A Big Thank You

A week has gone by and saying that Kalydeco has been approved is no longer an overwhelming shock to my system, we’ve absorbed the news and are trying to learn what the next steps will be for our family.

But for all of you that have done research trials in the past that have gotten us to this point, and for all those who are still waiting for their own miracle drug – I just want to emphasize that we have not forgotten any of you. We will never let Gavin feel like he is entitled to this drug. He will forever know the hard work of countless doctors, scientists and CF patients before him. He will know from this early age that nothing like this comes about without hard work and dedication. We are doing what we can to get him into the pediatric study to thank those who have participated in the past, and our way of paying it forward to those who are patiently waiting their turn.

To clarify – we are not sitting back on our heels waiting to get on this drug. Just ask Gavin’s pulmonologist how many emails I’ve been sending her for months – constantly asking if there is any new news on the upcoming study. BUT, we are doing what is ethically and morally right. We are going to work WITH the CFF and WITH Vertex. We are going to provide as much data possible. We are going to make sure Gavin is closely monitored throughout each stage of the study – and not just his physical status, but his mental and spiritual status as well. At the first sign of danger we’d pull him out – but we believe that tossing our kid a pill that has never been studied in a child his AGE (not his size, his age) is irresponsible. Every drug has a different effect on everyone, we’re going to march forward as veraciously as possible while simultaneously being cautious. We want this drug to be available to everyone – and Gavin needs to learn now that research is what gets us there.

So THANK YOU. Thank you to the scientist’s at Vertex – to all of the scientist’s studying CF. Thank you to the CFF and every CF charity. Thank you to all of the doctor’s who spend their lives dedicating themselves to this disease. Thank you to those who have EVER done a research study at any level – it took a piece of all of those to even come up with the concept of this pill. Thank you to every person with CF, or parent of a CFer who whole heartedly told me how happy they were for our family. Thank you for feeling like you could also explain to me how hard it is to know that you or your child don’t qualify for it, and the mixed emotions that are there – it makes me feel pretty darn special to know that you can open up to me like that.

And THANK YOU to every single person who has ever donated even a penny to the CFF, or any CF related charity. I am proud to be a part of a community who believes that your donated money needs to be going to RESEARCH – and Kalydeco is tangible proof that your money is going where it belongs. People often ask me why they don’t see commercials on tv advertising events, or why there isn’t apparel lined up in the stores – because we believe that the money you give us isn’t meant to go there – the money you give is to save lives. You will most likely hear about CF events through word of mouth, or the rare advertisement because we put the money where it belongs. Beyond grateful that Gavin - and Casey and I - are in the midst of such an amazing community.

We are acutely aware how lucky we are with Gavin and his health thus far. Knowing that many have passed away, or have been too sick to benefit from this drug is a thought always in the back of our minds. I too wish this drug was possible many years ago, but at the same time feel very blessed that it’s happening right now. No CF patient is more important than another, but an adult whose disease has severely progressed is in the need to be on this drug immediately. We are lucky to be in a stage where yes, we want him on it as soon as possible, but that waiting a month or so to figure out what path we’ll be headed down does not mean immediate life or death for Gavin as it does for many others. We’re attempting to quickly move forward as ethically and as safely as possible.

Thank you.

Thank you. Thank you. Thank you.

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! :)

Oh. My. God. It’s. Happening.

The excitement of today ranks up there with the excitement of our engagement…our wedding…the birth of our son…

KALYDECO WAS APPROVED BY THE FDA!

I’m pretty sure my daycare kids think I’ve completely lost it. I’ve been shaking, crying and on the phone all morning. I will never, EVER forget January 31st, 2012!

We were not supposed to get news from the FDA until April, so while we would have been excited then – getting the news this early brings my joy to an entirely new level.

My son is getting the chance to live a NORMAL life. For the first time ever -  a drug is correcting the underlying issues caused by the malfunctioning gene. I feel like I’m watching a Science Fiction movie – someone please pinch me, is this real life. Is this REALLY HAPPENING??!?!?!!?!?!?!

Yes, we will still hopefully be participating in the trial of this drug for kids ages 2-5 later this spring. We know that our clinic was chosen as one of the sites to be studied, just waiting on more news so we can begin the process of enrollment. From the get go, this study has not been about whether or not the drug works (it does!) whether there are any major side effects (there aren’t!) it’s about finding the correct dosage for children this age.

What the FDA approved was use of this drug for qualifying individuals over the age of six. So obviously, Gavin is not in that group BUT most drugs are approved this way, and often a doctor can determine whether or not a child under that age can use it (this case might not be that way, because of the extremely high cost – nearly $250K per year). Yet to me, that age doesn’t matter – Gavin WILL get that drug. It WILL be changing our lives. The face of Cystic Fibrosis has been changed forever.

