Doctors at KU Health System help test new break-through cystic fibrosis drug treatment

This story has been updated to reflect that KUMC was one of multiple sites that participated in the study and that, although doctors are hopeful that the new treatment will be approved by insurance providers, it is not a given across providers.

KANSAS CITY, Kan. — Doctors at the University of Kansas Health System are participating in a wold-wide test run for a new treatment, calling the latest discovery a game changer.

“I’ve seen a lot of patients die of this disease. I’ve been doing it for a long time,” Chief Medical Officer at the University of Kansas Health System, Steven Stites M.D. said.

“I’ve never seen anything like this.”

KUMC was one of 115 sites that participated in the study that led to a new drug treatment for people living with cystic fibrosis. The treatment was discovered in part by the Cystic Fibrosis Foundation.

Cystic fibrosis is a genetic disorder that leads to a thick build up of mucus in the lungs, digestive tract and other parts of the body. This treatment is a combination of three drugs, and it’s for patients with the most common genetic mutation. The drugs are supposed to help clear out breathing airways.

For Sarah Carollo, it’s working. She’s part of a trial group who’s been taking the drug since last summer.

“More than anything, I noticed that I could walk as fast as others,” Carollo said. “I noticed that I could pick up and move things at my work. Just an overall quality of life increase is just remarkable.”

With her new-found strength, Carollo gets to check a few things off her bucket list.

“I participated in a 5k since then, something I’ve never done before,” Carollo said.

Walking, running, picking things up with ease; these are things most people take for granted. But for those, like Carollo, living with cystic fibrosis, it means freedom to live.

CF used to take the lives of teens and young adults. Now because of these new drug therapies, that isn’t the case anymore.

“There are more people living with CF as adults than there are children. That’s because the improved survival overall,” University of Kansas Health System Pulmonologist Deepika Polineni said.

For Carollo and doctors alike, this medical breakthrough feels like a miracle. On Thursday, the treatment’s creators presented their findings to the New England Journal of Medicine.

Doctors say this treatment will be effective for about 90 percent of people living with cystic fibrosis.

It is available by prescription. The cost? $300,000 a year.

Doctors  are hopeful that the new treatment will be approved by insurance providers based on several patients who have had it approved and payments for similar drugs.

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