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Dublin boy paves the way for new muscle disease treatment

A Dublin boy has become a pioneer in how doctors treat a common form of Muscular Dystrophy.

A Dublin boy has become a pioneer in how doctors treat a common form of Muscular Dystrophy.

We first shared Addison Jones’ story back in 2015, when he started taking part in a medical study. Now, he’s helped make the treatment possible for other children like him.

When we met Jones two years ago, he loved his videogames and racing toy cars. Fast forward to today, and his eye for cars has graduated to real ones with a bit more horsepower.

The now fourth grade student, though, is in his own race of sorts -- one against a rapidly progressing disease.

Jones has Duchenne Muscular Dystrophy, which is the most common kind in children. It typically affects boys, and as he gets older, it is going to get harder to walk and even stand.

Two years ago, however, his family found hope. Doctors selected him as one of 10 boys in the state for a trial drug called Eteplirsen.

Every Friday, Jones and his family make the trip all the way from Dublin to Children's Healthcare of Atlanta for his treatment. What started out as a trial, has now been approved by the Food and Drug Administration.

Research shows Eteplirsen slows the disease's progression, allowing children to get stronger and walk for longer.

Children with Duchenne's are usually wheelchair dependent by their early teens. Jones can still walk, but he uses a scooter for long distances.

“He is getting up out of chairs [that] he couldn't before. He went with me the other day to Home Depot and said ‘I want to walk,’” says his grandfather, John Dykes.

While that might not sound significant, any sign of improved muscle strength is a huge victory for them.

“We now have the first ever approved drug by the FDA, so this is a very exciting and promising time for our patients,” says Pediatric Neurologist Dr. Han Phan.

Phan explains Duchenne's is caused by a mutated gene called Dystrophin. This drug helps to enhance the production of functional Dystrophin protein.

Even though there is no cure for Duchenne Muscular Dystrophy, Phan says this is progress.

“So we hope that with the approval of this drug, there will be more to come in the future,” says Phan.

“That's what we keep telling him. He's one of the pioneers with this drug,” says Dykes, “to the point that he was in Washington [D.C.] with the FDA and was actually part of them approving it and part of the reason they approved it.”

“It feels good to help other people like me,” Jones says.

The weekly trip to Atlanta and the portal in his chest to deliver the drug took some getting used to, but now he looks forward to his visits.

“Because I get my medicine and it makes me stronger,” says Jones with a smile.

He has the same lofty goal today as he did the first time we spoke -- to be the President of the United States.

What may have seemed like an uphill race, is now speeding closer to the finish line with this approved treatment.

Jones will continue receiving Eteplirsen for the rest of his life.

39 hospitals across the country were part of the trial in which Jones participated. According to Sarepta Therapeutics, the pharmaceutical company that makes the drug, Eteplirsen is now available for any hospital with the capability of giving infusions.

The cost of Eteplirsen is about $300,000 and is based on patient weight.

In February, the FDA approved another drug called Emflaza. According to the FDA, it is a corticosteroid that helps by reducing inflammation and suppressing the immune system.

The cost of Emflaza is approximately $89,000. The drug made headlines, because a version was previously available from Europe with a less expensive price tag.

Follow 13WMAZ's Karli Barnett on Facebook and Twitter.

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