And when he was pulled off of it he went downhill immediately. The drug is effective."
A second medication that has also shown promise in trials and that would likely work for Ryan is called eteplirsen. It has also shown extremely strong results in studies. For more than two years the drug was given to 12 boys with DMD. All reported improvement and none reported any side effects.
Patients and their families have requested the Food and Drug Administration give eteplirsen what's known as "accelerated approval" so patients like Ryan could have access to it right away, even as studies continue.
Dr. Jerry Mendel, one of the leading researchers into DMD, recently told congressional leaders and the FDA, "This drug works." Mendel and other researchers urged the FDA to fast track eteplirsen and get it into the hands of patients.
But the FDA has so far refused to grant accelerated approval for eteplirsen despite evidence of its efficacy. Catherine Chew, the FDA's Acting Director of Drug Information, recently wrote families with children afflicted with DMD.
"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. FDA is fully committed to make safe and effective drugs available for patients with Duchenne muscular dystrophy as soon as possible," wrote Chew.
Chew said the FDA has an "evolving position on these drugs." That answer -- and perceived foot dragging by the FDA -- has the Dunnes and other families furious.
"They have in their hands the data that shows it's effective," said Jennifer Dunne. "We don't understand why they aren't granting accelerated approval. They're taking kids' lives away every day they don't grant it. They're taking kids' lives away – it's that simple."
With tears in his eyes, Chris Dunne said, "Every single day matters. We don't have three to four years for trials. The kids who need this won't be around in three or four years."
The Dunnes say Ryan grows weaker every day and will eventually end up in a wheelchair because of the disease. They say eteplirsen is safe and effective. Asked if the medication might have negative side effects, Chris Dunne said, "The real side effect is they are going to die without the medicine."
In an attempt to bring more pressure to bear, the Dunne's and other DMD families are urging people to sign an online petition urging the FDA to grant accelerated approval for eteplirsen. They say if 100,000 people sign the petition, the White House might consider looking into the issue of accelerated approval.
"We need the FDA to make a decision," said Jennifer Dunne. "And we need them to make it today."
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