On July 2, 2015 Orkambi, the Ivacaftor/Lumacaftor combination pill, was approved by the FDA for cystic fibrosis patients ages 12+. This is a groundbreaking milestone for the cystic fibrosis community because it is the first medication to actually attack the underlying cause of the disease, instead of just treat the symptoms for patients with the double delta F508 gene mutation. I have the double delta F508 mutation, so I was over-the-moon ecstatic when I heard the news. I won't be getting my hands on the actual pill for another few months due to insurance processing, so I have a feeling I will be receiving the best Christmas gift this year.
I've always had high hopes that I'd see a cure for the disease in my lifetime. Orkambi is not a cure for the disease, but it is a giant leap closer. Considering the gene for CF was first discovered in 1989 (one year before I was born), and now, in 2015 a pill was approved to fix the underlying cause, I'd say research and science has come very far in just a short period of time. I have been a first hand witness (and guinea pig) for every medication and therapy discovered to treat cystic fibrosis. I think that is pretty special!
There is a reason we are all brought into this world when we are. Timing is everything. I can attest to that!
To give a deeper insight as to what Orkambi means to me, I will try and rewind to every thought that went through my head after hearing about the pills approval. My first thought was, guaranteed life. Now, I know nothing is guaranteed and life isn't promised, but I felt a new kind of confidence in the way I felt about MY life. I thought about singing. I'm completely aware of how horrific my singing voice is but I really don't care. I love to sing and if I can sing a song without getting short of breath and having to cough, then I'm proud. I thought about my laugh. No more will I have to worry about a good, belly laugh being interrupted by an annoying, productive cough. I saw my future. I will be able to have children and not have to worry about getting too weak to take care of them. The fear of leaving a family behind is gone. I've never really feared anything, except my disease. Lung transplants, oxygen tanks, IV's, feeding tubes... Those fears subsided within seconds - it was euphoric. I thought about dancing, running, the rain, the ocean. It was a tidal wave of emotion. I cried and laughed and cried some more. I called my mom, my stepdad, my friends. I felt light but so heavy. I felt everything.
I still haven't quite wrapped my head around it. It still feels so surreal. I know it won't truly hit me until I actually take the pill for the first time. I am beyond excited to start living without the weight of all those fears. I know it will be magical.