I always love going to the doctor (weird right?). Okay, maybe not always. But lately, after each visit, I walk away with more hope than the last. This particular visit happened to fall within a week of vertex announcing news of FDA approval for Kalydeco, an exciting new drug to treat the root cause of CF (yet only functioning for 4 percent of the CF population with a rare mutation). Regardless, this drug marks a new revolution in the treatment of CF, and there is so much hope that the drugs in clinical trials now aimed at targeting the most common mutation (double Df508 the one I have) will offer just as much promise as this one did (even if it takes a few more years to get it down pat)! *commence tears of joy*

So at my appointment today I got to hear some exciting stuff :

1) My doctor, who is the smartest man I’ve ever met and who is so incredible at providing CF care, is FAMOUS. What I didn’t realize is that he was interviewed on FOX news! Of course, I had to give him a hard time about his fame, but if anyone were to give the news, I would want it to be him. He is so intelligent and so committed to this cause it is unbelievable. Watch the video HERE.

FDA approves new drug to fight Cystic Fibrosis: MyFoxBOSTON.com

2. I learned that the drug was not expected to be approved until at least the spring. This caught a lot of the people at my clinic off guard, but it is good news none the less. This means that there is hope for these new CF drugs to speed through approval, getting it out there ASAP. The FDA is showing they are all in for a drug that could change the lives of CF.

3. I was offered hope to be a part of the studies that may lead to more drugs like kalydeco. Though it is difficult to get me involved in a study due to my bacteria (and the resources it requires to keep me isolated), it seems as if my doctor is pushing to get this drug studied in people with cepacia (the bacteria that is colonized in my lungs). We talked about how by next year, they will hopefully be in phase III trials (aka the last one). What this means is, if they are able to accommodate me, I will be able to participate in a study for probably a year. If I don’t get the placebo, I may be one of the first to feel the long-term effects of a breakthrough medication…. next year. WHOA. [keep in mind this is tentative and is by no means possible… YET]

4. My doctor gave me some awesome advice. He was talking about how kalydeco will probably not reverse existing lung damage, therefore, any new drugs probably won’t magically heal all the damage that CF has already caused. However, he told me how important it is now, while we wait for news of new medications, to take care of myself. If I can keep my lungs as healthy as they are now, hopefully some day, if there is a new drug to treat my mutation, the progression of my disease will slow down. Ergo, all the hard work of building up my lungs may pay off, meaning they will remain this healthy for years to come. again.. WHOA. To think that maybe, this could be where it stops. This advice gives me more motivation to remain compliant and keep doing what I’m doing! And will hopefully inspire others to do the same. It also got me really excited about the next generation of CFers… these drugs could stop the damage before it starts… suddenly CF will be a minor nuisance and not a lifetime of sickness.

5. The DJ in the sky (aka Nana… who plays the perfect songs just when our family needs them) played me an awesome song on the drive home. I was getting antsy, as it was hitting me that my life could be extended with all this new technology. I was reminded that this is the beginning of so many doors opening. Especially after the past week where I was feeling that I need to make more of my short life, getting stressed about opportunities I have yet to take, experiences I have yet to experience!  This put me back in check, making me realize that there is still time left. And this song played:

The line that hit me was Ain’t in no hurry. I’d Be a fool now to worry about all those things I can’t change. And the time that I borrow can wait till tomorrow. Cause I ain’t in no hurry today.” The only way I can describe the feeling is what its like to get an extension on a paper after you’ve been stressing about not having time to finish it. Instead, you can go to sleep, get your rest, and know you’ll have tomorrow to get it done. That’s not to say that I am going to procrastinate living because of the news that may lie ahead, but it is to say that I don’t have to stress so much about not having enough time. And that’s an amazing feeling.


It goes without saying that today was fantastic. It was just what I needed to strengthen my hopes and make me excited for my future.  Hopefully, in the years to come, these appointments will mean more and more hope and more and more excitement. But, like the zac brown band says, I ain’t in no hurry. In the mean time, I can’t control what will come, but I can only have faith that the CF foundation (with help from donations from readers like you!), doctors like mine, and companies like vertex will push for something that will change the future of CF. 

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