Thursday, May 21, 2015

Activating People and Families Living with Cystic Fibrosis in CF Care

There is a change going on in healthcare towards patient-centered care.  This is also happening at the Cystic Fibrosis Foundation.  And it's revolutionizing the way we do health care.

One of the signs I see this in my life is in my role as co-chair of my local CF Clinic's Parent Advisory Council (PAC).  The goal of a CF Patient Advisory Council is to allow patients and parents to partner together with their CF Clinicians on improving CF Care through our CF Clinic.  

Each month, I drive four hours round trip to up Dallas in an effort to partner with other CF parents and Bennett's CF Care Team to make sure he, and the other patients at Dallas Children's Medical Center's CF Clinic, get the best of care.

Next week, I will meet again with a group of CF clinicians, researchers, CF Foundation leaders, caregivers and patients for the second CF Care Model of the Future Design Meeting in Bethesda, MD.  Along with our workgroup, I will help present several aspects of a new vision we have for patient-centered CF care, with regard to 
CF Center Accreditation and within Patient and Family Advisory Councils.  

I wanted to share a document that I came across while working with the CF Model Design workgroup on patient-centered CF care. 

I found this timeline to be very interesting so I wanted to share with other CF parents/patients.
The document copied below shows a timeline of how the CF Foundation has moved towards having CF families more involved in what they are doing on the national level.  

I'll share more on this topic soon.  But in the meantime, I'd love to hear what others think about patient-centered CF care and ways we might better appreciate the CF patient/caregiver voice.

"Activating People and Families Living with Cystic Fibrosis in CF Care" 
Authored by Leslie Hazle, Kathy Sabadosa, Terri Schindler and Karen Homa

National quality improvement (QI) initiative launched to extend life expectancy through consistent implementation of evidence-based care.

Parents and patients join CF center care staff at Cincinnati Children?s Medical Center as part of the Robert Wood Johnson Foundation's Pursuing Perfection initiative to improve care.


National QI collaborative actively engages CF care centers, patients and families in learning how to partner and  improve CF care.

Parents of a child with CF and an adult with CF join the CF Foundation's Education Committee.


Strategic planning to set QI Worthy Goals with a primary goal includes patients and families as full members of the care team.

Patient Registry Report links health outcomes to national QI initiative goals.


Web cast highlights Cincinnati Children's CF Center partnership with parents to improve CF care.


CF Foundation publishes all accredited care centers health outcomes on

"Action Guide for Accelerating Improvement in Cystic Fibrosis Care"published and distributed to accredited CF care centers, features engagement of patients and families.


CF Foundation publishes the "Improve your CF Care" toolkit written by and for patients and parents on


Patients and families are included on practice guidelines steering committee and working groups.


CF Foundation's published strategic objectives include: "Deliver high-quality, compassionate
patient and family-centered care through our Care Center Network" and "Help patients and their families find new ways to improve their self-management skills."


First Patient and Family Advisory Board Track offered at the North American CF

Development of a National Patient and Family Experience of Care Survey is initiated.

First year-long QI collaborative focused on adult care is launched.


Patient and Family Experience of Care survey piloted at 25 CF Care Centers.


Patient and Family Experience of Care Survey administered at CF Care Centers undergoing CF Foundation accreditation site visits.

The CF Foundation begins supporting video conferencing in it's efforts so CF adult patients can be involved.

The CF Foundation updated their infection prevention and control guidelines in the spring of 2013 based on research that was also released in 2013.

The CF Foundation creates the CF Adult Advisory Council, which 
was identified as a priority in the strategic plan.

The CF Foundation ends the "Patient and Family Advisory Board Track" at the North American CF Conference, specifically for patients and families.  Instead, the CF Foundation invites CF Advisors/patient families to attend the entire North American CF Conference, reducing the cost for CF Advisors to make it more affordable for CF families to attend.  In addition, the CF Foundation announces it will live-stream portions of the CF Conference for CF families and patients to be involved remotely.

Patient and Family Experience of Care Survey administered at all CF Care Centers.  Every individual and family with CF will be asked to complete a survey every 6 months.  Prior to 2015, the survey was only administered to centers that were scheduled for accreditation site visits by the CF Foundation.

The CF Care Model Design Project and Coproduction Pilot Project launched to develop and implement plans so that all people with CF have access to high-quality, specialized and accredited CF care centers and prescribed therapies.

