Combo Treatment for Cystic Fibrosis Shows Promise

Researchers say therapy could help 15,000 people in United States alone

By Robert Preidt

Monday, May 18, 2015

SUNDAY, May 17, 2015 (HealthDay News) -- Combining two medications that target the most common genetic cause of cystic fibrosis improved lung function in people with the disease. The combination therapy also reduced the rate of lung infections and other problems, according to a new study.

The two drugs are called ivacaftor and lumacaftor. Ivacaftor is already approved by the U.S. Food and Drug Administration for treating certain people with cystic fibrosis, according to the researchers.

"Just a few years ago, ivacaftor became the only FDA-approved drug for the genetic defect in cystic fibrosis, but it only works for genetic mutations found in a small portion of cystic fibrosis patients. Our study showed that combining ivacaftor with lumacaftor helps patients with the most common cystic fibrosis mutation. This is an exciting step forward," said study co-author Dr. Susanna McColley in a news release from the Ann & Robert H. Lurie Children's Hospital of Chicago. She is the associate director of the hospital's Cystic Fibrosis Center.

Cystic fibrosis is a genetic disease that causes thick, sticky mucus to form in the lungs, pancreas and other organs. According to the American Lung Association, people with the condition have a shorter-than-normal life expectancy.

The current study was a phase 3 international clinical trial. It included more than 1,100 people with cystic fibrosis. They were all 12 or older.

The researchers found that 24 weeks of combined lumacaftor-ivacaftor therapy was safe and effective for people with two copies of a particular cystic fibrosis gene mutation called F508del. This mutation is found in nearly half of the patients with the disease, the authors said.

The study was published online May 17 in the New England Journal of Medicine.

"These groundbreaking findings will benefit around 15,000 patients in U.S. alone," said McColley, who is also professor of pediatrics at Northwestern University Feinberg School of Medicine.

"While significant progress has been made with supportive therapies for cystic fibrosis, developing treatments that address the underlying genetic cause has been a challenge," she explained.

"We will need more analyses and longer-term data to see if this treatment can alter the disease course and further extend the life expectancy of our patients," McColley said.

A U.S. Food and Drug Administration advisory panel met May 12 to consider the results of this study. This panel will recommend whether or not the FDA should approve the combined therapy.

SOURCE: Ann & Robert H. Lurie Children's Hospital of Chicago, news release, May 17, 2015

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