Saturday, October 22, 2011


I vowed to share our story through each step of this so here I go.  My mind has been in a million places the last few days so I’ll try my best to make this understandable.  First, let’s recap:  Brady is recovering from his recent endoscopic sinus surgery and seems to be breathing much easier.  He is making comments during meals like, “mmmmm, tasty mom” that indicate to me that he is finally smelling and tasting his food.  His appetite in general seems to have increased.  He is not snoring at night and can breathe through his nose during the day.  Awesome.  Want to know what is even more awesome???????

NEWS FLASH! Vertex Pharmaceuticals submits new drug application to the FDA on Oct. 19th for VX-770(Kalydeco—Kuh-LYE-duh-koh)! It is strange to see VX-770 with an officially trademarked name associated with it!   At first I didn’t like the name.  Now, it makes me think that CF is about to collide-aco with my fist.  Dumb I know…but it helps me remember the name.  It is hard to describe how it feels to actually realize a goal like this.  When I learned about VX-770, Brady was an infant and the drug was in Phase 1 trials.  The chances of any compound making out of that first phase are so small.  Years pass and VX-770 progresses through phases 2 and 3 of clinical trials with incredible results.  Today, the FDA considers this drug for marketing approval in the U.S.  When it becomes available…everything will change for us.  Am I dreaming? 

Today I also booked my flight and hotel for the North American Cystic Fibrosis Conference in Anaheim Nov. 3-5th.  I went back and forth over whether or not I should attend…it is super expensive.  A few weeks ago I decided I should just stay home and watch the webcasts.  Then the application went to the FDA and I got the most unnerving feeling in the pit of my stomach.  I felt so sick thinking about this conference going on without me so I instant messaged Brock at work, hoping he would agree to take time off work and let me go.  I feel like I’ll regret it for the rest of my life if I miss it, so I’m going.    

An hour later, I’m registered and my trip is booked.  You’d better believe I’m going to share everything I learn.  I know there are so many other CF parents out there still waiting.  For some reason, Brady has that random genetic mutation that gets to benefit first…I will pay it forward every way I can.  I feel so nervous and excited.  My son’s entire clinic team is also attending and I’m looking forward to “hanging out” with them in a social setting.  I can’t believe I’ll be rubbing elbows with the scientists who developed VX-770, and the President of the CFF, Dr. Bob Beall.  They are like celebrities to me and I’ve got butterflies just thinking about it!  I will also get to meet a few CF parents that I’ve come to know through Facebook.  It is all becoming so real. 

There has been a flood of information online in the last few days regarding the Vertex drugs and details of the clinical trials.  One man wrote that he had achieved a 99% FEV and had stopped most of his other CF medications after being on VX-770 for a while.  This is what I hope for Brady.  I hope he can someday stop his crazy shaky vest and constant expensive airway clearance medicines…and just take Kalydeco and enzymes…and be healthy. 

My primary goals at the conference are to:         
1) Meet and kiss Dr. Beall

2) Learn inside information about the trial for 2-5 yr. olds that Vertex is planning for 2012.  I need to know details for obvious reasons.  My hope is that the trial is set to start at the beginning of 2012!  This is the best, easiest, cheapest, most lawyer free way for Brady to get the drug.  I’m worried that if we try to obtain it by regular prescription, our insurance company will deny coverage.  The drug will be super expensive and has only been tested in children aged 6+.  Brady will only be 4 when it is approved so we expect a fight.  I need to find out the parameters of the trial.  Is it placebo controlled?  I want the real drug for sure.  Brady’s nurse seems to think that it might be designed more like a rollover study where everyone gets the real drug, because efficacy has already been proven.  Also, when exactly do they plan on enrolling and starting the trial for 2-5 yr. olds?  The sooner, the better. 
3) Meet and thank people from my CF community

It feels like something big has happened even though nothing has actually changed.  We are still just sitting here waiting for the little blue pill.  Still, there have been some ch-ch-ch-changes in my mind.  The weight of CF has begun to lift and I’m allowing myself to imagine distant futures.   It is starting to feel like everything might just be OK, so I can relax for a goddam second.  Last night, I slept better than I have in years.  It is just such an amazing flood of relief washing over me.  The profound impact that the Vertex news has had on me has allowed me to really cruise through Brady’s recent surgeries with a positive attitude.  Also, it is tough to be negative when he is so stinking brave about everything.  After both surgeries, the anesthesiologist told us that Brady wanted to hold the mask himself while inhaling the medicine to put him out.  He was a cool, calm, star patient and they were really impressed by him.  He is an unstoppable force.  Just imagine if he had a chance at a regular healthy life…oh wait, he does J  I am so happy, hopeful, and thankful.  Now I just need to figure out what to say to the people responsible for making this happen.  How do you thank someone for a gift like that?  I have less than 2 weeks to figure it out!  So far, all I’ve come up with is blubbering tears. 

I’ve been asked by lots of other CF parents to report what I learn at the Conference and I promise detailed updates!