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!!

13 April 2019

Bluebonnet Pictures 2019

One of the best parts of living in Texas comes every Spring when the bluebonnets and other Texas wildflowers pop up alongside the highway and in random fields.  The beauty lasts for only a few weeks but when you see them, you know Spring has sprung!
It's a societal norm in Waco to drop off your children in a random field, place them among the flowers and to tell them to say "cheese".  So, we did it this year just like we have almost every year since we've been in Waco.  Here are this year's pictures and, if you keep scrolling, you'll see how much these nuggets have grown.

Oliver, age 11

Bennett, age 9 

 Avonlea, age 5

 April 2019 (Bennett, 9; Avonlea, 5; Oliver, 11)

April 2015 (Oliver, 7; Avonlea, 21 months; Bennett, 5)

April 2014 (Oliver, 6; Avonlea, 9 months; Bennett, 4)

April 2013 (Bennett, 3; Oliver, 5)

April 2012 (Bennett, 2; Oliver, 4)

April 2010 (Oliver, 2; Bennett, 7 months)

01 March 2019

An update on Bennett - March 2019

Bennett had his quarterly CF appointment last week so I thought I'd share how he's doing health-wise.  I also thought I'd share how he's doing all around:

First of all, Bennett has blueish green hair these days.  It's starting to fade but after asking consistently for at least a year to dye his hair, I finally relented, shortly after family photos were taken.  Santa brought Bennett semi-permanent hair dye.  The directions said it would wash out in anywhere between 5-25 washes.  What the directions didn't say is how often a 9 year old typically washes their hair when they don't want the dye to come out. Ha!

On one hand, I'm so glad I finally gave in to his very simple request.  A friend of mine made the point that allowing him to change his hair color was a great way to give him control over his body, which I hadn't thought about but I agree.

On the other hand, I didn't realize that, in order to put the blueish green hair dye on blonde-ish brown hair, you have to bleach it first.  Bleach: what a terrible thing for CF lungs to inhale!  Nonetheless, I was already committed at this point when I realized it, so I grinned and beared my fears and went forth.  I told him, though, I didn't imagine a time when we'd be doing this again. ;)

Last week, Bennett went to see his CF Team at Dallas Children's Hospital for his quarterly CF appointment.  Bennett gained two pounds in 3 months so his GI doc was very happy.  Gaining weight is very difficult for people with CF.

We are currently giving Bennett 3 tube feeds daily, that's over 1500 calories and he's eating on top of that, which is wonderful.  His ability to gain weight is a good sign he is staying healthy.  He's still low in BMI so he needs more calories and more weight gain.  But, for now, he's at least on track with his growth.

Bennett's lung health is also looking good.  His PFTs were 92%, down from 95%.  But, considering that he hasn't gotten sick this winter so far, his Pulmonologist gave a good report and said his lungs sound good.  Bennett's throat culture results shows he continues to be free of psuedomonas and MRSA, for now.  We're starting back up nasal rinses and Flonase due to headaches, which may indicate sinus build up.  But we are hoping we can hedge that off early.


Bennett is doing well in school.  This picture shows him sitting next to the board he made to demonstrate his work in robotics.  You can see he's wearing his feeding bag.  Bennett is very open about his CF at school.  He sorta has to be open, considering he requires a tube-feeding each day at lunch.  We are grateful that the school is more than willing to help us daily keep him on track with his weight gain!  

Oliver and Bennett (and most recently, Avonlea) started a new school this year and it's going super well.  They are growing in incredible ways.  Acton Academy is a bit of a blend of homeschooling and a gifted/talented program.  Their school is very hands-on and self-directed, which requires a lot of motivation to learn on their part.  Thankfully, since the school has designed the learning process to be so much fun, my kids have very little problem with motivation.  In fact, they beg me to drop them off at school at the first possible moment and to pick them up at the very last moment of the day.

Their school year is split in to 7 different sessions.  In addition to the core subjects of Math, Reading and Writing, each session at school has a project-based focus.  During the first session, they focused on entrepreneurship.  Their second session was focused on learning about Ancient Greek culture.  Their third session was all about Robotics.  Their current session is about Plants and Gardening (they were making plant cell models out of Play-doh earlier this week).

As naturally awesome as this session is, gardening at school has presented challenges for Bennett due to CF.  Many of the bacteria, yeast, molds and allergies that Bennett's CF lungs are susceptible to getting live in soil and decaying things.  

These bacteria, yeast, molds and allergies can easily get kicked up into the air when dirt is moved around.  And Bennett can easily breathe in these tiny particles into his lungs, which can cause havoc in his body.  If and when Bennett gets a bacteria in his lungs, it will likely require weeks on IV antibiotics and/or inhaled meds and may include weeks in the hospital.  So, we are very hesitant to risk this happening.

I hated having to explain to the teachers (who are actually called Guides at Acton) that Brian and I believe there's no amount of playing in the dirt or near the dirt that is good for Bennett.  Out of abundance of caution, we also don't let Bennett play outside when the lawn is being mowed, either.  

Not all families with CF believe it's necessary to withdraw children's playtime in the dirt - "children with CF shouldn't live in a bubble" is often an argument - but, based on what we know about the risks, Brian and I feel more comfortable letting him learn a bit more hands-off during this session.

It's disappointing that Bennett can't play in the dirt.  But, we reason that we work too hard to keep him well, to do something that we know has a risk to undermine all of the work we've done so far.  I'm thankful that the kid's school partners with us and is willing to do whatever it takes to keep Bennett's lung health optimal for as long as possible.

The last thing I thought I'd share is that we just started Bennett in twice-weekly sports training class for 7-11 year olds to see if he can improve some of the issues that showed up on Bennett's most recent physical therapy assessment.  

Although this new sports training class is an added expense and means "one more thing" to Bennett's week and our family's schedule, we are trying to focus on Bennett's long-term health goals and believe that doing so will, not only build his core muscles and improve his lung health, but also teach him that being physically active is caring for himself.  

Thankfully, Bennett really enjoys working out and has made new friends.  A new friend said to his mother other night about Bennett; "he's a lot of fun." 

I'm so glad to have received a good report on Bennett's health and to breath a sigh of relief for another 3 months.  Brian and I are incredibly proud of Bennett's resilience and his willingness to try new things!