Wednesday, May 20, 2015

Bennett's Brigade Kendra Scott/Baby Bow Tie Fundraiser in Dallas Tomorrow!

We are super excited to attend the tomorrow's second annual 

Kendra Scott - West Village Dallas + Baby Bow Tie Fundraiser 
for Cystic Fibrosis in honor of Bennett 

Tomorrow, May 21, from 5-8pm @ 3699 McKinney Ave c305 in Dallas!

Kat Armstrong, owner of Baby Bow Tie has done amazing job getting ready for tomorrow's fundraiser.  Our family is so grateful for her generosity and hospitality as she is helping to host such a fun party to raise money for a cure for our son!

If you've never been to a Kendra Scott store, you've got to go.  It's pretty incredible.  And there is nothing more adorable than getting to see all of the sweet Baby Bow Ties in person!

We are also so very excited to meet Michelle Staubach Grimes, Dallas-based author of "Where is Pidge?" and supporter of the Cystic Fibrosis Foundation as she lost her best friend of the disease, who will be at the Kendra Scott + Baby Bow Tie fundraiser for a book signing!

I cannot thank Dyan Kethley enough for the beautiful photos she captured at our Kendra Scott + Baby Bow Tie event last year.  These pictures are so fun to go back and look at - we had such fun last year.  And, it's fun to see how much the kids have each grown over the course of the year.

We hope that if you're in the Dallas/Fort Worth area and can make it out to come see us, you will! For our out of town friends, if you'd like to participate, you can purchase a piece of Kendra Scott jewelry or a Baby Bow Tie with proceeds going to the CF Foundation on behalf of Bennett by calling the Kendra Scott store during the fundraising hours of 5-8pm.  Just call 214-528-4800 and they will mail to you!

Sunday, May 17, 2015

Cystic Fibrosis has taught me the power (and gift of) speaking out on behalf of others.

I have spent much time evaluating whether or not to write this post.  I have consulted many people on this decision and given it much prayer.  In the end it may, in fact, not be the right thing to do.  But I also don't think it's the wrong thing.

Cystic Fibrosis has and continues to teach me so many things about life.  One of those is the power (and gift) of speaking out on behalf of others.  Over and over again, so very many people have done it for me - for Bennett - and for our family, telling our story and advocating on our behalf in finding Bennett a cure.  How, I have wondered over and over, can I not do it for someone else?

This post is about Brian's and my friend, Brandon Bostian.  You might have heard about him lately in the news.

There are so few people in this world who love trains more than our friend, Brandon.  Brian and I met each other more than 10 years ago at a campus church at the University of Missouri.  This is also nhw we knew our friend Brandon.  We were all a part of a large community of friends who regularly spent time together on and off campus.

Among our college church community of friends, Brandon's name was synonymous with trains.  He loved talking about them.  He loved thinking about them.  And, we all knew, his life-long dream was to eventually drive them.  Everything Brandon did in college was with the ultimate goal to one day become an Amtrak engineer.

After college, Brian and I became engaged and married (Brandon, along with many of our friends, came to our wedding).  Brian and I moved away.  Brandon eventually moved away too.  We all stayed Facebook friends but otherwise lost touch.

...Until one random day a few years ago when I ran in to Brandon in Washington D.C. of all places, at Union Station.

I was in Washington for the Cystic Fibrosis Foundation's March on the Hill, a time when I advocate on Capitol Hill for a cure for Bennett.

I was with my little boys (Avonlea wasn't born yet and Brian was back at home working) and my parents.  We were about to all have dinner together when I saw a familiar face in the crowd.  It was Brandon.

It took Brandon and me a few minutes to get over the shock of seeing each other in the most random of places.  Well, it felt random for me.  But it wasn't random for him.  Union Station was practically at home, for him.

Union Station is where the Amtrak trains come and go.  Brandon, now an Amtrak engineer, had been off on a break and was hanging out at the station.  In hindsight, instead of being surprised to see him there, I should have known.  This was exactly where to find Brandon - with the trains.

I remember really loving getting to see him.  Our brief visit reminded me why I liked him.  He's a sweet and upstanding guy, the kind of friend anyone would be proud of.

I introduced him to Oliver and Bennett, ages 3 and 5 at the time.  And I introduced them to him.  I shared with Brandon about how I was in DC advocating for Bennett.  He shared a little bit about his experience as an Amtrak engineer.

