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A young man's family's race against time to find a cure for Duchenne muscular dystrophy

A young man's family's race against time to find a cure for Duchenne muscular dystrophy

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LOS ANGELES (Circa)--Paul and Debra Miller are in a race against time to save a generation of boys with Ducehnne muscular dystrophy. One of those boys is their son.

"Hawken was diagnosed with Duchenne muscular dystrophy in November of 2002," said Debra Miller, who lives in California with her husband and son.

Duchenne is a fatal muscle disease that affects 1 in 7,250 boys, according to a 2015 report from the University of Iowa. The disease doesn't allow bodies to repair after normal wear and tear.

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(L to R) Paul, Hawken and Debra Miller at the University of Southern California, where Hawken is a student.

"It doesn't work the same way for my muscles because I'm deficient in a protein," said Hawken Miller.

I've had to adapt my passions.
Hawken Miller, diagnosed with muscular dystrophy

That protein is called dystrophin.

"So when it breaks, it doesn't really grow back and that can turn into fat," said Hawken. He agreed to meet me at the University of Southern California where he is a junior. Between classes, internships and social activities, he doesn't really talk about Duchenne everyday. He says people don't really ask him about it or why he rides a motor scooter everywhere, but he agreed to talk to me about the effect Duchenne has had on his life.

"I've had to adapt my passions," he says, referring to the fact that growing up he wasn't able to play sports like most kids.

Lacking the protein Hawken does means leads to heart muscle disease, which means most people with Duchenne don't live past their 20s. Hawken is 21 years old.

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"We were disappointed to find that most physicians and very few scientists were really focused on this disease," said Debra Miller. "I think we always said, 'If being a victim and helpless would help our son, we’d be the best at it.’ But it’s not. And the only thing that’s going to help our son is by thinking and putting a plan as best we can to help him."

We’ve successfully corrected this disorder in mice as well as in dogs with the disease.
Dr. Eric Olson, UT Southwestern Medical

So in 2004, they started CureDuchenne, a foundation that has now raised almost $30 million for research into a cure. And they may have just funded the closest thing to a cure yet: Gene editing.

"What CRISPR-Cas9 allows us to do is to remove those errors in the DNA," says Dr. Eric Olson, the chairman of the Department of Molecular Biology at UT Southwestern Medical and the lead researcher behind this gene therapy that has the Millers so excited.

“You can think of it as a spellchecker," said Dr. Olson. "We’ve successfully corrected this disorder in mice as well as in dogs with the disease.”

If you see a slide of the muscle tissue of a mouse with Duchenne, you'll see a black slate. If you run it through CRISPR-Cas9 technology, you'll see a lot of green circles. That represents the dystrophin that was generated.

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What muscles with muscular dystrophy look like before and after CRISPR gene editing.

"All I can say is that green is my new favorite color," said Debra Miller.

It's difficult to say when researchers will be able to test this technology on humans, but Dr. Olson is optimistic.

The only thing that’s going to help our son is by thinking and putting a plan as best we can to help him
Debra Miller, co-founder of CureDuchenne
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“If things continue to go at the pace that they’ve gone so far, within a couple of years, we could be prepared to begin to initiate a trial. We’re talking years. We’re not talking decades. We’re talking less than five years," said Olson. “To develop a therapy takes a lot of effort from a lot of people and it takes a lot of resources. CureDuchenne stepped forward very early in this process. And they raised money and committed themselves to the effort that we had undertaken. So we have a great debt of gratitude to CureDuchenne as well as to the Millers.”

The Millers and Dr. Olson have since launched Exonics Therapeutics, a biotech company that is developing SingleCut CRISPR gene editing therapies.

The Millers are a little more cautious about their optimism.

“You know, we’ve been doing this a while, so I would consider it controlled enthusiasm," said Paul Miller.

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Hawken at his internship KTLA, a local news station in Los Angeles.

For their son, Hawken, the enthusiasm is even more controlled. He says that while death isn't constantly on his mind, it's definitely a worry.

"It’s definitely a concern, but you know, when it’s my time, it’s my time. I have faith that God has a purpose for me, and he ultimately knows what my path is going to be," said Hawken.

For now, he's busy studying journalism at USC, interning at a local news station in Los Angeles and looking for his next story.

“Keep going, and not let whatever you’re dealing with get in your way," said Hawken. "Because you’re just letting it win that way, and you don’t want to let it win."

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