BUT – we are at a point of not if, but WHEN. I cannot contain my excitement.

When we first heard about VX-770 – now known as Kalydeco – it was in the very early stages – and our hope was that maybe, just maybe, Gavin would get a chance to try this drug when he was entering middle school. Then it was maybe, just maybe around kindergarten or first grade…never, ever did we think that we’d get this kind of news before he even hit preschool!

A major plus for Gavin, our hope is that he’s actually on the medication BEFORE Cystic Fibrosis has any major effects on him. He has never cultured anything more than staph, he has never been on more than a handful of antibiotics, he’s never been on IVs, and he’s never been hospitalized. Because the drug cannot fix previous issues (lung scarring etc) and can only help to prevent them in the future – the fact that we’re starting from nearly a blank slate is AMAZING.

Below is a link to the press release from the FDA

http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm289633.htm

Gavin has been trying to comprehend this news – but because he’s never been “that sick” it’s hard to explain to him what this means to us. He keeps hugging me and telling me he’s excited – but I think the majority of his excitement is because of my own – but in time he’ll understand.

I knew in my gut that 2012 was going to be an amazing year – but I had no idea it would be this amazing before the first month passed.

I will share more news when I get it – but tonight is going to be a CELEBRATION!

Growing Up is Hard to Do

Slowly but surely, nearly all the “babyness” has left my little guy and he’s turned into a full blown kid.

He is quick witted, can tell a funny joke and knows how to get himself out of trouble with a funny phrase or look on his face. While he still needs a snuggle from his mama every now and then, we’re enjoying this new stage of parenting.

Gavin is well into potty training and the only time he wears a diaper now is at nighttime. The other day I forgot to change him back into his diaper before a nap, he stayed dry, so that was that, no more diapers during naps – cannot WAIT to stop buying diapers! I obviously have the necessary parenting pictures of him sitting on the potty – but  I can spare him the embarrassment of posting them online. I’ve never laughed so hard as the day he sat on the potty deep in thought with his LeapPad – apparently the need to do something while doing your business begins at an early age for boys (and I’m pretty sure this is the only time men multi-task ha!).

He’s also getting more accustomed with his new hockey skates. While he is still timid on the ice, his interest in growing up and playing for the Gophers & the Wild is beginning to trump any trepidations.

 

We’ve also received the snow that Gavin was anxiously waiting for – several more inches than the pictures below. And whether he has been watching his daddy use the snow blower from a distance (he cannot wait to “grow up and snow blow!”)

Or if he’s been out helping his daddy shovel (I swear I help too!)

The kid is just plain psyched to have all the fun stuff to play in!

Still cannot believe that we’re only a couple of weeks away from him turning three. Time flies when you’re having fun!

Moganko what?

So here’s the deal. I have this awesome…SERIOUSLY awesome, friend named Josh. Several years ago – not long after Gavin was diagnosed – I felt the need to find some adults living with Cystic Fibrosis. PROOF that life still happened even with this disease. I’m pretty sure we eventually connected after he commented on my blog and I clicked over to his site to see what he was all about (correct me if I’m wrong Josh).

After a few minutes of looking at pictures I realized.

Hey, I know that place he’s standing in!

Hey, he’s wearing a Twin’s jersey.

OMG.

He lives in Minnesota! YES!!!!!!

 

I don’t know why – but “meeting” adults with CF in different states made them…well, real…but not as real as finding someone living on my turf. While I crave the information all of my adult friends have given me – I have an extra sense of love for Josh and our similar love for our home state.

After a couple years of going back and forth online – a missed meeting when Gavin was in a horrible mood (weird) – we finally got to meet face to face at this years Minnesota Cystic Fibrosis Education Day. I also got to meet his wife and mom – both of which were fantastic individuals.

Now here’s the gist of my story, Josh has created a character named Moganko…

 don’t be fooled – it’s really them – just in seriously awesome disguises

Moganko is a puppet that helps kiddos with Cystic Fibrosis understand their disease, what their extra care is for, and why it is so important to keep up with treatments. (But don’t get me wrong – it’s just as much fun to watch as an adult!) Gavin has completely fallen in love with this character. He’s getting old enough to realize that not everyone around him has CF, and seeing someone else go through treatments just like he does has been a huge blessing to our family. Listening to songs/raps about the treatments he does/the medicine he takes/the extra work that is necessary brings a priceless smile to his face. While the life we live may not be normal – Josh & Moganko have helped Gavin realize that he’s not alone and that there are people out there just. like. him.

As a mom, I couldn’t ask for anything better. Life is hard enough – much less with CF – and following Moganko’s journey has made our lives even more enjoyable.

