Benefits of treating cystic fibrosis with drugs targeting the CFTR protein

What are the real-life benefits for patients with cystic fibrosis treated with drugs targeting the CFTR protein?

Andreas Freitag: The evolution, or the revolution, of these medications is going to have a profound positive effect for many, if not all, patients with cystic fibrosis.

Just briefly, patients with cystic fibrosis have different genetic mutations, and it is extremely important for us to know that because these medications are designed specifically to deal with the patient’s specific genetic mutations. Knowing what their mutations are is going to be really important because then you can pick these various medications which are designed to fix the problem. There are 2 major categories: one is called “potentiators,” the other one is called “correctors,” and depending on where the problem lies, we can use one or a combination of these medications.

We have already seen a positive effect in the vast majority of patients using these new medications. They might be on 1 medication, or they might be on 2 medications, and now possibly even 3 medications, to potentiate or correct the protein, or the gating problems, or the receptor problems, and that has a positive effect in terms of lung function, less symptoms, overall well-being, and reduction in terms of chest infections. This is going to be good for patients who are just starting out with the disease, that is, the newborns, but it is going to be also potentially helpful and beneficial for patients who are struggling with or dealing with their chronic disease.