Breakthrough drug helps those with cystic fibrosis

3:49 PM, Feb 2, 2012   |    comments
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UNDATED (CBS) -- The FDA approves a new drug doctors are calling a cure to a deadly disease. And some Oklahomans played a part in making it happen. The average life span for someone with Cystic Fibrosis is only 37 years old, but now doctors are calling this the biggest development for the disease since it was first diagnosed.

For the past 9 years, little Caprie and her family have been living with the realities of Cystic Fibrosis. Her mother says, "My child was going to die young, that she may not get married, that she may not even graduate from high school."

But then back in July of 2009 they learned of a clinical study. So they quit their jobs, sold their house, and moved to Tennessee. The mother says, "We were willing to give up everything we knew, give up all of our family that we had here and all of our support just for a chance for our daughter to be healthy."

Within 3 weeks they noticed it was working. She says, "I just sat on the curb and bawled and bawled and bawled."

Dr. James Royall with OU Children's worked on some of the trials. He says, "It will save lives starting immediately. I'm happy to use the word cure, it's a little hard for someone who has been in CF to use the word cure."

Since she started on the drug, Caprie has gained 30 pounds and her lung function is the best it's ever been. With FDA approval, other families now have the same hope. One patient says, "When I found out it was approved I don't think I've ever been so happy in my entire life except when I gave birth to my three kids."

The drug will only work on about 4 percent of Cystic Fibrosis patients with a specific mutation, but Dr. Royal says its proof the process works; giving hope to everyone else. He believes in 10 years or so there will be drug for the most common mutations as well.

The drug will be in pharmacies next week.