New treatment could improve underlying cause of cystic fibrosis

HOUSTON – The only treatment someone with Cystic fibrosis has ever had is to help with the symptoms until now.

15-year-old Regan Stigall wakes up 3 hours early every morning for treatment with a variety of machines and cocktails of medications used to help with her cystic fibrosis, a genetic disease, that gradually damages lungs to the point of not functioning .

"She has to stand still while she does it, which is a challenge to find the time and to find the hours just fit all of her treatments in," her mother, Gabby Stigall, said.

A drug just approved by the FDA, called Orkambi, could dramatically improve the underlying cause of CF in 50 percent of patients.

Texas Children's doctor, Fadel Ruiz, said patients are already asking for it.

"We are very excited because it's one of the medications that's going to allow us to have a significant impact, we hope, over the process that affects patients with cystic fibrosis," Ruiz said.

"I know they're hesitant to call it a cure, because on an individual basis you don't know how it's going to affect you but it's a huge stride for the community in general," Regan Stigall said.

She speaks for her generation of patients used to treating symptoms and dealing with a fragile life expectancy but never really improving.

"Of course you don't want your child to live with that," Gabby Stigall said. "I remember when she was first diagnosed as just a tiny baby, and them saying that the median age of survival was 32."

But with drugs like Orkambi on the horizon, Regan anticipates a longer, stronger life than generations of CF patients before her.

"It's not applicable. I'll be OK one way or another," she said.

Although there's not yet an exact date on when this drug will hit the market, Texas Children's Hospital says they will begin prescribing it very soon.