Josh’s goal is to get Moganko teamed up with the Muppets – yep those Muppets - to create awareness for this disease. And we need your help. No, I’m not asking for money – just asking that you take a moment of your time to check out his website

Mogankoforcf.org

AND/OR, take a moment and ‘LIKE’ Moganko for Cystic Fibrosis Awareness on facebook. In both locations, Josh has posted amazing videos, blogs, songs etc. all helping kiddos just like mine live a little more peacefully with this disease. Josh’s goal is to get 2,000 likes on his Facebook page by Thursday as he heads into a (not so secret) meeting that is working toward his goal. Please help him out with a ‘LIKE’ and please SHARE this link on your facebook page. I know my friends will do anything to help my little guy live a long and healthy life – and this is a completely free way to help! Mucho love and thanks in advance!

 

 

On a slightly different note – this year’s Great Strides Walk is on May 5th and we will be walking here in Rochester (vs the Twin Cities in years past). If you would like to walk with us – or walk in a different location – please let me know and I will get you all of the information you need!

Welcome 2012!

So I’m a few days late to welcome in the New Year, but I still cannot believe that 2012 has already arrived. I feel like I blinked and the holidays were over. It didn’t help that I got the awful stomach bug going around just a couple days before Christmas and then Casey getting it on New Years Day. Luckily Gavin never got it – and he had a blast hanging out with his cousins while they were in town

While I was sick in bed, Gavin got to head out on the ice for his first time on skates. He was a little hesitant as his “skates were toooooo big”
(which they were) and the ice was “too slippery!” (duh) but he went out there for the first of many times to come!

And for those of you who noticed the figure skates, no worries – kid just got hooked up with an early birthday present from Grandma & Grandpa Groettum – his first pair of hockey skates! He is beyond excited to get out on the ice again!

If you haven’t caught on. The kid is OBSESSED with hockey. Talks about it constantly. Plays it constantly. Watches it on tv constantly. It has become an evening ritual for a hallway game to begin between dad & son…and it’s an extra bonus when Gavin pulls out his rain boots (which he pretends are skates) and football helmet.

And if that wasn’t enough – lucky kid gets to hang out with his grandpa right in the action during our local high school games

The kid is only two, but already has some major Spartan pride. Guess if we ever move we better stay close to make sure he ends up going to school there!

Also during the holiday break, we took Gavin to see Sesame Street Live. We had second row seats and I don’t think Gavin has ever sat still for that long in his life! He loved the action – but made sure the characters didn’t get too close for comfort :)

Gavin’s other love is “construction work.” Every object can easily turn into a “bammer” (hammer) and when he got a construction guy costume (Melissa and Doug) that came with tools as well as the necessary safety goggles – he was psyched! I let him bang/saw away at a couple pieces of styrofoam from another gift – only to later regret it as I tried to vacuum up a countless number of styrofoam fragments.

I seriously cannot believe that he will be three in just a few weeks. While he still likes to snuggle up with his mama, he’s getting to be such a big boy. He’s funny as heck, talks non stop (wonder where he got that trait from…) and is such a big helper around the house. He is in underwear all day at home – and only wears diapers at night or if we’re busy running around since he has a fear of public toilets – but don’t we all to some extent! :) Last night he informed Casey and me that he would be reading us our nighttime story…

 

For now I’m going to spend the remainder of naptime at our house enjoying the fresh air…yes, fresh air in January. In Minnesota. It’s 50 degrees here and the windows are open. SO surreal. But looks like today is the end of the warm streak so I’ll live it up while I can.

New Room & Snow!

This past weekend was an exciting one for our little guy – not only did we get our first major snowfall (looks like this one will stick around) but we surprised him with his new big boy bed!

It’s amazing what a difference a different bed and a little rearranging can do to a room.

The before:

The after:

Neither his dresser or armoire were originally blue. I bought the armoire off craigslist and painted it blue earlier this year (it was an ugly mix of pinks and blues). We received his dresser from Casey’s parents as a present when I was pregnant, and while it matched his crib it did not match his new trundle bed (which I bought off craigslist – perfect condition, handmade & from a very expensive store for less than $100!) so I went ahead and painted it blue. I am thinking about buying new knobs for each, but for now the brown works!

I thought I’d have a tough time moving him out of the only bed he’d ever known – but his excitement trumped any of my worries. Little did he know that this would be his last night in his baby bed…

   

And his first night in his new big boy bed was a total success! He never fell out and he’s so excited about it that he asks to go to sleep!!!

Like I mentioned before – Gavin also got his other wish this last weekend…snow!

We completed our first “frosty da snowman”

 

And spent a lot of time “shoveling,” aka moving snow around – but at least he’s getting the idea and little does he know that his lungs are getting a great workout!

The crisp air, and snow covered ground has made it finally seem like Christmas is coming!