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Fighting for drug

Newfields family fights for approval of 'miracle drug' for ailing boy

Union Leader Correspondent

February 23. 2014 7:26PM

Lucas Currier, 11, of Newfields, loves sports even though he's lived with Duchenne Muscular Dystrophy, which causes the decay of muscles, for most of his life. His family hopes to encourage the U.S. Food & Drug Administration to resume the accelerated approval process for a drug which has shown promising results. COURTESY 

NEWFIELDS — A local family hopes federal officials will resume the study of a "miracle drug" which could help a boy who loves sports, but gets weaker all the time since his muscles can't regenerate.

Lucas Currier, 11, who attends the Cooperative Middle School in Stratham, was diagnosed with Duchenne Muscular Dystrophy (DMD) at the age of 4, according to his mother, Linda.

She said only one in 3,500 boys are afflicted with DMD, which prevents the body from producing the protein dystrophin. As a result, his muscles are unable to regenerate due to the fatal disease.

While Lucas still can walk, Currier said he started using a wheelchair last year. Despite this, Lucas still loves playing all kinds of sports, she said.

"Lucas keeps us motivated," and they all make the most of their time together, Currier said.

Currier said Lucas, who has been obsessed with sports from a young age, is extremely competitive and plays baseball, hockey and power soccer. She said he has been enjoying watching the Winter Olympics.

The Curriers have high hopes about the promise of a new drug — eteplirsen, which was developed by Sarepta Therapeutics. Their anticipation is offset by the frustration with the progress of the U.S. Food and Drug Administration's approval process.

Last year, due to the "overwhelming positive results" of the eteplirsen trials, the FDA granted Sarepta an accelerated New Drug Application (NDA).

Under the accelerated timeline, the FDA could approve eteplirsen for use by the end of the year as opposed to the normal schedule, which could delay access until 2018.

However, the accelerated application was put on hold in November.

Currier said the FDA was concerned about recent issues with another dystrophin-producing drug called drisapersen, which is being developed by Glaxo Smith Kline. She said drisapersen has not shown the same results as eteplirsen, which uses a different process and has exhibited no side effects during a 120-week trial.

According to FDA Spokeswoman Sandy Walsh, federal law prohibits them from discussing the status of drugs under agency review.

"We at the FDA recognize how devastating this disease is to patients and their families, as evidenced by the hundreds of letters submitted to the agency," Walsh said in an e-mail.

While eteplirsen is an unapproved drug and there are no "approved therapies" to treat DMD, FDA regulations allow access to investigational drugs to individuals or groups, according to Walsh.

"However, please know that we will work diligently to review any request made to expedite its review," Walsh said in an e-mail.

Earlier this month, families, clinicians and researchers pleaded their case in Washington, D.C., to encourage the FDA to resume the accelerated application process for eteplirsen.

"In the time they're taking to consider this, kids are dying," Currier said. "Lucas can't wait four years."Currier said some people with DMD have lived until 32, but the average lifespan ends around 20.

She said rebuilding lost or weak muscles would allow greater mobility and quality of life.

"What this drug does is help create dystrophin – it's a miracle," Currier said.

Currier said while he was attending summer camp, Lucas met one of the dozen boys who was taking eteplirsen as part of a clinical trial. She said his roommate didn't require a wheelchair and the drug seemed to stabilize or improve muscle function.

Currier said she hopes to convince other residents to write, call, e-mail or contact their federal officials to ask them to sign a letter asking the FDA to resume the fast-track process for eteplirsen. She said about a dozen federal legislators have signed the letter to the FDA.

"New Hampshire is one of the states we haven't had to fight," Currier said, adding she's gained the support of Gov. Maggie Hassan, the two U.S. Senators – Kelly Ayotte and Jeanne Shaheen – and U.S. Rep. Carol Shea-Porter.

Currier said she will contact U.S. Rep. Ann Kuster as she continues to campaign for support.

"This could be the first generation of kids with DMD who could live," Currier said.

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