27 March 2016

Happy Easter!

He is Risen!

Baylor's Heisman's trophy winner RGIII always says, "know your why."  
As the pastor at our church said to us this morning, 
"Today is our why.  Today is why we do what we do."

"Because He lives, I can face tomorrow, 
Because He lives, all fear is gone,
Because I know He holds the future,  
Life is worth the living,
just because He lives!"
-Christian hymn

 Happy Easter,

23 March 2016

VLC 2016: Most Current Cystic Fibrosis Science Update from the CFF

Two weeks ago, I had the privilege of attending the Cystic Fibrosis Foundation's Volunteer Leadership Conference (VLC) in Washington DC.  

I was one of more than 500+ parents, volunteers and CF staff who attended this conference that offered us an opportunity to network, share our experiences, gain new insights from other members of the CF community, learn more about what the Cystic Fibrosis Foundation (CFF) is doing and hear the latest on the CFF's effort to find a cure for CF.

This blogpost (one of two) will summarize the parts the state of the science in our effort to find a cure for CF.  

For those who have 2 copies of the F508del gene mutation, Ivacaftor (Kalydeco) and Lumacaftor (Orkambi) is currently available on the market.  But, as you can see from the slide above, it reaches only about 30% of the normal CI- transport.  This is an improvement from the less than 10% most people with CF have.  But, fortunately, there are three potential medications coming down the pipeline that might help.  The first is VX-661 (whose trade name will be Tezacaftor).  The second is VX-152.  The third is VX-440.  In the lab, it looks like the combination of Ivacaftor (Kalydeco) + VX-661 + VX-152 might offer the greatest benefit to people with CF.  But, the Ivacaftor + VX-661 and VX-440 may also be a good option. 

For people like Bennett who have only one F508del mutation, Ivacaftor + VX-661 + VX-152 may still be the best option, however, the drug benefits will be lessened from potentially 80% normal CI- transport to possibly 50%.  

Thankfully, VX-661 might be available as early as late 2017 or early 2018.  And it is hoped there will be a Phase 2 study in people with CF evaluating VX-440 or VX-152 in combination with Ivacaftor + VX-661 as early as the second half of 2016.  It is still unknown when that study will begin.  Check out this Vertex Research Pipeline:

So when might these 2nd generation correctors be available to patients with CF?  Dr. Boyle, from the CFF, said probably in 3-4 years ("maybe between 2020 and 2022").

Although it was not spoken about in depth, Dr. Bill Skach reminded us that CF Foundation is working on finding a cure for every class of CF gene mutations.  (Check out the CFF's pipeline here:

The CFF's goal is to find a cure for 100% of those with CF.  Ninety-five percent of people are expected to benefit from Kalydeco and a corrector medication.  There are a remaining 5% who will not benefit from Kayldeco + a corrector drug but there are efforts to help them as well, including the 3% who have nonsense mutations and the remaining 2% without nonsense mutations.

A new initiative has been started for those in that last 5% of CF mutations who will not benefit from corrector and potentiator drugs.  This initiative is called the "Nonsense and Rare Mutation Initiative" where 40 million dollars has already been committed to find a cure for these people as well.

A few of the other projects that the CFF is working on include a "New Technologies Research Conference" where they are focusing on gene expression, editing, deliver and stem cells" (which, by the way, there will be a gene-editing plenary at the North American CF Conference this fall).  The CFF is expanding their CF Foundation Therapeutics Labs with a focus on nonsense drug discovery, stem cells and gene editing.  And the CFF is working to develop the next generation of CF researchers will more training opportunities and career development.  

According to Dr. Bill Skach, the CFF is funding more grants, funding more labs and putting in more resources, towards a cure than ever before...all while also putting more resources towards expanding the programs to help those with CF live a healthier and happier life.

Dr. Boyle gave an overview on the top 5 Emerging Topics in CF Clinical Research.  They include: a worldwide effort to enroll clinical trials, an explosion of CFTR modulator trials, new anti-inflammatory clinical trials, continued work with the FDA and a new focus on lung transplantation.

The CFF is working to improve enrollment for CF Clinical Trials by supporting and working with other CF Clinical Networks.  This is good for everyone in our 70,000-person worldwide community.

There has been an explosion of CFTR modulator trials.  This means that there may very soon be competition for Vertex.  This is such amazing news as more and more labs are knocking on the CFF's door to get a piece of the Finding-A-Cure-For-CF pie.

