Cystic fibrosis drug FDA-approved after U study

The U’s Cystic Fibrosis Center was one of several clinics to test the drug.

Kali Dingman

A University of Minnesota center played a role in the testing of a cystic fibrosis drug approved by the Food and Drug Administration.

Vertex Pharmaceuticals chose the Minnesota Cystic Fibrosis Center as one of the clinics to test Kalydeco, which could help treat certain types of the disease.

A person with cystic fibrosis, an inherited disease that mainly affects the lungs, is only expected to live into his or her late 30s — a significant improvement from a decade ago when those with the disease rarely lived into adulthood.

But with the new drug, approved by the FDA about a month ago, doctors can treat the underlying problem for a less-common type of the disease.

The drug works in patients who have the G551D version of the disease, which makes up about 4 percent of cystic fibrosis cases.

The Cystic Fibrosis Center, part of the University’s Amplatz Children’s Hospital, is one of the largest centers for the disease in the country and treats 500 patients, said Terri Laguna, assistant professor of pediatric pulmonology.

Instead of fixing the symptoms of the disease, like blockages of airways and declining lung function, the drug helps proteins that form in cells find their way out of the cell.

“The drug is incredibly effective because other medications treat the symptoms of cystic fibrosis while this one defeats the problem,” associate professor of medicine Jordan Dunitz said.

During the study, some patients were given the Kalydeco pill, and others got a placebo. At the end of the study, all the patients were able to receive the Kalydeco treatment.

In order to remain effective, the pill must be taken for life. Most insurance companies are agreeing to cover the $294,000 annual price for the drug, Laguna said.

The drug has been approved for patients ages 6 and older. Next month the University will begin testing it for children ages 2 to 5.

Because Kalydeco was made specifically for one type of cystic fibrosis, Dunitz said it is one of the first drugs to personalize medicine.

Researchers are still looking to create a drug that will help the patients who have the most common type of cystic fibrosis, Delta F508. The drug will treat patients by combining the existing medication with another perscription that helps stabilize proteins in the cells, Dunitz said.

Communications junior Nikolas Job has the most common type of cystic fibrosis and will not be able to take Kalydeco. Even so, he said the drug will benefit everyone with the disease.

“This drug will ultimately benefit everyone with CF because it encourages them to stay healthy so when they do find a cure one day, they will be that much more receptive to it,” Job said.

Job must use a nebulizer three times a day in order to break up the mucus that builds up in his lungs. He also takes a pill every time he eats that helps digest his food. In addition, he said it is important to stay active.

He remains optimistic about the advancements for another drug that will treat his type of cystic fibrosis.

“The medicine for cystic fibrosis is becoming really good,” Job said, “and they are coming up with good medicines that will benefit all people with CF.”