Wednesday, February 1, 2012

I woke up with a headache

This morning I woke up with my head throbbing. Spending a day on an emotional high has taken it’s toll and today I am just plain exhausted! Between the crying, the laughing and the wine I had at our celebratory dinner, I could have slept a full 12 hours.

I want to take a second and answer some questions that have been asked of me lately.

First, even when Gavin gets on the medication he will continue his treatment/medication regimen. The pill (aka blue lightening!) does not fix pancreatic insufficiency, so he will continue to take pancreatic enzymes with his food so he can absorb it’s nutrients. Until there is documented proof that the Vest and inhaled medications are not needed, Gavin will continue to do two sessions a day. Honestly, if that were to happen it would be years in the future – if ever. BUT, I have no problem continuing on with them as long as his disease is controlled, whatever it takes to keep him healthy and alive!

Second, yes the fight to get this pill covered by insurance is going to be an uphill battle. But I struggle with everyone’s immediate freak out about it. HAVE FAITH IN THE CFF AND VERTEX! They are on our side and they are already working hard on leading the way in the fight. It has been a mere 24 hours or so since the announcement, decisions and a plan are going to take a bit of time to get into action. So before everyone thinks they need to get “lawyered up” and panicked, let’s take a minute to breathe and see what they are getting set up for us. And for those of you looking at the combo of 770/809 – remember the fight we are going to fight now against insurance companies will only make your battle easier! How many times do we need to be told that EVERYONE who qualifies will get the drug before the panic stops? Where there’s a will there’s a way, and there are too many CF parents out there, and CF patients out there, that know how to fight and to demand a drug for this not to happen. Doesn’t mean it will be an easy path, but it WILL happen.

Third, I have been hearing rumors on what the pediatric study on 770 will entail. Some say there will be a placebo and some say there will not be. Some are also mentioning that it will include a day of being hooked up to an IV and getting blood drawn several times. I will not jump to any conclusions until I see the paperwork describing the exact process but it is BEYOND discouraging to hear parents say, “oh I’m not going to put my child through that…it’s too much…no way, we’ll just fight to get on the drug immediately…” SERIOUSLY? Do you think that this pill just appeared? Do you not realize that THOUSANDS of research studies have happened to get us to this stage? That people have been poked, prodded and studied for decades to HELP YOUR CHILD OUT? That generations of CF patients went through studies knowing full well it wouldn’t help them – but that it would help children in the future? YOUR child is that child of the future. And now, you choose to be selfish? “Why thank you everyone else who went through studies, I’d like to just skip that and take the drug thank you very much.” Explaining to my (almost) three year old why he needs to go through this is NOT going to be fun obviously, but a day or two of testing and a possible placebo for a few weeks before getting the real thing (or possibly getting the pill right away) is NOTHING compared to the work that so many have done ahead of ours. OUR CHILDREN WOULD NOT BE LIVING THE LIVES THEY ARE WITHOUT THE RESEARCH THAT HAS HAPPENED AND IS CURRENTLY HAPPENING. So yes, dealing with a lot emotionally and physically for my three year old will be tough in the moment – but the long term payoff is priceless. And someday he will be able to say that he was a part of this groundbreaking history, that he is a major component in the progress of controlling/curing Cystic Fibrosis. Obviously I would love to run to the pharmacy right now and hand Gavin the pill today – but welcome to reality people, such a breakthrough deserves a little extra work. And any research found with this study will also have a HUGE impact on the studies happening for the 770/809 studies that will help the other NINETY SIX PERCENT of the CF population. So yes, it’s  your decision as a parent to enroll a child or not into a study – but choosing to avoid this study that will help out everyone – not just the 4% dealing with this disease (especially when it has already been proven to have no major side effects) is in my opinion, selfish. There are so many families out there wishing they could partake. Our family will do whatever it takes to get a cure/control for our child but we will NEVER stop fighting, helping, researching, fundraising etc. until this is possible for every single individual faced with this disease. Fighting this disease is a balance between being selfish and selfless, and as the adults we need to teach our children that their hard work has a priceless payoff.

(and I’ll step off my soapbox now…)

And no, Gavin is not guaranteed to be a part of this study – but for his entire life we have lived in the positive (no maybes, no ifs, but WHENs) and I refuse to think any other way when it comes to him participating. We’re definitely glass half full kind of people – life is too short to be pessimistic, especially when we’re fighting for LIFE!

Fourth, like I mentioned before – even with Gavin being one of the few that qualifies for the drug we will never stop in our fight for awareness, funds, and a cure. We will continue to fundraise. We will continue to get our story out there. We will continue on our quest to get anyone willing to listen more educated on the disease and what it entails. Gavin’s other mutation is df508 and the 770/809 combo being studied is just as important to us as 770/Kalydeco. I have too much love for all Gavin’s CF buddies to stop here. And if you ever have a question on anything CF, the drugs – what it means – why he does his treatments etc – please ask me anything, there is no such thing as a dumb question. If I don’t have the answer I will get you the answer we both need.

It’s my blog, it’s my opinion – and if you’d like to go off on your own tangent please use your own blog/facebook status – any negative comments posted will be immediately removed by the author…ME! :)


  1. I'm sure that by the time Gavin is 5-6 and the F508 geneotype has their version on the market with over 60% saturation, the price will be lowered for everyone. They just need to make up for so many years of R&D and trials.

    This is going to be a good year for CF.

  2. I confused, what are people "lawyering up" for? This is supposed to be exciting news!

    I can't see what could be wrong with wanting to participate in the research study. My husband is on the waiting list for the study for his mutation. If he gets in, it means he gets to start taking the med sooner AND help get it approved for everyone else!

  3. I have heard from people who were in the 770 study that their pancreatic insufficiency was reversed by this drug. They said as long at there wasn't scarring and the pathways in the pancreas were just blocked, the drug worked to reverse the blockage and they no longer needed the enzymes. Either way, I think it's great, but wouldn't it be awesome to be able to get off the enzymes too!

  4. Great job! Years and years of health to all our CF warriors...I can't wait to get Aidan in the Delta study! A little EMLA cream goes a long way for the blood draws, we use it all the time! Megan

  5. Congratulations for a brilliant post, the best I have read yet - I love your attitude and outlook! And of course, awesome that your little man can benefit! This approval is so exciting for all with CF and that is something to celebrate! My little Tom is DDF508 and I look at this development as the first step in his future! Big cheers to you and your family - wise words! Natalie x

  6. Alright,so this is the first time I've ever posted on a blog...not even sure I'm doing it right but... I just had to say how much I AGREE WITH YOU!!! I am a mother of a 2 1/2 year old boy with CF and I have read many blog posts, Facebook posts, etc. ALL with negative comments and "lawyer" talk. What happened to the huge sigh of relief, the overwhelming feeling of happiness, and the hope! I personally bawled by eyes out when I heard the news! Such joy! Our family will not benefit from this drug...right now. But it is paving the way for us in the future. We are grateful and will stop at nothing to see that everyone has their blue lightning! This includes, studies, research, fundraising and education. Thank you for sharing your opinion and helping one momma realize I am not in the twilight zone!

  7. I agree totally. It has been so emotional, but so thrilling. Great gratitude to so many, great hopes as well.