VX-661 is already involved in clinical trials.  The VX661-108 trial, evaluating those with 1 F508del mutation and one residual function mutation (typically those who are pancreatic sufficient) is likely to complete in 2017.  The VX661-106 trial, those with 2 F508del mutations, may finish later this year.  The VX661-109 clinical trial is still working to build it's site capacity.

Inflammation is part of the devastating cycle of CF and it's damage on the lungs.  So, it's really great to see there are some potential anti-inflammatory medications in clinical trials.  

The fourth topic of Emerging Topics in CF Clinical Research is that the CFF is working with the FDA.  The CFF has put out a guidelines paper on CFTR modulator development, has a partnership with the FDA Pulmonary Division, holds a join workshop with the FDA, CF researchers, parents and families and surveys physicians and patients.

The CFF recognized there was a recent negative FDA ruling on Kalydeco and residual function mutations.  It was not a surprise as it was based on mainly lab data.  They are evaluating the best path forward for residual function mutations, negotiating other potential challenges with FDA and is look at the use of placebo/role of in-vitro testing in FDA approvals.

The last Emerging Topic in CF Clinical Research presented by Dr. Boyle was that the CFF is taking on lung transplantation.  Lung transplantation is the last resort for many with CF at end-stage disease. The CFF wants to increase support during that period of time.  

The CFF recently held a CF Lung Transplantation Summit that began evaluating how to improve pre-transplant, transplant and post-transplant care.  Dr. Boyle said, "we know lungs are rejected faster than kidneys and hearts.  We need to find out why."

In the next VLC blog post, I'll share more about the good things the CF Foundation is doing to improve care for people with CF!

19 March 2016

After sick, there was Sic 'em!

Several weeks ago, on a quiet Friday afternoon the day after Bennett got out of the hospital, the doorbell rang.

I was the in backyard pushing Avonlea and Bennett in the swing when I heard Oliver yell: "Somebody is here!  And they are holding a ball!"

Thankfully, everybody was dressed (not always the case when you have a toddler) as we opened the door to find an outgoing man with a quick speaking northeastern accent wearing a BU shirt and holding a Baylor basketball in his hands.

This man, who we had never met, seemed to know each of us by name.  But he was looking specifically for Bennett.

The tall man introduced himself in a way that would delight any child, "My name is Timothy.  Can you say baloney?  Then you can say my name, Timothy Maloney."

Coach Maloney, as he is known by the Baylor Men's Basketball team, came to our home to bring Bennett a basketball signed by the Baylor Men's Basketball players and then invited Bennett to come to a real live Baylor basketball practice.  

"Can I come?!" Oliver asked.  Coach Maloney touched the top of Oliver head.  "Of course you can!  Your whole family can come!"

A dear friend and sorority sister had asked me when Bennett was still in the hospital if Bennett had any interest in Baylor basketball.  Turns out, she has a close friend who is related to Scott Drew, the Baylor Men's Basketball Head Coach.  She thought she might be able to find a way to cheer Bennett up after what he had gone through.  I didn't know much more than that until Coach Maloney showed up to our house.

Before he left, Coach Maloney said, "call me next week and we'll set up some time to have you guys come to practice" which I promised we would.

After having briefly hosted such a fun visitor with such a generous gift and offer, I took a video the next day after Coach Maloney's visit to capture Bennett's thoughts:

I had planned to call Coach Maloney on Monday...until it became clear that Bennett wasn't going to be able to go back to school for some time more.

Our family was profoundly disappointed that Bennett could't go back to school.  It was hard enough that Bennett couldn't see his friends and get in his normal routine.  But on top of that, we didn't think we would get a chance to see the Men's Basketball team practice this season.  It just added insult to injury.  Even this, a very special opportunity, CF seemed to rob.

But, upon hearing this, Coach Maloney reassured me we would figure something out, one way or the other, when Bennett felt better again.  His reassurance allowed me to put my focus back on getting Bennett better again.

Thankfully, Bennett was eventually able to get back to school.  So, we scheduled a visit to the Men's basketball practice as soon as we could.

We were so delighted when Bennett did finally feel better enough to go back to school and we finally got to visit the Baylor Bears on their practice court.

