SOCIAL MEDIA

09 June 2021

Trikafta Approved!

Joy!  That is what our family feels today!! 

 

This morning, with one eye open and the other still shut, I woke up, rolled over and opened my smartphone.  I clicked a notification to find a link sent to me by another mother of a child with cystic fibrosis. My heart lept: 

It's approved!!!!!! 

A few minutes later, Bennett walked in my room. I was delighted to share with him the good news, "Hey buddy, your medicine has been approved!"  He grinned and gave me a high five!  It was the news we were waiting for.

Trikafta, the first medication approved by the FDA that benefits Bennett's age group and Bennett's genetic mutation by addressing the underlying cause of his cystic fibrosis, is now available for Bennett to take!  We are now within weeks of starting this new potentially life-changing therapy.

If you've prayed for Bennett, walked for Bennett, raised money on behalf of Bennett and/or followed Bennett's blog, THANK YOU!!!  It is our hope that you will celebrate this huge gift with us!!  You are part of his story!  You are part of *this* story!!


We have so much to be grateful for.  Bennett is 11 years old and has stayed very healthy so far.  His lung function currently sits at 89%.  However, he has never had a cough or acquired a lung infection that required IV antibiotics.  We hope the Trikafta will increase Bennett's lung function to 100%, which would be considered normal for any child.

Bennett is in the 40th percentile for his height but he struggles most with his weight.  Despite a g-tube and daily tube feedings, Bennett's weight is in the 13th percentile (his doctors prefer his weight to be at 50 percentile).  So, we hope Trikafta will help Bennett's GI system work more efficiently allowing him to keep the nutrients he takes in.  And, if possible, we hope Trikafta might even allow him to get rid of the g-tube (something he's never lived a day in his life without).  Additionally, we hope Trikafta will extend Bennett's life expectancy.



Trikafta, which has been approved for people ages 12+ since October 2019, affects every person differently.  We don't know if it will work well for Bennett.  There have been some side effects that have been difficult enough for some people that they've had to stop taking the medication.  But clinical trials seem to indicate that children have tolerated the medicine well.  So, we are very hopeful.

I wrote in my blog many years ago about what it feels like when the CF community rejoices at the approval of a drug for those with cystic fibrosis, but a drug in which your child cannot benefit.  This was the case in 2012.  There was so much excitement and so much hope.  But, we couldn't benefit because Bennett didn't have the right gene mutation. 

I am very aware that, today, there are around 10% of people with cystic fibrosis and their families for which today is a painful day.  They are excited for the CF community.  But they know their random genetics mean they must wait longer until they can benefit from a drug that will increase their life expectancy and give them relief from this cruel disease.  They are on a clock, a race for a medication like Trikafta and each day that goes by is another day lost.  This is also the case for so many CF families around the world who do not yet have this medication available to them in their country.  For these families, I just want to say, as exciting as today is, there is a sadness I feel with you, as well.  Your time is coming too and I will be anxiously waiting along with you, working to help us find a medication that benefits your child, your family member, you.  We are in this together, as the CF Foundation's tagline says, "Until it's done."

Lastly, I want to thank the hard working people at Vertex and the Cystic Fibrosis Foundation who worked so hard to make this dream a reality.  Bennett's story began many years before Bennett's born, before we knew we would become a part of the cystic fibrosis community.  Thank you to all the scientists, investors and brilliant minds that set forth this venture philanthropy model of discovery to bring this medication forward.  Thank you to those clinicians and clinical trial participants who tested this medication so we can feel confident about it's safety.  Today's joy isn't only because Bennett has hope like never before.  It's because we realize the gift we've been given through the work and sacrifice of so many before us.

We can't wait to share when Bennett receives Trikafta in hand.  And we can't wait to see what happens.  Trikafta is not a cure.  CF is not going away.  But it's hope that CF will have just a little less control over Bennett's life, and for that, we are eternally grateful!

Today is a very exciting day!!!  We hope you will celebrate with us!!  CHEERS!!!!