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A parent fights to speed FDA approval in hopes of saving son's life

Wednesday - 3/26/2014, 5:20am  ET

Niko.JPG
Niko, whose given name is Nikolai, has Duchenne Muscular Dystrophy, a genetic disease that affects only boys -- and kills them young. (Courtesy the Arnold family.)

WASHINGTON -- This is a story about the intersection of hope, fear and risk.

Werner Arnold feels it every day. His 9-year-old son, Niko, has Duchenne Muscular Dystrophy, a devastating genetic disease that only affects boys -- and kills them young.

"They don't live as long as normal children," says Arnold. "They live, if they are lucky, to 20 years old."

The Manassas, Va., father has been searching the Internet for answers and for information about other boys who have the same disease. And that's how he heard about Ryan Dunne, a Colorado kid the same age as Niko, with the same symptoms and the same poor response to available treatment.

Dunne's parents are leading a petition drive to get the Food and Drug Administration to put a promising, but still experimental, drug on the fast track for approval. Arnold and adult members of his family were quick to sign the petition.

"If it can provide some kind of hope and longevity, why not? They deserve to live as long as we do," Arnold says, his voice filled with a father's anguish. "I don't want to lose him."

The petition has taken off online, making it the latest example of how families are using social media and the Internet to press for access to experimental medications they feel could help their desperately ill children.

"They are asking for hope, and hope is a good thing," says Dr. G. Kevin Donovan, director of the Pellegrino Center for Clinical Bio-Ethics at Georgetown University Medical Center.

But he says this is a two-edged sword: Sometimes parents are asking for drugs in early stages of development that have not been proven safe and could hurt, rather than help, their child.

When people turn to social media and appeal for the release of potentially life- saving drugs, "it's heartbreaking," Donovan says. But parents have to understand they are not asking for "guaranteed treatment."

Donovan, who is also a professor of pediatric medicine at Georgetown, says there is the additional ethical issue of "jumping the queue," with its accompanying debate on who should decide which children should get an experimental treatment and which should not. Donovan says a parent's desire to protect and heal their child is incredibly strong, and admits that if his kid's life were at stake, he would not hesitate to ask for a drug he thought would be beneficial.

There are also ethical and legal questions for the drug companies involved, which makes them reluctant to release experimental drugs even on an humanitarian basis.

That was the case with Josh Hardy -- the 7-year-old Fredericksburg, Va., boy whose story made national headlines. He got a dangerous infection after a bone marrow transplant, and his parents waged an all-out social media campaign to get access to unapproved treatment after an initial refusal from the drug company.

Niko Arnold's case is different. His family is not appealing for access to a drug in the testing stage that's not available to others. Instead, they are urging the FDA to speed up the approval process for a new drug for Duchenne Muscular Dystrophy.

Donovan says there is less risk inherent in the "fast track" process, but there is still the chance that in the rush to release the drug, testing might not be as rigorous as it would be otherwise.

In the midst of all this focus on drugs in development, some have suggested it's time to reconsider the entire FDA approval process to cut the bureaucratic obstacles and make it more efficient.

"I think it is a reasonable question to ask," says Donovan. "And I think it is not a bad idea to review the FDA's procedures to see if there is a way to safely accelerate them."

He says the FDA is "notoriously cautious," because in the past, some drugs were released that were later found to be harmful. And, while it may be possible to adopt an accelerated approval process for some drugs, it won't work for all.

"I really think it is a mistake to go around the testing process entirely because I think that puts patients at risk."

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