Cystic fibrosis, a genetic disease that causes the body to create thick and sticky mucus, can now be controlled, thanks to a new breakthrough research.
In an international clinical trial, treatment with two medications, which targeted the most common genetic cause of cystic fibrosis, improved lung function and lowered the rate of pulmonary exacerbations.
The Combined Lumacaftor-Ivacaftor Therapy was shown to be safe and effective for cystic fibrosis patients.
The mucus formed due to this disease leads to chronic infections and increased loss of lung function. In the pancreas, the thick mucus keeps enzymes from reaching the gut to digest food. The median life expectancy is 37 years, and available treatments mostly focus on managing symptoms and consequences of the disease.
McColly, an Associate Director at the Cystic Fibrosis Center at Lurie Children's said, while significant progress has been made with supportive therapies for cystic fibrosis, developing treatments that address the underlying genetic cause still is a challenge.
The research claims that combining Ivacaftor with Lumacaftor helps patients with the most common cystic fibrosis mutation.
The study is published in the New England Journal of Medicine