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New drug keeps cystic fibrosis symptoms at bay

By Ed Yeates | Posted - May 21st, 2013 @ 11:55pm

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SALT LAKE CITY — Cindy Baldwin, 24, sits at her piano these days and plays a different tune. Life's music has never been better.

For the first time, her life-threatening disease appears to have reached a plateau — a miracle plateau, in her definition, because the progressive damage to her lungs from cystic fibrosis (CF) may have ended.

"It's been amazing to see the difference just in the way I feel," she said. "I told my husband that I feel like I've traveled back in time, because I haven't felt this good since I was probably 12 or 13."

Baldwin now takes a drug recently approved by the FDA called Kalydeco. For the first time, it's treating the biochemical pathway of the disease at the level of the genetic defect.

Only two pills a day appear to be holding the disease at bay. For babies diagnosed with CF, the drug might be able to stop the development of any disease at all.

It's been amazing to see the difference just in the way I feel. I told my husband that I feel like I've traveled back in time because I haven't felt this good since I was probably 12 or 13.

–Cindy Baldwin

"As long as they take the medications they won't get these serious diseases, and they'll essentially be able to have normal lives," said Dr. Theodore Liou, a CF specialist and associate professor of medicine at the University of Utah. "We're not going to reverse the lung damage that's already there, but we're going to stop the process from making it any worse."

Even more amazing is a new five-week-old addition to the Baldwin family. Pregnancy usually threatens all sorts of complications for CF patients, like additional stress on the lungs, susceptibility to every cold or infection, and the risk of not carrying a baby full term. However, Baldwin's pregnancy surprised her and her doctors.

"I was really healthy all through the pregnancy," she said. "Every doctor who treated me, all the pulmonary specialists and the OBGYNs — all of them were just amazed. And, I carried my baby full term."

Like most CF patients, Baldwin was skeptical at first. She was given an assembly line of prescribed drugs, but one was different. For her kind of CF, Kalydeco was a life-changer.

"It just kind of blew everybody's expectations out of the park. It was incredible," she said.

The drug has been a miracle, Liou said, and not just for Baldwin. More than 12 other Utahns have been treated with the drug so far.

What is cystic fibrosis?
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 30,000 children and adults in the United States (70,000 worldwide). A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that:
  • clogs the lungs and leads to life-threatening lung infections
  • obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food
More information: Cystic Fibrosis Foundation

"Every single one of the patients who are now taking the medication is reporting these incredible improvements in life," Liou said. "I had one patient describe it as, ‘It feels like I need to cough but there's no resistance whatsoever. There's nothing there anymore to cough.' "

While Kalydeco currently treats only a small portion of CF patients, another medication is now in its final stage of clinical trials and will most likely be used in combination with Kalydeco to go after the other variations of CF.

"That would be the majority of CF patients and would hopefully begin to get me out of a job," Liou said.

Together, the two drugs promise to be a literal breath of fresh air for those with CF. Though Baldwin still does a few breathing treatments on her home machine as a backup to make sure the lungs remain clear, she's doing almost everything she wants but without tiring, running out of air and getting sick.

For the first time, Baldwin and her husband talk about doing normal activities and having more children, which they didn't think was in their future.

"We are having conversations that we've never had about looking much further into the future than we were ever able to do before," she said.

The University of Utah, along with about 100 other medical centers across the country, is currently playing a major role in the clinical trials for these new drugs.


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