Good news may be on the horizon for the tens of thousands of people affected by cystic fibrosis. Drug manufacturer Vertex Pharmaceuticals announced that it may have developed a drug combo that can significantly improve lung function for patients suffering from the disease.
Cystic fibrosis affects nearly 70,000 people in the U.S. It is a genetic disorder that causes thick, sticky mucus to build up in the lungs, digestive tract, and other areas of the body, making it very difficult for sufferers to breathe. It is one of the most common chronic lung diseases in children and young adults — and the most deadly. Cystic fibrosis kills most sufferers in their 20s.
But a new drug combination created by Vertex may offer some relief, improving the quality and quantity of life for cystic fibrosis patients. According to the company's phase-two study, the combination of Vertex's Kalydeco, a medication approved in January, and the experimental VX-809 drug improved lung function by at least 5 percentage points in 46 percent of patients. About 30 percent of patients saw improvement of 10 percentage points or more. Lung function is the most critical marker indicating the progression of the disease for sufferers.
Cystic fibrosis develops as a result of a genetic mutation. Millions of Americans carry the defective gene but do not have any symptoms, because a person must inherit the mutation from both parents to have the disease. The patients who participated in the study had two copies of the most-common cystic fibrosis-related gene mutation, called F508del.