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Health Beat: Miracle drug for cystic fibrosis

Published On: Dec 03 2013 02:12:18 PM EST   Updated On: Dec 03 2013 05:43:27 PM EST

Cystic fibrosis is a hereditary disease that causes thick, sticky mucus to build up in the lungs.


Cystic fibrosis is a hereditary disease that causes thick, sticky mucus to build up in the lungs.

Just decades ago, children born with CF would live only into their teens.  Today, the life expectancy has increased to 37 and a new miracle drug is expected to have even better results.

Out of the 25 pills 8-year-old Caleb Nolan takes every day for CF, Kalydeco is his favorite.


"So it is very important and very helpful," he said.

Kalydeco is the first medication to target the underlying cause of CF, a single protein called CFTR that doesn’t work right.

"In the past, we've only been able to treat the complications of cystic fibrosis," said Dr. Frank J. Accurso, professor of pediatrics, University of Colorado Denver School of Medicine and Children's Hospital Colorado.

CFTR doesn't allow fluids to clean the surface of the lungs that can cause deadly infections. 

"The new treatment, Kalydeco, actually makes the protein work better," Accurso said.

It clears out the lungs, reduces symptoms, and possibly also prevents further damage to the lungs.

Caleb still has to use a machine to shake loose mucus in his lungs, but he's feeling healthier than ever before.  

"To know that we have something that we give him now every day that's changing the way his body feels, correcting the underlying defect, there aren't any words for that," said Melissa Nolan, Caleb's mother.

Kalydeco currently is approved only for use by four percent of CF patients with a specific gene mutation. The U.S. Food and Drug Administration, however, has now fast-tracked Kalydeco-combination-treatments that may help up to 65 percent of cystic fibrosis patients in the near future.

The drug costs $300,000 a year, but almost all insurance companies will help with the cost.

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DOWNLOAD and VIEW the full-length interview with Dr. Frank J. Accurso about a miracle drug for cystic fibrosis