Cystic Fibrosis (CF) is a progressive genetic disease that affects the lungs and digestive system as well as many other organ systems.It causes thick, sticky mucus to build up in the lungs, which leads to life-threatening lung infections. There are approximately 30,000 people with CF in the United States and 70,000 worldwide.
Have there been advances in the treatment of CF?
In short, YES.
Just over 60 years ago, children with CF rarely made it to kindergarten. Today, the average life expectancy for someone with CF is 41 years old.
The most exciting part is that researchers are on the brink of changing the face of CF, transforming it into a disease that people live with instead of die from. In 2012, the FDA approved a life-saving drug called Kalydeco, the first-ever drug of its kind to address the underlying defect in CF rather than just the symptoms. The drug promises to extend lives by decades, but only for 4% of the CF population with one particular genetic mutation. That’s probably why it was named the 2012 Forbes “Drug of the Year.”
Most importantly, Kalydeco serves as proof of concept. In fact, there are promising new therapies in late-stage trials that will extend these advances to the nearly 90% of the CF population who all share the most common CF mutation (df508). Unfortunately, these approved and impending therapeutic advances do not benefit the outlying group of approximately 6% of the CF population with rare mutations.
Without any government funding, research for life-saving therapies for everyone with CF, including the outlying 6%, depends entirely on the generosity of individual and corporate donors.