FDA Approves Cystic Fibrosis Drug - The Wall Street Journal
Kalydeco, Drug That Treats Root Cause Of Cystic Fibrosis, Approved By FDA
This news is big. HUGE in fact. This is the first time that there has been a drug to tackle the root cause of Cystic Fibrosis and not just a symptom. People throw around the word CURE left and right, and this my friends is the closest thing to a cure that I believe we will see.
More than 1,000 mutations in the CFTR gene have been identified in people with CF. I have the classic and most common ΔF508, and one other mutation. This drug, which was approved by the FDA today is for only one of these mutations, G551D, as far as I understand. While G551D is NOT the most common, it causes CF in about 4% of the CF population, some 1,200 people in the United States. I have heard of other CF friends (G551D's) who were participants in the trial phases of Kalydeco, and who claimed it was immensely helpful.
Just to clarify, since this is my blog, this drug will probably not affect me. Not only am I the wrong mutation, but I am post transplant and so my health worries lie elsewhere, such as infection/rejection. Even if they were to develop a drug for ΔF508's, in a way it is too late in my own life. At most I could imagine it helping perhaps with my sinuses and G.I.
But that is not what I want to talk about.
I want to talk about my childhood prayers. My wishes on every birthday cake. My wish for every dandelion I blew into the wind.
A cure for CF.
And although I am fully aware that this is not exactly a cure for all CFers, I now have hope for that dream. For those babies being born today with the gene mutation of G551D, it is coming true.
This might snowball into other drugs being developed for other CFTR mutations.
If just one child can be spared the suffering and pain I have had to endure because of Cystic Fibrosis, than my wish has come true.
1,200 people being CURED. What an awesome, amazing, exciting day in the CF community.
And ΔF508 is next guys, just wait and see. Correcting G551D shows that it is possible.
We're coming up!
If anybody reading this has ever donated money to the Cystic Fibrosis Foundation I want to tell you, as I hold back the tears - THANK YOU. It certainly has helped a hell of a lot during my lifetime!
Thank you God, from whom all blessings flow ...