Our visit was a quick one but it was nice to reconnect - the way good friendships do.

When I returned home, Brandon contacted me via Facebook and wanted to buy a Bennett's Brigade t-shirt.  Brandon wanted to support Bennett.  He lived far away and we hadn't been in touch for several years, but that didn't change who he was as a person.  Brandon cared for people and Bennett, someone whom he had only barely met, was one of those who he cared about.  That meant a lot to me.

Brian and I are overwhelmed with sadness since we learned our dear friend Brandon's name was released as the engineer of Amtrak 188, the train involved in a high-speed accident that killed 8 and forever impacted the lives of all those on board.  We can think of no one more responsible or caring than Brandon.  This accident is a tragedy on so many levels.

Brian and I don't know what happened during the accident.  We haven't heard from Brandon since the accident and I would not expect we will for some time.  CF has certainly taught us about the value of being patient when a trauma happens.  Sometimes the best gift one can give a friend is to just be there, quietly and patiently waiting to help when and if the opportunity arises.

But, in the meantime, we are comforted to know about Brandon is the kind of person who would have done whatever he could to care for his train and the people for whom he was responsible. Ever consistent and faithful, if Brandon said he would do something, we all knew we could count on him to do it.  And, as much as Brandon loves train (which is a a lot), he cares about people even more.

Additionally, Brian and I are mourning all those hurt or killed while traveling on Amtrak train 188. For Brandon and for all those affected, may Psalm 34:18 speak to all those invovled:

"The Lord is close to the brokenhearted and saves those who are crushed in spirit."  

Thursday, May 14, 2015

CF Advocacy: Meeting with TX Medicaid Officials

In January, I was asked by the Cystic Fibrosis Foundation's Public Policy department (in Bethesda, MD) to join them (in Texas) as a CF parent-advocate at a meeting in Austin with Texas state Medicaid Officials, along with several CF doctors and CF clinicians from around the state.  I always jump on a chance to to advocate for  those with CF, so I was delighted to come!

About 50% of children with Cystic Fibrosis and about 25% of adults with CF in the US are on Medicaid.  So, it's very important that those making decisions in Medicaid understand the disease, how daunting it is to just stay well and how important it is to have access to a Cystic Fibrosis Care Center.

Our lobbyist, Laura, explained at one point, "When you get cancer, everyone knows that you need to get to an accredited cancer center, like M.D. Anderson, as soon as you can. What's lesser known is when you have CF, you need to be cared for at a Cystic Fibrosis Care Center."

The CF Foundation is aware that with changing health care laws and the rise in health care cots, there is a genuine concern, for those with CF, that insurance plans will not cover their medications, exclude their CF doctors from being in-network or throw challenges in their way such as requiring prior authorizations or refusing medications without the patient trying a generic med first.

This is why the CF Foundation set up this meeting in Texas.  They want to Medicaid to really understand the impact their decisions may have on our CF community.

CF is a very expensive disease.  But, we explained, CF is can be less expensive if we can catch bacteria before they colonize in a patient's lungs or if we can give medications quickly, before it becomes a lung exacerbation.

Our group, which included a Cystic Fibrosis Foundation representative, a Texas lobbyist who works on behalf of the Foundation (who does things like provides local and state contacts and serves as an local expert on state health laws, issues, etc.), a CF doctor, a social worker, and nurse coordinator from Texas Children's Hospital in Houston, a CF doctor from Dell Children's Hospital in Austin, a CF doctor from San Antonio and myself.

For about an hour, we explained the disease, shared why patients require so much medical care and walked through the kinds of challenges patients run into in an effort to keep themselves out of the hospital.

At one point, I was briefly given the floor to explain what Bennett goes through each day.  It was an empowering moment - to tell my story to someone who can help.

I shared about Bennett's struggles from birth and interspersed it with his daily life and care. The room was quite.  I could tell that people were intrigued.  While I shared, I no longer saw each person as a position of power, but, instead, a mom, a dad, grandfather.  The interest, surprise and concern on their faces made them real.  And I hoped that my son's story also too made CF real.  One official waited until I finished and then she asked, "how old is he again?"  I answered: five.  There was a pregnant pause.  It's hard for anyone to hear how much a five year old has to go through, and that's when he's well.