Both boys really enjoyed watching the boys do their drills and scrimmage.  Although I caught a giant yawn from 2 year old Avonlea, she had a great time.  She kept yelling, "They running!  They running!"

Since practice was after school, the kids were very excited for a snack while they watched their afternoon's entertainment.  The basketball players ran and ran for a long time.  It was everything I could do to keep myself from offering them something to eat as well!  Those guys work HARD!

Throughout the practice, when the players stopped to drink water, they would come, look us in the eyes, shake our hands and say hello.  It was SO kind.  We felt so valued.  Here we were just coming to watch them, like flies on the wall, and it turned out they felt us important enough to stop and meet. 

I don't have a picture of this (although it will always be seared in my memory) but at the end of practice, the basketball team (players and coaches) huddled together in a circle and end in prayer.  When they gathered together, Coach Maloney turned to Bennett and welcomed him into the circle. 

Then, Coach Maloney turned to the rest of our family and asked us all to be a part.  He said, "come on all of you guys, you are a team."  I loved hearing that because there might have been no other time I have more felt this but when Bennett was recently in the hospital.

As we gathered our team and joined with the Basketball team in their huddle, Coach Drew acknowledged us and turned to Coach Maloney to share about Bennett's fight with CF.  Coach Maloney shared with the team what a strong guy Bennett is and then closed out the practice in prayer.

Afterwards, we were asked if we would stand with the team for a picture.  It was SUCH a special experience overall.

In fact, the next day in kindergarten, Bennett stood in front of his class during "Show and Tell" to tell how happy it made him.

A few days later, after seeing a practice, we decided to bring the kids to an actual Baylor Basketball game, which they had never had a chance to go to.  But the only last minute tickets were able to snag were in the nosebleed section. 

Upon hearing we were coming to the game, Coach Maloney came to visit us right before the clock started, which was such so fun.  But when he saw we were in probably the worst seats possible, he said "I'm going to try to get you guys back to a game in better seats."

And so he did.  For the very last game of the 2016 Baylor Basketball season, we had seats that were practically on the floor.  And they were FUN!!

We sat close enough that Oliver and Bennett were able to get out of their seats to give high-fives to the players as they were introduced.

We got there early enough that Avonlea had a chance to meet with some of the Baylor cheerleaders which made her happy.

We had such a wonderful time together  Bennett still had to go to the bathroom a million times.  But it was so worth it!  Getting so close to see the Baylor Bears play was a fun immersive experience.  

There were a couple of insights I gained through this experience that I thought I'd share:  

The first insight I gained is: 
Love feels pretty amazing after loss.

Having such a fun unique and special experience offered to us by the kindness of strangers was so meaningful after having been through several weeks of hell.  

I was exhausted from having to watch my son suffer.  Bennett was tired of being poked and feeling badly.  Avonlea and Oliver were over having to be away from their parents.  And Brian longed for normalcy.  This experience was a family experience that brought us together and gave us something new and exciting to think about.

The second insight I gained is:
Time slows during the good moments, just as it does during the really horrible ones.  

When we were visiting with Coach Maloney, watching the team practice or attending a game, it felt like time slowed.  We were able to enjoy sights and sounds.  We were able to be fully present in an experience that felt bigger than us - both of which made it feel like time somehow slowed.  

The same thing happened when Bennett was in the midst of his pain.  Time slowed way down.  So, it's nice to be able to counteract that slowed time of being in the hospital, feeling lonely, experiencing pain with time doing something we enjoyed, feeling included and experiencing joy.

The third insight I gained is:
Making good memories is especially important after which point bad memories have been made.

Bennett's last hospital experience was pretty horrible.  And one of the things that saddened me the most was that Bennett is now at the age where he will remember it.  It's one thing when your child is hurting but he's too little to remember.  It's another when you know he knows what's going on and won't likely forget it.  

But having this Baylor Men's Basketball team experience reminds me of a quote I shared recently from my friend Emily who told me about Bennett's journey with CF: 

"Will it affect him? Yea. What will he remember? How in the horror of the hospital experience, you and his dad were his relentless comforters and companions and advocates -- and never left his side. How he wasn't in any of this alone. How it was a family journey. How home is where the family is. How through the worst pain and fear and agony, we can survive... and rise and thrive have to remember, this will be just one memory of one experience in a quilt of many more. And most of those other ones will be wonderful patches."

This one was a wonderful patch.

