Vertex...So Close!

So, we all know that there is no cure for Cystic Fibrosis. I have always known this. No cure. It is the worst. Even though this is true, I, my family and friends, have always hoped that one day some one would find a cure; or something close. Since 2006, Vertex Industries has been working on two amazing drugs that seem to me, unbelievable. When I heard that Vertex was working on medications to target the actual cause of Cystic Fibrosis, my heart literally leapt with joy. I remember reading about VX-770 and VX-809 (the two drugs) for the first time earlier this year. Reading about how these drugs would target the defects in the mutations G551D and DeltaF508. Was I reading it right? Yes, I was! These two drugs deal with my two gene mutations!!! As i kept reading about them I started to cry. Tears of joy rolled down my face. I seriously couldn't believe it. I scrambled calling my momma and reading it to her over the phone. Basically, VX-770, which is in the last phases of clinical trials before getting approved by the FDA, helps the CFTR proteins open up and work properly. VX-809, in phase two trials, corrects the placement of the CFTR proteins, so then VX-770 makes them work. Can you even imagine how smart the people have to be to figure out these messed up cells and fix them must be?! Amazing. Cystic Fibrosis is so much more complicated then people describe it to be, which makes it very hard to even imagine that scientists are making such a breakthrough already. I have talked to people about the VX-770 study. Lung function has improved like crazy, like almost 15% higher. That is a big deal, beyond a big. I have read what someone who is in the clinical trial wrote and said they can't believe they feel HEALTHY. Now, a lot of us with CF will say we feel good, because to us coughing everyday is our normal good. To feel healthy I can't even imagine. This woman said that she has even backed off a little bit on doing nebulizers, and her lung function has still improved. Crazy! If I can stay healthy enough, I hope to become involved in these life changing clinical studies. These drugs could change my life. Yes, I know this is not a cure, but hey, I think its getting pretty damn close! Knowing that these target me specifically (my mutations) it could help me have so many more tomorrows. It gives me hope to continually want to try and get even better so that when these get approved, my lungs will again hit PFT numbers that I never thought I would see again. Please continue to believe, have hope, faith, and pray that these drugs will continue in their incredible movings toward getting approved by the FDA; because when they are, it will be THE best day of my life.

To help you understand what it looks like (not my x-rays):

These are "Normal" Lungs

(notice how clear they are)

These are lungs of Cystic Fibrosis

(the cloudiness is scarring)