I can remember distinctly, I was sitting in the sunroom of my house around my sophomore year of high school when I first read about Vertex Pharmaceutical's work with a drug for CF that would treat the underlying cause. I remember crying, thinking, finally, someone is undertaking this feat. It had been 13 years since I was diagnosed, and my parents were told there would probably be a cure in 10, given that I was born around the same time work with the CF gene was being discovered. Some cautious, reasonable person was talking about how long it takes for drugs to move through the pipeline... they were saying something like "It will probably take over 5 or 6 years for us to see any results from this" So in my head, I calculated... I'll be a senior in college.... at 16 that seemed like hundreds of years away, it seemed like the day would never come. It seemed like, in between that time, I would only get sicker, I was already averaging 2 visits a year in the hospital.
Well, now I'm a Junior in college, healthier than ever, and Vertex is applying for Priority Review and approval by the FDA for their first drug researched to treat the underlying cause in a rare 4% of people who have the G551D mutation. Thats about 1,200 people whose lives have the potential to be changed by this drug! And from what it seems like based on trials that are being sped along by Vertex for vx-809, a drug that treats the underlying cause of those with Delta F-508 (me along with half of those with CF and another 40% who have one of the mutations), similar news is probably just around the corner. Last time I was at the doctors, the researcher there was telling me how they are expanding the size of their phase 2 trial to get more data which means more significant results if improvement is shown. I could just see the anticipation and excitement in his voice, his eyes, and his smile!
Hearing stories about people who have been on vx-770 (the one that is currently seeking approval) is overwhelming. Some people report leaps and bounds in PFTs, sweat chloride levels coming down, and even a great reduction in coughing (now this is all hearsay, but STILL!). It's almost surreal. Imagine living with something then all the sudden this pill is available, and it makes all your symptoms so much less. Every time I hear some new news, I choke back tears.
I don't know much about Vertex Pharmaceuticals, but I know they are a company that is willing to invest in something that, by worldwide numbers, not many people struggle with; but that many, many are affected by whether it be parents, friends, relatives, or doctors and nurses; and not to mention the immense hardships it causes to those who live with it every day. If nothing else comes out of all of this, at least there is the assurance that there are some good people in our healthcare system, willing to explore and research every option to try to make the quality of our lives better, Vertex has proven to be just those type of people, and I am so grateful for that.
