Health Watch

Cystic fibrosis breakthrough

Wednesday, February 22, 2012

The New England Journal of Medicine calls it a milestone. Patients who've tried it say it's changed their lives. Now, there's a new breakthrough in the treatment of a lung disease that kills patients by their mid-thirties.

Growing up, Angela Riddell loved swimming and softball, but cystic fibrosis made it hard for her to play.

"[I] couldn't run, [I] was constantly coughing and couldn't catch my breath," said, Angela Riddell.

Angela's spent most of her life in and out of the hospital, struggling to survive.

"Rough, it's been real rough," Angela said.

Cystic fibrosis is caused by a genetic abnormality. It results in thick, sticky mucus building up in the lungs and digestive tract leading to life-threatening infections and difficulty maintaining weight.

"Patients are dying right in the prime of their lives," JP Clancy, M.D., a professor and director of CF and clinical and translational research at Cincinnati Children's Hospital Medical Center, explained.

Doctor Clancy believes a new oral drug could change that. VX770 targets one of the genetic mutations that leads to CF and reverses it.

"It's really unprecedented and many people consider it a game changer," Dr. Clancy said.

In a phase 3 clinical trial, adult participants taking the drug gained an average 10.6 % more lung function after 24 weeks and put on seven pounds. Children increased lung capacity by 12.5 % and gained eight pounds.

It has put Angela back in the game. Before the drug, her lung capacity was at 48% and now it's shot up 23 points to 71% and she's also gained 20 pounds.

"I can see my future and it's getting older and seeing grandbabies," Angela said.

Neither kids nor adults in the study experienced side effects that were clearly related to the new medicine. It's important to note the drug only targets one of the genetic defects that leads to CF. The FDA is expected to approve VX770 early this year.

Additional clinical trials are underway targeting some of the other common mutations that cause the condition.

If you would like more information, please contact:
Jim Feuer
Media Relations Manager
Cincinnati Children's
(513) 636-4656
Jim.Feuer@cchmc.org

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