I don't know what, yet, will come from our meeting.  But, I know, on a whole, our group all felt very heard.  There was a desire from Medicaid to add more CF Centers in parts of Texas that do not have them.  I was very glad to hear such a genuine concern for patients who don't have reasonable access to a CF Center in their area (in some parts of Texas, families much drive 6 hours or even fly) to get care - and that care can require monthly doctor visits.

This interest from Medicaid to add more CF Centers prompted a discussion on what actually goes in to a CF Center.  One CF doctor explained that the CF Foundation accredits CF Care Centers and requires more than 50 patients, a CF doc who specializes in CF, a dietitian and respiratory therapist.  Also, the Center must have a lab who can provide necessary CF lab work and provide sweat tests for official diagnosis.  At the time, I had not realized just how much goes in to making a CFF-accredited CF Clinic.

And then, we talked briefly about reimbursements for doctors.  I did not realize how little reimbursement CF doctors get and how much support they must receive from the CF Clinic's hospital in order to serve their CF patients.  Several times, I heard a CF doctor explain, "I don't do it for the money, I do it because I love it."

After our meeting, we had lunch before everyone hit the road to headed back home.  I made sure to thank the CF clinicians and tell them, oh behalf of all of us CF families: we are incredibly grateful for them.  I feel this way about the Foundation, as well - which is not an entity, really.  The CF Foundation is a group of people who get paid to support families with those with CF.

Tuesday, April 21, 2015

The Way CFers Measure Health Status - aka, how is Bennett's CF these days?

Bennett had his 3-month Cystic Fibrosis Clinic appointment last week.  So, I thought I'd share a little bit about how that went and how that tells us how Bennett is doing with regard to his Cystic Fibrosis health.

There are five primary measurements that the Cystic Fibrosis Clinic clinicians typically use during a regular CF Clinic appointment to determine how a CF patient is doing.

These measurements include:

1.) Oximeter Reading
2.) Weight gain/loss
3.) Pulmonary Function Tests/PFTs
4.) Throat Culture Results
5.) Lung sounds

Each time we go to the CF Clinic (which is every three months when Bennett is healthy), Bennett has to go through a round of invasive testing to help the doctors determine whether or not he may be getting sick.  

One of the first tests Bennett must undergo are evaluation of his vitals.  Bennett's fever is taken (to indicate current infection/virus) as well as his blood pressure.  Bennett submits one of his little fingers for testing with an oximeter.  An oximeter measures the amount of oxygen in his blood.  Typically we want to see 100% but anything over 94% is best.  Bennett's last oximeter rating was 99%.

The second test often used to monitor CF weight loss/gain.  Due to malabsorption issues in CF, Bennett's lack of weight gain has been an ongoing problem.  At our last appointment, the doctor, dietitian and I decided to change his enzymes, increase his tube feeds and increase the amount of calories in his formula.  These changes look like they are benefiting him.  Bennett gained 2 pounds since our last CF appointment 3 months ago so this shows Bennett is doing well!

The third test is called a Pulmonary Function Test or PFT (see video above).  Children under 5 sometimes do PFTs for practice but CF patients under 5 typically do not do PFTs because they require good technique to provide an accurate reading.  It requires taking a breath and then, as fast as you can, pushing out as much air from your lungs as possible.  Our clinicians say a PFT FEV1 of anything over 80% is good.  So, Bennett's blowing 90% for his first real time is excellent.  He will get better at the technique in the coming years.  Bennett's PFTs showed us his lungs are working at full capacity! Woo-hoo!!

A fourth test we do at each CF Clinic visit is called a "throat culture."  This is when the respiratory therapist or nurse puts really long cotton swabs against the back of Bennett's throat in order to get a culture of any bacteria that might be in his throat.  This throat culture is sent to a lab.  It takes several days to get the throat culture back but we typically learn within a week.  Bennett showed no new bacteria or infection in his lungs (outside of typical Staph, which is normal in CF).  SUCH A BLESSING!!!  

The last test that Bennett often undergoes at his regular quarterly CF Clinic visit is a listen of his lungs by the CF Pulmonologist Doctor.  Our pulmonologist said Bennett's lungs sound great so we are not worried about any infections at this time.  

We go back to Dallas to the CF Clinic in July where we will go through all of these tests again.  But, for now, we are celebrating the small blessings of weight gain, clean throat cultures and good PFTs!

