New cystic fibrosis treatment changing lives for patients

BURLINGTON, Vt. (WCAX) A new drug therapy is aimed at improving the treatment for people with cystic fibrosis and part of the testing is happening here in Vermont.

Cystic Fibrosis, or CF, is a progressive genetic disorder that causes frequent lung infections and affects how a person breaths. It affects thousands of people in the U.S. and can eventually be deadly.

"For a while, I was in and out of the hospital once, maybe twice a year for a few weeks at a time," said Erin Evans, a Vermont woman who was diagnosed with CF as a baby.

Evans says she had a relatively healthy childhood, even playing sports. It wasn't until after college, a little more than a decade ago, that her symptoms got worse.

Dr. Charlotte Teneback with the University of Vermont Medical Center says treating it is mostly about controlling the symptoms.

"A lot of what we call 'airway clearance,'" said Dr. Teneback. "Using therapies to try to loosen the mucus in the lungs, get rid of the mucus in the lungs, enzymes, and vitamins to maintain nutrition, and often a lot of antibiotics to help control and slow down the chronic infection."

Evans says treatments used to take her three, sometimes four hours, per day. Now, thanks to a new medication called Trikafta, her treatments are down to about two hours and three pills each day. She joined a national clinical trial for the therapy in 2017 at UVMMC, sponsored by Vertex Pharmaceuticals. Trikafta was just approved by the Food and Drug Administration this month.

"What we've seen from the trials is very significant improvement in lung function and also improvements in nutrition, fewer illnesses, fewer hospital stays," said Dr. Teneback, who was the site principal investigator for the trial at UVMMC.

Dr. Teneback says the new therapy will help 90 percent of CF patients. She says the other 10 percent are not forgotten and that research is being done to find a more effective treatment for them as well.

"I could breathe clearly, I wasn't coughing all the time. I've had more energy than I've had in over a decade," said Evans.

She says will never truly know whether she took a placebo or the actual drug, but to her it was an obvious difference. She even played soccer this summer for the first time since she was 17.

"This medication has just changed my life completely and I'm hopeful for the future of CF," said Evans. "I feel like this is a really good step in one day finding a cure."