New experimental drug could be breakthrough in cystic fibrosis treatment

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by KREM.com & Danielle Grant

KREM.com

Posted on November 20, 2011 at 8:03 PM

Updated Monday, Nov 21 at 12:49 AM

COEUR D’ALENE-- A new experimental drug for cystic fibrosis treatment is showing some extremely promising results, and a family from Coeur d’Alene says it could completely change their lives.

In essence, this new drug could give a young boy his life back.  But the pills aren’t on the market yet, which means Brady Schroeder and his family have to wait.

Rebecca and Brock Schroeder say their 4-year-old son Brady was diagnosed with cystic fibrosis just three weeks after his birth, which is a deadly genetic disease that cuts many young lives short.

Doctors said Brady would be lucky to live to see his upper 30s.

Brady has to be hooked up to a machine to inhale his medicine for an hour twice each day.  Brady also straps into a vibrating vest that helps loosen the mucus that gets clogged up in his lungs.

Rebecca says she dedicated her days and nights to researching her son’s disease, and found information on a drug called Kalydeco.  If the drug is approved, it will be the first to treat the actual disease instead of just the symptoms.

Dr. Michael McCarthy says he didn’t believe he would ever see this happen 20 years ago.

“It’s almost like a man on the moon project,” Dr. McCarthy said.  “Now we know what’s going on with the disease and we’re going to come up with a fix.  They put a ton of fundraising, blood, sweat and tears into doing it.  All of the sudden, here it is, and it’s a very unique and exciting story.”

So far, Kalydeco has seen major success in clinical trials.  Patients can breathe easier and it could give some cystic fibrosis sufferers a second chance at life.

Rebecca says she dreams of the day Brady no longer has to fight the disease with machines every day.  Rebecca says she knows that day is coming, but it is still bittersweet.

The breakthrough drug only works on 4% of cystic fibrosis cases.
    
The Schroeder family says they are a little impatient and want to get their hands on the drug.

Officials say Kalydeco is on track for possible FDA approval as soon as next April.

This would not be possible without the Cystic Fibrosis Foundation, which is the non-profit that invested $75 million donation dollars into the drug.

To learn more or to donate, click here.

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