Like a seismograph to an earthquake, these tests indicate the beginnings of a lung infection and the progressive nature of CF.  So, every single good test result is a blessing. Every. single. one.

There are other measurements that tell us how Bennett is doing - such as chest X-rays, CT scans, bloodwork and his own feelings about how his body feels.  But, these are our main indications of how things are going when everything else is looking good.

The question I often get is, "How is Bennett doing these days?"  And my answer is: We've made it to 5 years and 7 months with no lung infections.  So, he continues to do amazing!

Tremendously thankful for today,

Monday, April 13, 2015

Seriously, how close are we to a cure? CFF State of the Science Update 2015

Several weeks ago, I attended the Cystic Fibrosis Foundation's Volunteer Leadership Conference in Virginia. 

One of my very favorite parts of VLC is the State of the Science because it is when the CF Foundation provides their annual science update.  I wanted to share what I learned there (feel free to watch the entire conference here:  

There was a lot packed in to a small amount of time.  But I narrowed down the most important parts of this update to four main points of interest:

1.) Kalydeco Update
2.) An update on the Combo Drug (hopefully to be out this year)
3.) VX-661
4.) 2nd Generation Modulators
5.) Stem Cell Biology/Gene editing
6.) Personalized/Precision Medicine

The "State of Science" at VLC was presented by (from L to R) the Cystic Fibrosis Foundation's CEO Bob Beall, Preston Campbell, the CFF's Executive Vice President for Medical Affairs, Michael Boyle, Director of Adult CF Programs/Associate Professor of Medicine at John Hopkins Hospital and Bill Skatch, the CFF's Vice President for Research Affairs.

This picture demonstrates the five ways the CFTR mutated genes work incorrectly.  At the bottom, it shows how many people have that particular gene.

Bennett's F508del gene is a Class II (87% of those with CF have at least one gene in this group). Bennett's 621+1G>T gene is a Class I (12% of those with CF who have at least one mutation in this class).

There are two ways that scientists are working to help fix the underlying reason for Cystic Fibrosis. These two ways or types of medicines are called "potentiators" and "correctors."  This explanation is super important to understand in order to follow the rest of the Scientific Update.

For those types of CF mutations where the protein is already sitting up at the surface, ready to turn on or ready to work but doesn't work correctly, "potentiators" are considered most helpful.  "Potentiators" are what we call medicines that can open that channel up or potentiate the channel because the channels are already there, they just need help with opening that gate up.   Kalydeco is a potentiator drug.

The more challenging problem in CF, but the one that scientists are making the most progress on, are those genes that benefit from medicines called "correctors."  "Correctors" are those medications that seem to benefit those with CF mutations in Class II - where the protein has not even made it up to the surface yet.  In order to fix this mutation, the protein needs to be moved up to the surface.  The reason it's called a "corrector" is because it corrects the folding so the cell allows it to get to the surface and work.  Lumacaftor is the first corrector drug.  It is believed to benefit those with F508del/F508del mutations.

KALYDECO UPATE: Kalydeco (also known as Ivacaftor) looks like it benefits those with CF mutated genes in Classes III, IV and V.  Another 20 mutations are just about to start being tested in clinicial trials for benefits of Kalydeco.  Kalydeco alone may benefit about 15% of those with CF.

COMBO UPDATE: There is excitement in the CF community because in a few months, the FDA will decide approval on the combination of Ivacaftor (Kalydeco) and Lumacaftor for those with two F508del mutations. Data shows that patients clearly benefit from the Combo drug (although, this drug is not strong enough to fix the condition for those with one delF508 mutation).  If approved, those with the most common CF genetic mutation would take a pill twice a day.  The FDA has set the date for May 12th to meet about the Ivacaftor/Lumacaftor combo.

VX-661 UPDATE: For those, like Bennett, who are not expected to benefit from Kalydeco or Lumacaftor, there is another set of CFTR Corrector, VX-661, that is hugely promising.

I learned during this talk that VX-661 is a drug that the CFF accelerated because of its potential.  Apparently VX-661 wasn't supposed to be in clinics until 2017 or 2018 but the CFF saw potential for this drug and decided to put in 25 million dollars towards getting this to patients faster.

Currently in clinical trials, VX-661 looks like Lumacaftor but works better, has less drug interactions and has a better safety profile overall.