Tim Maloney, you are part of our family now!  Thank you for opening your heart to us.  We love you!

Coach Drew, Jane and Amy, thank each of you for hearing and responding to our story.  It is your faithfulness that helped bring laughter to some really sad hearts, especially Bennett's.

And to the Baylor Men's Basketball team, thank you for reminding us what it means to be a team.  Thank you for using your team to encourage and love on ours.  

15 March 2016

5 Things You Might Not Know About Waco from HGTVs Fixer Upper

When Brian, Oliver and I moved to Waco right before Bennett was born, Waco was most known on a national level for the David Koresh Branch Davidians.

I have to say it's pretty fun to see your town on TV each week.  It's been fun to live in the mecca of all things french-country and fixer-upper-ish.  So many people from around the nation travel here each weekend to experience Fixer Upper in the flesh.

But, there is only so much you can get from TV.  So, here's my list of things you might not know about Waco just from watching Fixer Upper:

1.) The closer your house is to McGregor, the more likely your house is to shake.  
When we first moved to Waco, I thought that constant rumbling and shaking of our home was because of a nearby train.  But then, why would a train just suddenly stop making noise as quickly as it started?  Then, I thought it was an earthquake.  But there were no reports in the media.  Later, I learned from a friend it was just SpaceX testing rockets.  Apparently, McGregor houses a SpaceX rocket testing site.  Check out this guy's video about it here.  Seriously, this makes our house shake, ever so slightly, several times a week.  It's the weirdest thing.

2.) MARS makes Snickers, M&M's, Starbursts and Skittles in our backyard. 
Founded in 1976, Waco's MARS plant has been known to make 85 percent of the popular Snickers candy bars produced in North America.  You would think they would offer tours to all the little school children wanting to learn how candy is made.  But this isn't Hershey, Pennsylvania.  No candy handouts or tours here.  Boo!  This high-security plant is not open to the public.  If you want your MARS candy, you'll have to drive down the street to the local grocery store. Still, it's kinda fun to drive by yellow and red M&M character statues every day on the way home from school.

The crazy number of birds in Waco are so iconic, Congress Clothing even sells tshirts with birds on it.
3.) Every Fall in Waco will remind you of a scene from Alfred Hitchcock's movie, "The Birds."
Ever seen the Alfred Hitchcock movie, "The Birds?"  Well, if you're out and about on a fall evening in Waco, you will almost certainly instantly feel like you're in a horror movie scene.  The birds are called grackles and thousands of them descend on the Central Texas area at dusk each fall making an incredibly loud noise.  They arrive each Fall because they head south for the winter.  There are so many of them that Waco has even had to resort to a variety of measures to control them.  Electricity lines are filled with them as are every branch of trees.  They hang out on roofs and on the top of people's cars.  I've never seen them be aggressive but they are fear-inducing none the least.  It's creepy.  But it's also a little cool to see, too.

4.) The only traffic you'll ever see in Waco is on the street of the most current Fixer Upper episode home.  
I haven't been able to find a map of all of the Fixer Upper homes in the area.  But the one thing about living in Waco is almost everybody knows somebody who has been on the show.  So, it's relatively easy to find houses that have been redone.  But, in case you don't know which house is currently being remodeled, follow the Waco traffic.  It's the only time you'll run in to it.

Photo by Snaptastic Shots

5.) The one monument everybody in Waco loves most is the Waco Suspension Bridge.
People get their engagement photos taken on this bridge (see here).  People wear tshirts with a picture of bridge on it.  People buy coffee mugs and ornaments with a tiny suspension bridges painted on them.  It's crazy.  It's so ingrained in the Wacoan culture that I even arranged to take the kids out there for our family Christmas photos this year.  The Waco Suspension Bridge has been here since 1870 and you aren't a Wacoan if you don't fall in love with this monument of Waco history.

There are lots of great things to see and do in Waco besides the Magnolia SilosHarp Design Co., Lula Jane's, Junque in the Trunk, Sironia, Spice Village, Ninfa's, Dichotomy, the Olive Branch, Common Grounds, Cameron Park and the Zoo all come to mind as our favorites.

But the most important thing you might not know about Waco from HGTV's "Fixer Upper" is how most out-of-towners who move to Waco, initially finding themselves disappointed by the small town "have-nots", eventually fall in love with this quiet close-knit family-oriented community.

Come visit us and see for yourself,