A new study showed that the drug ivacaftor significantly improved lung function in some patients with cystic fibrosis, a life threatening hereditary chronic lung disease that causes thick, sticky mucus to build up in the lungs, digestive track, and other areas in the body.
The phase 3 clinical trial study was led by Bonnie W. Ramsey, MD of Seattle Children’s Research Institute and the University of Washington.
Lung function improvement
Researchers found that approximately 4 percent of patients with the gene mutation called G551D that causes cystic fibrosis (CF), who were treated with ivacaftor, also known as VX-770, showed a 17 percent relative improvement in lung function that was sustained over the course of 48 weeks.
Researchers evaluated lung function in patients 12 years or older who carry at least one copy of the G551D mutation. The third and final study in a series designed to assess VX-770’s effectiveness and safety before approval, included 161 patients at multiple healthcare centers who received at least one dose of VX-770 or placebo.
Patients with G551D treated with VX-770 also showed improvements in other areas critically important to the health of patients with CF.
Reduction in Sweat Chloride Levels
Cystic fibrosis patients who participated in the study showed significant reductions in sweat chloride levels indicating an improvement in the body’s ability to carry salt in and out of cells, a process that causes CF if defected.
Improved Weight Gain
Patients involved in the study also experienced decreased respiratory distress symptoms and improved weight gain, which is good for CF patients have difficulty gaining weight, especially during childhood as they have reduced lung function and chronic infection.
Those who received VX-770 gained on average 7 pounds compared to those in the placebo group who gained approximately one pound, the authors wrote.
Impact of Cystic Fibrosis
Approximately 30,000 children and adults in the U.S and 70,000 people worldwide have cystic fibrosis, caused by a defective gene that affects many parts of the body, especially harmful to the lungs and pancreas. In the late 1950s few children with CF lived beyond the age of five and today with advances in research and medical treatments made it possible for patients with CF to live well into their 30s, 40s, and sometimes beyond, but the disease is still life threatening and researchers have been trying to find new ways to improve lung function in patients with the disease. VX-770 might just be the next step to an even better form of medication to improve lung function in CF patients.
"Our study shows that we are now able to improve the quality of life for cystic fibrosis patients with the G551D mutation with the administration of VX-770," said Dr. Ramsey.
Published by Medicaldaily.com