This is a drug Bennett should benefit from. So, I'm very hopeful! They did not give us an idea of when it would be on the market but I do know they are in Phase III clinical trials so happening quickly!

Studies show that the benefit from VX-661 is even better Lumacaftor and may work with those with just one F508del mutation.  So, it could benefit up to 87% of those with Cystic Fibrosis.

Here are the current upcoming studies for those with one F508del mutations.  There is a study for those with two F508del mutations, a study for those with F508del and a Class I mutation (stop codons), a study for those with F508del with a Class V mutation and a study for those with F508del and Class IV mutation.

That covers about 90%+ with Cystic Fibrosis.  I am so very excited!  I have looked in to getting Bennett in one of these studies but, from what I found, these studies are happening in children and adults over the age of 12.  

2ND GENERATION MODULATORS UPDATE:  I'm not just excited for VX-661 for Bennett.  I'm also very excited about 2nd Generation Modulators.

This is a part of the Update that literally went so fast I neither understood it nor valued it.  In fact, I have had to spend several hours rewatching video from VLC over and over again so I could type what Dr. Skatch shared during this few minutes on stage.  This is amazing stuff but it's complicated.  So, as not to mess it up, I'm just going to write what he said had you listened to him speak.

Here is 2nd Modulators explained by Dr. Bill Skatch (pretty much verbatim):

"It's very clear that alot of effort has been put in to CFTR.  And the patients that have these mutations are going to have tremendous benefit.  But some of these things are going to take time and certain mutations are not going to benefit from these correctors. So to reach that 100%, there are several paths we are taking.  One path is to look at other types of treatments or channels.

To explain, you have nice cilia beating and mucus is moving along in the lungs of a healthy patient. But in a CF patient, where there is a defect, that water layer shrinks so cilia can't beat and mucus piles up up.

One of the strategies is to think: are there other ways we can improve that water layer?  Can we restore moisture to the lung that doesn't require CFTR modulator compounds?

So, the CFF is working on several projects that are designed specifically to target for other proteins, not just CFTR.  One of those proteins is a sodium channel.  

As you know, chloride moves through CFTR.  But sodium moves through another channel, that you can consider to be a partner of CFTR.  And it turns out, one of the reasons that water layer shrinks down and collapse is because the sodium channel absorbs too much sodium.

So we are working several companies to develop molecules that will specifically target the sodium channel (it's called ENaC).  If we can shut down that channel, there is good evidence, in the laboratory, that the water layer can be restored. We believe that that will help cilia beat better and help move that mucus up.

The nice thing about this is: it doesn't matter what your CFTR mutation is.  This type of therapy has the potential to work in all CF patients - because all CF patients share that problem with same dehydrated mucus and this very thin layer of water.

So the CFF is working with several companies such as Parion that have very exciting new molecules that can specifically attack the sodium channel."

"The corrector molecules were once a crazy idea - to take a broken protein, that doesn't move through the cell, to repair its folding, get it to the surface and then turn it on.  That seemed really impossible 15 years ago.  But now, in the test tube/laboratory, this 2nd corrector molecule is showing that is as good as Kalydeco.

What's happening now is there is a very large effort, in terms of 2nd generation screening.  There are multiple companies looking at new and better chemical compounds.  Millions of compounds were screened last year.  We are looking at very diverse compounds.  We are thinking, the more we look, the better chance we have to finding the best molecule.

We also got much smarter, in terms of how we are screening.  We are using human airway cells to try to screen how these molecules work.  We are targeting these molecules to specific parts the CFTR protein, because we now have an idea of how the protein is broken.

Our goal is develop modulators - correctors - that will be as good as Ivacaftor in the F508del patient population.

What does this look like?  We are thinking, as we move in to the future, we are probably going to need, for many of the folding mutations, two correctors plus a potentiator - a three drug combination.

That will hopefully to get us where we need to be.  This will hopefully open the door for a large number of CF patients."

"If everything goes perfectly (15% of potentiators + 50% F508del homozygotes + 40% F508del Heterozygotes), that covers about 95% of those CF patients.  But it leaves 5% of CF patients that don't make CFTR that can be potentiated.  

