Possible breakthrough in cystic fibrosis treatment

Global Business

Jenny McGlincy spends a portion of her days inhaling medicine through a nebulizer while a vibrating vest shakes loose thick mucus from her airways. It’s a common treatment for cystic fibrosis patients. Hendrik Sybrandy reports.

“I don’t know anything different,” McGlincy said. “I was taught that it’s just another thing I have to do every day. I have to take pills with my food, I have to inhale medicine.”

CGTN’s Hendrik Sybrandy reports.

But the Centennial, Colorado woman’s life may be about to change. The disease she was born with, that 80,000 other people in the world share, could be tamed by a new triple-drug combination called Trikafta, which, for the first time, attacks the genetic mutation that causes the often fatal disorder.

It was approved by the U.S. Food and Drug Administration in October and is currently under review in Europe.

“I mean it’s really a transformative therapy that’s going to change what it means to have CF going forward from the day of the approval,” said Dr. Jennifer Taylor-Cousar, a pulmonologist at National Jewish Health in Denver.

Clinical trials of Trikafta were conducted at that hospital and others. Taylor-Cousar, who helped lead the Denver study, said improvements in lung function came almost immediately.

“Within a few days people felt so much better, they had better energy, they were coughing less, they were able to clear the mucus in their lungs out much better than they ever had been before,” she said.

“It’s turned my life around significantly,” said Hannah Wiberg of Boulder, Colorado who has been on the new drug therapy for more than a month now. She used to rely on oxygen 60 percent of the time to combat shortness of breath, but no longer.

“That’s a huge difference,” Wiberg said. “Just ease of life, like grocery shopping is now not a chore… I feel like I can participate in life again.”

Trikafta is quite expensive but at least some insurance companies are expected to cover it. Patients younger than 12 years of age are not currently eligible for it but that may change. 90 percent of patients are projected to benefit from Trikafta which could turn cystic fibrosis into a chronic, manageable condition.

“So it’s definitely not a cure but we do believe it’s going to completely slow down the progression of CF,” Taylor-Cousar said.

“It’s a game-changer,” McGincy said. She looks forward to fewer hospitalizations, better lung capacity, even a bit of weight gain. Most importantly, life expectancy for patients, only in the 40’s until now, should increase with this new discovery.

“That means more years with my husband, with my daughter and my friends, that’s unbelievable,” she said. “That’s what it’s all about. We’ve been waiting for so long.”

She’ll be starting the new drug therapy soon.

“It’s hope,” she added.