Why Combination Therapies Hold Promise for Cystic Fibrosis

Many with cystic fibrosis are pleased to hear about the progress in the field, and that a new combination therapy may be able to help them.

What is combination therapy?

So, it's great that there is a combination therapy that may be able to help people with cystic fibrosis, right? But, what does that really mean? What is combination therapy?

Sometimes referred to polytherapy, combination therapy utilizes more than just one medication or modality. So, a patient will have multiple therapies to treat a single disease. Typically, these therapies are pharmaceutical.

How does this therapy help CF?

Combination therapy can be extremely useful to those who have cystic fibrosis because using two different medications can often effectively target the genetic cause of the disorder. As a result, the lung function of those who have CF is greatly improved. Not only that, but combination therapy is also capable of lowering the number of lung infections that those with CF will have to suffer through, as well as an array of other problems.

Which drugs are combined?

So, it is important to know that combination therapy is useful - but of course, you have to know which drugs are being combined! The two used for those with cystic fibrosis are ivacaftor and lumacaftor.

The researchers behind this study note that ivacaftor has already been approved by the United States Food and Drug Administration for purposes of treating a select group of people with cystic fibrosis. Dr. Susanna McColley co-author of the study, and she explained in an Ann & Robert H. Lurie Children's Hospital of Chicago (where she is the associate director of the Cystic Fibrosis Center) news release, "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."

Why this is important

Finding out how combination therapy is effective is extremely important because it can greatly improve the lives of those with cystic fibrosis. Because a thick and sticky mucus generates within the lungs, pancreas, and other organs, certain day to day activities can become unbearable, and the genetic disease ends up being overwhelmingly debilitating. The American Lung Association has pointed out that those with CF have a significantly shorter life expectancy than most others. Clearly, as a result, it is crucial to find ways to treat the disease - not only to make life more bearable for those who have it, but to find a way to prolong people's lives, and potentially avoid it altogether in the future.

Learn about the clinical trial for this combination therapy and how it impacted people with cystic fibrosis.