This 5% is very complicated - nonsense, stop-codon, splicing variants, deletion variants, etc.  These are going to require a completely different type of therapy. One way to go after these is to develop therapies, therapies that are applicable to all patients (such as the 2nd generation modulator idea). Another way is we can target these mutations very specifically.

If we look ahead, what do we want to do?  The goal is to take the therapies we have and make them better until we have a cure for all patients."

"We know what the problem is.  The problem is in the gene, the DNA.  It's a mutation, small deletion, which is altered.  That mutation is translated into RNA. And the RNA in the cell is a template from the DNA.

So, if you have a mutation in your DNA, that mutation is then translated in to the next step of the biological problem, which is RNA.

RNA is used to make protein.  So the defect of the the RNA is then translated in to the protein defect. And the protein, if it's CFTR, is what causes symptoms.  So, this paradigm is very basic in biology.

For the past 50 years, the CFF has been working on the symptoms - better therapies for the the mucus, better therapies for the infection, better therapies for nutrition.  This has had a tremendous burden on the patient in terms of the amount of effort and time.  But it was had tremendous affect on  lifestyle and lifespan and making life better.

We are now entering in a very early stage, of moving backward along a path to get to the more basic defect.

When we talk about correctors and modulators, we are talking about protein-based therapy. Those molecules work on patients that have specific types of protein abnormalities.  But some mutations don't allow the protein to be made.  In some patients, they don't make the so modulator therapy will never work.  Those patients need different types of therapies.  One of those therapies is to move back on more layer is the RNA.

That RNA molecule may be manipulated in a couple of ways.  One, in a project we are working with Shire, is to simply take normal RNA and put it back to patients.  If we can take normal RNA in to cells, then those cells will make normal protein and that normal CFTR will function in any patient.

So there is a lot of challenges for this.  But we are very encouraged by the results of Shire.  Shire is moving very quickly. We hope to have RNA therapy coming in to patients within a year to two, maybe by the end of this year, but hopefully in 2016.  We just committed 15 million dollars to that."

STEM CELL BIOLOGY/GENE EDITING:: "The CFF is also working on ways of addressing stop codon mutations, specifically.  The CFF recently put in 6.7 million dollars into a company called PTC to try to develop a molecule that will help the ribosome read through to those stop codons to make the protein.

It was unfortunate that that molecule did not work as well as had hoped.  But have not given up. We are going back and starting several collaborations with new companies to look for better and more effective molecules that will work on these stop codons and nonsense mutations, specifically, to allow the protein to be made, which then can be potentially augmented by potentiators and potentially correctors, if the protein doesn't fold correctly.

How about the one time cure? If we could go back to the DNA...if we could prepare the DNA in the cells that need to make CFTR, then those cells would make normal CFTR and all of this process would be completely normal.

Five years ago, this was an absolutely impossible dream. But what has happened in the last five years is there is some very exciting technology, where you can go into the cell and highly specifically target regions of the protein, regions of the gene, that are defective and repair those defects.  This is called gene editing.

There are a couple of ways to go back about this.  There are a lot of technical difficulties to make this work.  But this is tremendously powerful and really provides, for the first time, the ability to go into the cell and remove the CFTR defect.  Instead of treating the disease, this is essentially removing the disease.

One way this might work is to take cells out of the lungs of patients, identify which cells are the critical cells needed to restore the airway, go in to those cells and edit the CFTR gene so that now it makes that normal protein and put those cells back in to the lungs of patients - have those cells grow, reproduce and populate the airways, then it would be possible to basically eliminate the need for correctors, potentiators or any other kind of therapy

Just last week, we brought 30 investigators who are experts in stem cell biology to the CFF.  We had a fantastic discussion about what cells we need to target, how to the cells behave, how do we grow them so they can actually graft in lungs etc.

I will say there are many obstacles but it was a very encouraging discussion. There are a group of scientists out there that are really excited to work on this problem.

In December, we invited a group of scientists to the Foundation to talk about gene editing.  We are bringing those people into the CF field.

This is where we would like to go.  This is a long project. There are many obstacles. So, we see this as a long term goal but as a way to totally transforming the disease.

As we look forward, we are looking personalized medicine or precision medicine.

One of the key roles is the maximize the function of CFTR.  This means we take the patients particular mutation, their genotype, and we target therapies that will maximally allow that genotype to function.

We want to combine the best types symptomatic therapies with the best type of genetic therapies (protein-restoration therapies).

