New drug, new hope for student with cystic fibrosis

Published On: Jul 29 2013 10:30:25 PM EDT   Updated On: Jul 30 2013 10:40:00 PM EDT


Imagine being told your child will never grow old and will die in their 30s.  Imagine being told they carry a gene that could be an early death sentence for their own children.  That is the reality of living with cystic fibrosis.   It's a disease that ravages the lungs and digestive system and many people can carry the gene and not even know it.

Kalydeco, a newly-approved drug by the Food and Drug Administration, is giving new hope to people living with a rare form of the disease. A UNF student is one of them.

"Now that I'm on it, I feel like I have a new life," the student said.  "It's twice a day, 12 hours apart, so once in the morning, once at night."

It's a routine the student doesn't mind a bit, considering the alternative.  She was diagnosed with CF at 15 months.  She was always tired, out of breath and as a teenager, was in and out of the hospital for two weeks at a time.  She even had to use a chest compression vest to help her breathe.

While millions of people can carry a cystic fibrosis gene, it takes two people with the gene to pass it to their child.

According to the FDA, Kalydeco isn't for all CF patients.  It's for patients with the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.  Only about 4 percent of CF patients have that mutation.

"To have a drug on the market that lets a parent tell a child that they will lead a normal life if they take this pill is truly miraculous," said Jim McCarthy with Cystic Fibrosis Foundation North Florida.

McCarthy says donations are the key to finding treatments like Kalydeco.

He wants to save those suffering from cystic fibrosis.

"We're not going to stop until we can get to all of them," said an emotional McCarthy.  "And that's our goal.  That's our goal."

There is another drug working its way through FDA trials that can be used to treat 50 percent of the people living with cystic fibrosis.  Again, it's all thanks to donations that fund research.   If you would like to help go to


The views expressed below are not those of News4Jax or its affiliated companies. By clicking on "Post," you acknowledge that you have read the Terms of Service and your comment is in compliance with such terms. Readers, please help keep this discussion respectful and on topic by flagging comments that are offensive or inappropriate (hover over the commenter's name and you'll see the flag option appear on right side of that line). And remember, respect goes both ways: Tolerance of others' opinions is important in a free discourse. If you're easily offended by strong opinions, you might skip reading comments entirely.

blog comments powered by Disqus