22 October 2019

Trikafta is what we're afta!

Excitement in the CF Community is through the roof!  Yesterday, the FDA approved Trikafta, a triple combination medication that is believed to eventually benefit 90% of people with cystic fibrosis!  This is EXCITING NEWS!!!!  This is no cure.  But CF clinicians and patients both say this is a GAME CHANGER!!!

Adults in our CF community who have been in clinical trials with this drug have been talking for months about the significant changes they've seen personally.  This drug, for many people, has meant fewer hospitalizations, increased lung function and healthier feelings overall.  It's almost hard to wrap my brain around!

In order to benefit from Trikafta (pronounced "Try-Kaft-Uh"), the FDA says one must have two specific CF gene mutations: F508del and a nonsense mutation.  Bennett has both.

The FDA also says one must be 12 years old.

Bennett just turned 10 last month.

This means that he will now need to wait two more years to benefit, unless the FDA approves the drug for his age group before then.

To give perspective on what that might look like - there are currently 3 other drugs already on the market for people with cystic fibrosis (none of which benefit Bennett), the latest medicine, Symdecko, was approved for adults ages 12+ in February 2018.  It took more than a year, in June 2019 before it was approved in children ages 6-11.  So, our wait could be a while.

So, we're in that gray space.  The space of almost but not quite.  GET EXCITED!!!!! But wait...

It means fewer hospitalizations, increased lung function, healthier feelings overall are to come for Bennett...but not yet.

As a mama, these dual feelings are like what one might feel like, if having waited in line for hours to get on a much anticipated roller coaster ride, the ride stopped working, just as it's finally time to get on it.  It's elation met with disappointment.  It's a muffled joy.  It's hope deferred.

Sure, comfort comes in the form of reassuring thoughts: "they'll get the ride up and going.  It will be your turn really soon."  But the wait feels particularly difficult to endure now.  The build-up so intense. Pinned up hope aches for a place to go.  It's so close now.  But, knowing it's not our turn just yet gives way to fears: could this thing I've waited for so long actually be illusive?

I have thought many times today and yesterday about a blog post I wrote several years ago when the first CF drug, Kalydeco, came out on the market.  I felt a similar feeling then too.  But, as I reminded myself then and do again today - rescue helicopters are going to come back for my loved one too.  I just have to hold on and wait a bit longer.

To be honest, waiting can be a gift - because it gives me great empathy for the 10% of people with CF with two nonsense mutations who have NO drug available right now from which they benefit.  They don't get the privilege of waiting for their son to turn 12 so they can have a medicine too.

I think of my friend and CF adult, Emily, who is fighting tooth and nail to move science forward faster, in time that she herself would benefit.  Her work and the work of her nonprofit Emily's Entourage has given us all hope that one day, sooner than later, 100% of people with CF will have something that will slow down this awful disease.  But until then, she painfully waits.

The CF Foundation continues to work on this as well, regularly reminding us: we won't stop until it's done.  And the job isn't done until ALL people with CF have a drug that makes a meaningful difference on their health...and even still, until there is a cure.

So, while our family waits a little longer, we will know this is the same ache many still feel in the CF community.  And while we wait, we will experience the anticipation of getting access to a bottle of hope called Trikafta.

Bennett moves his cart of medicines and machines to his room for bedtime treatments.

I tried to explain to Bennett last night news about this drug.  I tried to manage his expectations, just as I try to manage my own.  I explained that if this drug works, like people are saying it does, it might mean he wouldn't have to go to the hospital as often.  It might mean he could stop some of his medicines at some point.  He might even be able to stop doing his twice-daily Vest. 

Doctors don't yet know what this drug will really be able to do for a person.  Drugs affect people in different ways.  And different clinicians are more or less comfortable testing a drug's potential.  It will take a series of trials and errors to find out just how this drug will help Bennett, once he gets there.  And researchers will begin studying the drug on a larger scale once more people have access.

Bennett asked if he would still have to do his pump.  I told him I didn't know.  But I hoped this new medicine might allow him to stop having to get so many tube feeds.  He is very excited about the idea that anything, even remotely like a cure, might be within reach.

For now, we head in to flu season, praying for God's continued protection.  And we will just keep doing his CF meds as normal. 

We can't stop now.  For Bennett, hope is on its way.  Trikafta, we can't wait to get our hands on you!!!!!

**Note: To all those in the CF Community who have come before us, thank you.  Thank you for your diligence in fundraising...thank you for putting a laser focus on drug development...thank you for continuing to persevere until a cure is found.  We are a part of a larger story that has been happening long before we were here and it feels amazing to be able to experience this particular chapter of the story.  Also, to our extended community who loves and supports Bennett, I hope you can take joy in this celebration as well!  You have contributed financially and physically to see a cure for Bennett.  It's happening, guys!!!  So, thank you!

01 October 2019

Double Digits!?!

Today, Bennett turned double digits! Bennett is 10 years old! It's hard to wrap my brain around.  He's halfway grown. 

When I met him for the first time in the NICU, his body swollen and near death, I didn't have any idea the amazing adventure I would end up going on with him as his mother.  Over hills and through dark caves, splashing across creeks and sliding down rocks, I would experience the pains and joys of 10 years of caring for Bennett with cystic fibrosis.  Each adventure would reveal a jewel, a tiny gift from the the experience, a sliver revelation of who Bennett is inside.  I recognize this adventure is not over.  But, ten years in to it, I am incredibly grateful for the trip so far.  How lucky I am to be his mother.

So, who is Bennett, at age 10?  Bennett is playful, outgoing and loves to be with people.  He very tender-hearted and sensitive to other's feelings.  He lives in the moment and is always up for something exciting.  Bennett is brave, so very brave.  He is resilient and strong.  Bennett is compliant and easy to please.  He is wise.  Bennett rarely complains.  He is great friend to others and incredibly lovable.  Bennett is passionate about superheros, has a weak spot for gummy bears, and loves surprises.  In his free time, he enjoys playing with Lego men, singing to The Greatest Showman soundtrack with his sister and dressing up, especially if it makes you laugh.

Today, on Bennett's birthday, I decided to take the children to get flu shots.  I hated that it fell on Bennett's birthday but I felt it was important enough for us to get the when we could.  Bennett asked to be the first one to get his flu shot, as an example to his sister and to get it over with.  He was a rockstar.

Ultimately, the whole crew got their flu shot...despite little sister's brief protest.

Avonlea felt a little better about the process of getting a flu shot today when she learned she could give her doll her own flu shot too.  (I guess she reasoned:"if you can't beat 'em, join 'em.") She started by cleaning the area.

We're thankful for Hillcrest Pediatrics for being so kind to let us have a syringe so baby doll could get her shot....

...and a band aid that matched her mama's. 

Tears all gone - just smiles and the confidence that we're doing everything we can to keep Bennett healthy!

After flu shots and errands, we celebrated Bennett with a Pizookie from BJ's.  Here's to double digits, Bennett!  May we find a cure for cystic fibrosis and, maybe one day, even celebrate you in the triple digits!  Happy Birthday!!