Monday, February 13, 2012

CF Advances and my thoughts on it...

Here's an article about Kalydeco. Press Announcements FDA approves Kalydeco to treat rare form of cystic fibrosis

So it's kinda old news, but I wanted to write about Kalydeco. It's the newest drug on the market for CF, more specifically for CFers that carry the G551D mutation. It is a small portion of the CF population, around 5%, but it is still a huge wonderful step in the right direction for all of us. I am EXTREMELY happy for those it will help, this is what I fight for, fund raise for, and dream of. They are currently working on another drug in connection with this one that will target the most common gene mutation DF508, I carry 2 DF508's. It is in clinic trials right now, and when I spoke to my doctor he said depending on those results it may be up for the FDA to approve in as little as 2 years. It took Kalydeco about 2 1/2 years with trials and the FDA approval, it was the fastest the FDA approved a drug, ever. I'm hopeful for the drug that will help me.

That being said, it's hard to put into words my thoughts on new drugs and my thoughts on a cure being found, but I'm going to try. I grew believing I would die young. Now I don't want this miss con-screwed, my parents never said your gonna die young, but they also were always completely honest with me in an age appropriate way, and instilled in me how important it was for me to do my treatments and take my pills, because yes the predicted age is young, but no statistic holds your future, God does. I for a long time had hope for a cure, and was continually told they were so close, 10 more years, 5 more years, it's coming. I went through getting promising new drugs and treatments such as Pulmozyme, TOBI, and The Vest. I seen results with all these things, but it did not stop my progression. As I got older I came to believe a couple things, I was not going to live to be 30, and there was not going to be a cure in my lifetime. I still believe one of those things, I'm 29 my birthday is in October so I think I will make it another 8 months, God willing. I still believe there is not going to be a cure in my lifetime. I'm optimistically/Doubtful, I hope that there will be for the new generation of CFers, but I'm very doubtful for some of my generation. The new drug has been proven in the short term to improve lung function by about 10%, more for some, and the ability to gain average of 7 pounds, which don't seem like much but is a huge deal for some of us. Let me put that in perspective for what that would do for me, if the one for my gene comes through and I'm in the same health I am now. My lung function is about 37% out of 100% right now, my weight is at 130, I need to be at about 140. So if I was to get the drug it would bring my lung function to about 47% and my weight about 137 (these are estimates). I would still be under weight by 3 pounds, and my lungs would still have permanent damage, and I would still breathe with less then 50% lung function. Now let me tell you I would be grateful for that, I really would, but honestly it's not going to improve my life that much. What it might do is, pro-long my need for a lung transplant, or for better drugs and once again yes I would be grateful for that, just like I am grateful for the other drugs and the vest getting me to where I am now. It's just a never ending race, I am chasing my life expectancy, and quite honestly I get tired of running the race. I would never give up, but not because of me because of Colton, Brad, and the rest of my family, they would kill me themselves if I gave up! This is my outlook for me in the situation I am in right now. I think this is huge for the young children and young adults with CF and it will most likely have a huge impact on their life. In an article I read about kalydeco it said that of the CF population 49% is above the age of 18, that is great but that still leaves 51% children, to me this is still very much a children's disease. More the half are still dying in childhood. The average number of CFers in the United States is 30,000, and that never changes because people die daily from CF and babies are newly diagnosed daily. Both those facts hurt my heart. But that's also why I continue to raise money, because I may have doubt's for my chance to see a cure, but I haven't given up hope for the babies. I just Pray I am proven wrong!

There is a whole other reason why I don't think we will have a cure but I'm not going to go into that one right now. Maybe another day.


  1. I have to admit that your post has one of the most realistic views on the Kalydeco I have read yet! This drug is great news for the CF community, but I think so many people are seeing it for much more than it is. A 10% increase in lung function is amazing, but it is a far cry from a cure. I think it may lead to more discoveries in helping the CF community in the future, but I am not expecting to be cured in my lifetime. Like you, I hope I am proven wrong!

  2. Very interesting. Would like to hear why you think there will not be a cure?