Kalydeco is a little like the first effective antibiotic. It is the ultimate gateway drug to a new family of drugs, genetic drugs. Vertex isn't resting on its heels, before Kalydeco saw human trials, other CF mutations were being researched and still are. The reason the third most common CFTR mutation was chosen as a target drug has a lot more to do with advancing the first true genetic drug through to human trials. As dumbfounding as this accomplishment is, G551D was about the easiest mutation to do.
Just the idea of replacing aspartic acid with guanine in human DNA sounds like science fiction. I have a fair understanding of what's going on and I marvel at the fact it is finally here. Swapping out nucleic acids is just a step to repairing deletions and so forth. As a CFTR moderator Kalydeco has the numbers as you do.
My prediction is that Vertex and others are going to be offering genetic drugs that will arrest the progress of most CF within three years. It is a matrix, Sam is young and Kalydeco has a certain trade off being 30% or so improving. I would take that improvement in a flash. Use caution by monitoring Sam's kidney and liver functions as everybody should with a fast tracked drug or protocol.
I guess my logic would be this; kalydeco will improve Sam's lungs and GI tract not to mention his head and urogenital areas. Sam is enjoying a certain level of health that can be bettered by a third, if Kalydeco can reduce the mucus factor by this much, maybe it's enough to eliminate all the infections that go with it. From what I have read, many people are seeing considerably fewer GI issues or they are less severe.
Delaying damage until the perfect genetic drug comes along sounds worth doing, the improvement in Sam's life in the meantime would pay double.
LL
