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Four-year old fights fatal disease with new treatment

Thursday, September 07, 2017 - 02:19

A four-year old California boy with Batten disease fights for his life with a newly-approved treatment. Bob Mezan reports.

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Ely Bowman is a four-year old bundle of energy. He loves driving his car in the backyard of his Irvine, California home. He practices his bowling technique with his parents, Bekah and Danny. And has an affinity for talking dinosaurs. But every two weeks, things aren't so fun. That's when Ely goes to Children's Hospital of Orange County (CHOC) where he fights for his life. Ely has CLN2, one of many types of a fatal disorder called Batten disease. His brother, Titus, died of it last year at the age of six. The disease is extremely rare, only occurring in about 2 to 4 of every 100,000 births in the U.S. Inherited from parents who each carry a recessive gene, the disease has no cure. But a breakthrough treatment is giving the Bowman family hope. Doctors are giving Ely a newly FDA-approved drug, Brineura, which acts as a replacement for an essential enzyme that his body doesn't produce, called TPP1. Ely's doctor, Raymond Wang, explains. SOUNDBITE: Raymond Wang, Children's Hospital of Orange County physician, saying (English): "Imagine this enzyme is a little Pac-Man that's going into the nerve cells and eating up the junk that's building up inside neurons. Because he can't make it himself, he is now getting every two weeks an infusion that goes into his brain and it basically does the job that the enzyme does in you and I and it helps to chew up that stuff, the waste products." Bekah Bowman believes it's working. SOUNDBITE: Bekah Bowman saying (English): "He's not falling like Titus was. And he is not having any seizures yet, which is really incredible to be a four-year with CLN2 and to not be having seizures." The treatment doesn't come cheap. Brineura can cost as much as 700,000 dollars for just one year. While the Bowman's insurance picks up the majority of the costs right now, they will have to re-appeal for approval next year. But right now the Bowmans aren't focusing on bills; they're savoring the moments that they have with Ely. SOUNDBITE: Bekah Bowman saying (English): "I see Ely playing in the pool or even at the park and I just appreciate it in a whole different way because I know what he's fighting and I know what that means for him to be able to run around do that kind of stuff."

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Four-year old fights fatal disease with new treatment

Thursday, September 07, 2017 - 02:19