The Results Are In

I began my day with some of the most wonderful news today.

I was doing my treatments, checking my phone for anything new, as I usually do. I was almost caught up on my Twitter feed when I saw this Tweet:

On here, I have mentioned that they are making great advances in medicines for treating CF at the genetic level; fixing the defective protein, the cause of CF within the gene. I mean how amazing is that? 

So today they announced that the results of a clinical trial are in. This trial studied people with two copies of a certain gene mutation and found that the drug showed "significant improvement in lung function" and other symptoms (like weight gain) and therefore, with these results, the drug company will be submitting a New Drug Application to the FDA by the end of this year, with hopes of drug approval in 2015!

I had tears of joy. See, I don't have the exact gene combo for this trial or this drug as it is right now, but I wholeheartedly believe that very soon (as in 3-6 years) it will be available and proven useful for someone like me. We are SO CLOSE to getting the closest thing I can to a cure! So close! If I get to start taking this drug, it won't completely remove all of my lung problems, they have suffered some damage and scarring that is irreversible. But to know that someday, there's even a chance that my disease could be genetically "fixed", and life could actually improve a little? That's... Amazing. 

There is another clinical trial in the works right now (Phase 2) that is studying use and effectiveness of this med in people with 1 or 2 copies of the gene mutation, and that's the one I'm anxious to hear about. 

But this is fantastic, blessed, stupendous, wonderful news that could be life-changing for half of the CF population. I'm elated for so many CFers who will have their lives changed by this. 

There are a couple other drugs being developed that have me almost as excited too. One of them is an inhaled version of an antibiotic I take when I'm in the hospital. If it is ever approved, it could reduce my hospital stays and significantly help me too. All great news, all the more reasons to keep donating to the CF Foundation because they put almost all of their funding into drug development. 

In other news, I'm still kicking my CF's ass at the moment :). I started doing more at the gym and I'm approaching 7 months out of the hospital, AND without antibiotics! The last time that happened was when I was, like, 10? 

If you'd like to read more about the cilinical trial, go to cff.org and it's on their home page.