Promising, but pricey treatment offers hope to people with Cystic Fibrosis

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KANSAS CITY, Mo. -- There's a new weapon to fight a serious genetic disease. Cystic Fibrosis causes sticky secretions in the lungs and digestive system and takes many lives before age 40. A pricey, but promising drug could help change that. Orkambi is for teens and adults with the most common CF gene mutation. About half of CF patients could potentially benefit.

14-year-old Henry Freeman must take a lot of medicine daily to live with CF.

"Close to 40 pills," said Henry.

None may prove more important than Orkambi. It just received FDA approval. Henry took part in the study at Children's Mercy Hospital.

"I feel like I can breathe a lot better," he said.

His mother, Stephanie Freeman, added, "He's been doing great. He's been sick less, and he's been growing."

Dr. Philip Black, a pediatric pulmonologist at Children's Mercy, says the drug gets close to the root cause of CF and could be a turning point in reducing serious lung infections and early deaths.

"Possibly to the point that CF is not gonna be the cause of death for any of these people," said Dr. Black.

But Orkambi has a startling price -- $259,000 a year. The Freemans believe their insurer will cover it, but will the price puts the drug out of reach for some?

"There's a commitment by the company that makes it and the foundation that that won't occur," said Dr. Black.

He's referring to the Cystic Fibrosis Foundation which supported the research.

"It's nice to be able to tell our friends when we're doing the fundraisers and what not (that) we're getting results," said Stephanie Freeman.

Henry's brother organized a tennis tourney that raised $60,000 for more research.

"It gives me a lot of hope," Henry said, referring to the approval of Orkambi.

He has hope that there will someday be a cure for CF.

Orkambi is not a cure, and it's not known yet how much it will reduce the need for other medications. That's led some to say it's an awfully high price for something that's a life-long treatment and not a cure.

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