One of the coolest advancements in CF medicine in the last decade are modulators. These are drugs that are a capable of successfully improving CFTR function, which means they correct/potentiale the underlying issue with CF: the defective CFTR mutation.
Modulators are so impressive because they show that it is feasible to hope for medications that are going to be so good at fixing the mutations that patients can reasonably expect to see a significant decrease in their lung function decline.
There are three major modulator therapies approved, all produced by Vertex: Kalydeco, Orkambi, and Symdeko. I won’t get into too much of the basic detail, but Kalydeco is the most effective because it potentiates one of the easier mutations of CF. Orkambi is effective in patients with the most common mutation, but since that mutation is more complex, it is more difficult to fix, and therefore it doesn’t improve CFTR function as well as Kalydeco. Symdeko is a newer generation of Orkambi for a similar group of patients, but it’s shown to be safer for patients, though about equally as effective.
Modulators are the reason it’s okay to be excited about CF medicine. They’re groundbreaking drugs that, incredibly, improve the underlying issue with CF. I’ve only been heavily involved with CF science for about 3 years, but I’m not sure if it was previously believed you could have such a significant improvement in CFTR without doing gene therapy.
I want to be careful of prognosticating, but I do believe the cure to CF will be some sort of gene therapy. With that being said, modulators are something to be very, very excited about.