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Miracle Drug for Cystic Fibrosis
– Reported, November 19 2013
DENVER, Co. (Ivanhoe Newswire) — Cystic fibrosis (CF) is a hereditary disease that causes thick, sticky mucus to build up in the lungs. Just decades ago, children born with CG 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 eight-year-old Caleb takes every day for CF, Kalydeco is his favorite.
So it is very important and very helpful, Caleb Nolan told Ivanhoe.
Kalydeco is the first medication to target the underlying cause of CF, a single protein called CFTR that doesnt work right.
In the past weve only been able to treat the complications of cystic fibrosis, Dr. Frank J. Accurso, Professor of Pediatrics University of Colorado Denver, School of Medicine and Childrens Hospital Colorado, told Ivanhoe.
CFTR doesnt allow fluids to clean the surface of the lungs that can cause deadly infections.
The new treatment, Kalydeco, actually makes the protein work better, Dr. 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 hes feeling healthier than ever before.
To know that we have something that we give him now every day thats changing the way his body feels, correcting the underlying defect, there arent any words for that, Melissa Nolan, Calebs mother, told Ivanhoe.
Kalydeco currently is approved only for use by four percent of CF patients with a specific gene mutation. However, the FDA 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.
