Tuesday, January 31, 2012

Some pretty awesome news

Most of my CF friends (okay, probably all of you) already know this by now. In fact, most of you beat me to the punch and have already blogged about it, tweeted it, shared it on facebook and ordered a cake with the news scrawled across the top in creamy frosting. (Or maybe the to-die-for cake I ate at my dad's house tonight in celebration of his birthday is just fresh on my mind.)

Anyway, for those of you not in the know, the good news is that today the FDA approved a new drug for the treatment of cystic fibrosis. This drug (called Kalydeco) is the first of it's kind, targeting the underlying cause of CF rather than just the symptoms. Very simply put, it helps the faulty CF cells function normally. Kalydeco targets a specific gene mutation (G551D) that only about 4% of the CF community has but for those people, this drug will be life-changing. Let me rephrase that: for some of my dearest friends, this drug will be life-changing.

I don't carry the G551D gene (I'm a double DF508, in case you were wondering), but that doesn't put a damper on my excitement. Today is an important milestone for the world of CF! It is a giant step towards finding a drug (or perhaps a combination of drugs) that will benefit every single one of us, and maybe even one day... a cure.

I have to be honest. I've not been very optimistic about seeing a cure in my lifetime. I've never been able to realistically picture myself as an old woman. I've always believed whole-heartedly that it was a cause worth fighting for, but deep down I've never felt that I'd personally see a cure. But then today, hours after first hearing the news about Kalydeco, I was sitting in my car alone and I suddenly started to cry.

I cried thinking of all the people who haven't made it this far; people like my own sister and several of my friends who aren't able to share in my excitement today because for them, this breakthrough didn't happen soon enough. I cried thinking of the short conversation I had with a friend of mine just last night. We were talking about Kalydeco as well as other drugs that would someday follow when I told him, "I promise to hang on if you do." (Who knew it would be approved the very next day?) And I cried because for the first time that I can remember I am not just hopeful, but I really believe.

This may not be a cure, and it may take months or even years before all the kinks are worked out and drugs like this are available to everyone, but this is BIG. And it's happening RIGHT NOW. So tonight lets raise our glasses to those who will benefit from this drug, to those who were taken from this life too soon but remain in our hearts, and to HOPE.

Our future has never looked so bright.



  1. Amen!!! I still feel like my brain can't even process what a HUGE deal this is.

  2. NO way your a double delta too, never would have guessed that one! LOL :) And not surprisingly I agree with everything in this post, including your thoughts on a cure in our lifetime. I though am still a little doubtfully optimistic, does that make sense? Probably not. But anyways Cheers to those it will help I am truly happy for them, and am proud to say my efforts to raise money contribute to these treatments! The cake with yummy frosting sound like a great idea though :)


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