Much Anticipated!

 Goosebumps.

Chills.

Estatic.

HOLY CRAP.

These are all the words I thought as I read the latest from Vertex on the Phase III clinical trials starting for VX809.  Our drug.  The version of Kalydeco (the blue "miracle" CF pill I blogged about in November).

Here's the article released Monday...
http://www.thestreet.com/story/11853335/1/vertex-readies-late-stage-cystic-fibrosis-drug-studies.html

Why do I love this?  Of course there are several reasons.  But one of the most important (other than the fact that this drug will add DECADES to our kids' lifespans) is it's what I promise people when I ask for money every year.  That their dollar matters and is changing the face of CF.  I love reading back to my letter campaigns or talks from the year previous.  Everything I had said would happen, did.  And then some.

But this time, the stats changed.  For the "sooner. "

VX809 wasn't set to be FDA approved until 2015.  Now, if all goes as planned it could be 2014.  And to top it off, Vertex has been given approval to shorten the study from the typical 12 months to 6 months.  I'm salivating...

And if you needed more to go crazy excited about it, it would be this: The study is being opened to include children ages 6-11!!!!

I haven't heard yet if our CF clinic in Madison will be conducting the study for children or not, I am certainly going to push for Cayden to be in the study if they are!

This is such a big deal and a great move forward.  It won't "cure" CF.  The kids will still have to do their treatments, take their enzymes, but the basic defect of CF will be fixed.  On Kalydeco and VX809, patients with the Double DeltaF508 mutations test negative on the sweat chloride test, have increased PFTs and weight gain!  It's like a negative screen for CF...it is simply amazing and unheard of.

I am so ready for this year.  CF-bring it!