Have you heard the news??
Now you have :)
Every time I read this article, I want to jump up and down. Phase 2 of the VX-809 and Kalydeco study was successful in patients with deltaF508 mutation, the most common mutation that causes Cystic Fibrosis! (Actually, roughly 90% of CF patients have at least one copy of deltaF508.) In case you didn't know, studies testing new medicines go through three major phases. Phase 1 often screens for the safety of the medication and is usually tested in a small group. Phase 2 is a bit larger and tests the effectiveness and safety of the drug. Phase 3 is the final stage before approval from the FDA. In a Phase 3 trial, larger groups of people participate in it to affirm the drugs effectiveness, decide on common side effects, and figure out the best way to provide the drug to the public market. If a drug makes it through all three phases, it is sent to the FDA for the official stamp of approval. This can be a rather long process, often taking months or even years from the time the phase 3 trial is complete to when the drug is released to the pharmacies.
However, I am still thrilled with the results from this study. I have one copy of a deltaF508 mutation and one more rare mutation. Therefore, I could potentially be part of the Phase 3 study! It would mean so much to me to be part of this study. For starters, I would make a huge difference in the CF community. As well, I personally could receive huge benefits from this medicine. I could gain weight, increase my lung function, and maybe, just maybe, know what it feels like to inhale deeply! I'm going to try my best to get into a study somewhere, even if it means driving all over the country.
We live in exciting times in the CF community, my friends. I can't wait to see what God has in store for the science community in the near future!