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Groundbreaking Treatment For Muscular Dystrophy Held Up By FDA, Family Says

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Credit: CBS13

Credit: CBS13

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SACRAMENTO (CBS13) – A family is fighting for the life of their young son. He had been undergoing treatment, getting an experimental drug through a study at the UC Davis Medical Center. But that study has ended, and now they fear that he could lose his battle.

Ryan Dunne goes through the motions other 9-year-olds take for granted. He struggles, his muscles failing him more and more. He has Duchenne Muscular Dystrophy; his muscles are wasting away.

“Things are tough, he has a hard time getting up and down stairs,” said Ryan’s mother, Jennifer Dunne.

As his muscles give out, it’s a near certainty the disease will rob Ryan of his mobility, and his life before he’s 30.

“It’s a progressive disease. Every day he gets weaker,” Jennifer said.

There was a time when the downward spiral slowed: When Ryan’s family was flying weekly from Colorado to Sacramento, taking part in a clinical trial with the UC Davis Health System.

“DMD is actually is the most common form of muscular dystrophy in children. It’s actually due to absence of one of the most common and ubiquitous proteins in the human body: dystrophen,” Dr. Craig McDonald said.

Dr. McDonald heads Duchenne research at UC Davis. His team developed a six-minute walk test to gauge muscle strength. The test is now used worldwide.

“These boys are losing muscle tissues, they’re losing muscle fiber, progressively over time,” Dr. McDonald said.

Some of the boys in the study were given one of two medications known as exon blocking compounds. The medication was thought to slow the disease and improve outcomes. Ryan’s family says it did just that.

“He had better color, better stamina, energy, didn’t fall into bed every night saying ‘I’m tired,’” Jennifer said.

The study ended; the data was encouraging. But the drugs that showed so much promise for Ryan and thousands of other kids with DMD are in limbo.

“When he was pulled off the drug, he went downhill immediately. The drug is effective,” said Jennifer Dunne, Ryan’s mom.

Eteplirsen is one of the two drugs. So why hasn’t the FDA approved eteplirsen? Or, at least, given it accelerated approval to enable patients to receive it while it’s studied further?

“We don’t have any more time for Ryan. He loses muscle integrity; he gets weaker every single day. We don’t have the three or four years to send this back for repeated trials,” said Ryan’s father, Chris Dunne.

The Dunnes are urging the FDA to accept and promptly review a new drug application for eteplirsen and to use the accelerated approval pathway.

The FDA responded in a letter, writing that many assessments are ongoing, and that they are legally prohibited from revealing all they know about the medication.

The Dunnes’ supporters refuse to leave it at that. They’re raising awareness through drives and flash mobs, like one that was organized at Ryan’s Denver-area school, trying to gather 100,000 petition signatures to change the FDA’s mind.

For those who know and love Ryan, quitting is not an option.

“It’s obvious, it’s so black and white. There are no ‘ifs’ and or ‘buts,’ they have to move. We have no more time, no more time,” Jennifer said.

The family realizes that even if they get their goal of 100,000 signatures on the petition, it may not have the result of the FDA giving approval to the drug. But, they say they have no other choice than to fight for their boy’s life.

There’s a March 29 deadline to sign the online petition. If you’re interested in joining the Dunnes’ cause, head over to http://theracetoyes.org/.

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