Tuesday, January 31, 2012

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…


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


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!

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