Little's Life

Little's Life

Wednesday, November 13, 2013

Gratitude in the Face of Disappointment

It's November; that means it's time for FB's annual 30 Days of Gratitude status updates.  This is the second year I've joined in.  I think it's good to take a minute and express your thankfulness about the blessings in your life.  We have SO much to be thankful for.  God has been so good to us.  

So today makes my second post in a row about derailed hope in the Duchenne community.  Last time, I wrote about how GSK's phase III trial on the exon skipping drug, Drisapersen, failed to meet it's endpoints.  Well today, I write to let you know that the competing pharmaceutical trial, Sarepta's exon skipping drug eteplirsen, was just denied accelerated approval from the FDA.  In a nutshell, the FDA thinks the trials were too small and the data not strong enough to move forward on.  They've asked Sarepta to regroup and try a new Phase III trial with a larger group of boys and place half of them on a placebo.  Sigh.  Duchenne is such a rare disease, that it may not be possible to find that large a number of boys who qualify.  The second problem is that no one will want their child to be in the placebo group.  So we've really been punched in the ribs on this one.  It will take years longer now to get this drug out to all the boys who need it.  Years that we don't have.  This is a rapidly progressively deteriorating disease.......the math doesn't add up. 

  The FDA basically just told us all that most of our boys are going to die or have muscles that are too wasted to be helped by the time this drug is available (if it is ever available).  So in the wake of all these setbacks and heartaches, what am I thankful for today?  THE FDA.

Yep, those guys.  They have an incredibly difficult job, seeking out which drugs to give the green light to and which ones to send back to the lab.  I don't envy the FDA for a minute.  I applaud them for doing the best they can with the data they are given.  I may not always agree with them, but I don't work there.  I don't have a full grasp of what approving a drug entails.  What I do know is that the FDA hasn't denied eteplirsen because they hate people with muscle disease, they've denied it because it didn't meet the criteria they judge new therapies by.  This is not a forever denial, it is a "not now, go back and try again and give us new data first" denial.    Unfortunately, with Duchenne, it means more boys will pass and more boys will decline.  This delay may quite possibly cause Little to progress too far to benefit from the therapy by the time his formula is ready.  And that sucks.  Big fat hairy donkey balls.  But if regrouping and reformatting the trials are what ultimately lead to drug therapies that save future DMD kids - well then. 

I was told 2 years ago that my son would never get better, that he'd die before he was legally able to buy his first beer.  I've come to terms with that.  I don't want it - but I understand that's the way this genetic condition plays out.  I've accepted that my son will not lead the life I had envisioned for him while I was pregnant.  The realist in me takes whatever glimmer of light is out there with a grain of salt.  Yes, when things looked like they were working out for Andrew to be one who received a drug therapy formatted to his mutation, I got excited.  I praised God.  But my prayers have always said,  "God, if it be Your will, then....."  I have always known that these drugs might not come in time to save my son.  If they ever do, I will dance with the joy of a thousand angels in my heart, but I will not survive this journey if I don't stay grounded and humbled.  And today, with the news that none of those drugs have Little's name stamped on them at the moment, I still praise God.  He has given me so much.  He gave me Andrew.  He is letting me keep him right now, when He could have allowed a miscarriage or a stillbirth to "ease" my life and heartache - save ME from a life with Duchenne.  But instead He allowed my son to come to be.  He is allowing me to love Andrew for as long as he is on this earth.  How blessed am I?

And when the time is right, the formulas are right, and the data is there - the FDA will approve a therapy for Duchenne.  I know something is coming, even if it is not in my son's lifetime.  And I am grateful.