4. New medication for cystic fibrosis
More than 30,000 people in the U.S. are living with cystic fibrosis. The condition is hereditary and characterized by thick, sticky mucus that clogs airways and traps germs, leading to infections, inflammation and other complications.
CF is caused by a defective cystic fibrosis transmembrane conductance regulator protein. A class of drugs called CFTR modulators can correct the protein’s action, but medications developed before 2019 were only effective in a subset of people with certain mutations.
“This new class of medication as a modulator helps the protein channel correct the misfiling and move it throughout the cell to the membrane where it actually functions. This new class of medications is very exciting and gets to the root cause of what’s causing cystic fibrosis,” Dr. Elliot Dasenbrook, director of the Cleveland Clinic adult cystic fibrosis program, said at the Cleveland Clinic Medical Innovation Summit.
A new combination drug that the FDA approved in October 2019 is designed to provide relief for patients with the most common CF gene mutation, which is estimated to represent 90% of individuals living with the disease.
“This is a huge impact for patients. As a pulmonologist, I monitor a patient’s lung functions very closely, so to have an improvement of 10-14% is going to translate into two things. Down the line, we know that patients are going to live longer, and when the lung function is improving like that, we know they’re going to feel better on a day-to-day basis,” Dasenbrook said.