Many patients today are going to continue to need symptomatic therapies.  We are going to continue to work on better therapies.

We hope as we bring these new types of therapies to younger patients in children and restore CFTR function, then those individuals will never need any type of symptomatic therapies.  This is our overall goal."

This was a fantastic update.  Here's the bottom line: My heart is overwhelmed with hope.  For most of those with CF, we are SO close!!  And no one is going to be left behind.

Monday, April 6, 2015

The CF Care Center Accreditation Process

I recently had a chance to learn more about the Cystic Fibrosis Foundation's CF Care Center Accreditation Process, the process that makes sure the care that Bennett is getting at his quarterly CF Clinic appointments is at or above standard, as a part of the work I've been doing with the CF Care Model Redesign and during my trip to the CF Foundation's Volunteer Leadership Conference.

I found learning about this CF Care Center Accreditation process to be incredibly valuable to me as a CF caregiver.  The more I know about what goes in to Bennett's care, the better I can be about paying attention to whether or not he's getting the best of care.

I know other CF families feel the same way.  So, I wanted to share my learning with other CF patients and families who might be interested in the nuts and bolts of how a CF Care Center is accredited.

This information came from a presentation by Bruce Marshall, director of clinical affairs of the Cystic Fibrosis Foundation.  Feel free to watch the webinar presentation yourself here:  I'd love to hear comments at the end of this post as we continue to look for ways to improve it!

So, what is a "CF Care Center" and why it is important?
CF Care Centers are medical clinics within a local hospital partially funded by the CF Foundation that operates multi-disciplinary care for those with Cystic Fibrosis.  There are more than 110 cystic fibrosis care centers and 55 affiliate programs nationwide, including 96 programs for treating adults with CF. (Affiliate programs are smaller CF Care Centers who report to a larger CF Care Center.)

Typically, a CF Care Center includes at least one CF doctor (typically either a pulmonologist or pediatrician with CF training), a nurse, a respiratory therapist, dietitian and a social worker.  Those who work at the CF Care Center are considered part of the CF Care Team.

According to the CF Foundation, "this high quality of specialized care available throughout the [CF Care Center network] has led to the improved length and quality of life for people with CF."

Slide is from presentation from the CF Foundation's Volunteer Leadership Conference 2015.

The History of the CF Care Center Model:
CF Care Center Accreditation started in 1961 with the accreditation of two centers.  This Accreditation took place only 6 years after the Cystic Fibrosis Foundation was formed in 1955.  CF Care Center Accreditation was one of the first initiatives of the Foundation as a way to prolong survival.

In the 1960s, 1970s and 1980s, there was rapid growth of accredited CF Care Centers, mainly in pediatric programs as CF was primarily a pediatric disease.  In the 1980s, however, adult programs really begin to grow.  In the mid to late 90's, adult model programs were evaluated.

In 2000, the CF Foundation began mandating that all CF Care Centers provide adult programs.  These adult programs ranged in a spectrum from CF Care Centers providing adult-oriented CF physicians but sharing the pediatric CF clinic's multidisciplinary team all the way to true CF Adult Care Models, which included a separate and distinct adult CF Team.

In the last year or two, the last pediatric-adult shared programs have separated so that now all adults with CF at every CFF Accredited Center have access to their own adult CF Team.  Now in the US, we now have a mature pediatric and adult care model for all of those with Cystic Fibrosis.

The Accreditation Structure
There are 4 explicit standards that makes a CF Care Center an approved Cystic Fibrosis Foundation CF Care Center.  They are:

Physician Leadership - properly credentialed physicians and physicians experienced in CF care
A multidisciplinary team - such as a respiratory therapist, dietitian, social worker.  Other team members can include a pharmacist, physical therapist, psychologist, chaplain, a patient advocate, etc.
Participation of the CF Clinic to participate in the patient registry, an IRB patient-consented observational study that is used to evaluate much of CF care.
Meeting of clinical teaching and research requirements

The CF Care Center Accreditation CommitteeThese four standards are enforced by a CF Care Center Committee, made up of 9 pediatric and 9 adult CF Care Center Directors, elected representatives from pediatric, adult and affiliate programs. Committee members serve two year, renewable terms.

The CF Care Center Committee's Mission Statement is "to support the CF Foundation Mission by fostering exemplary care of all individuals with CF through: promotion of standards of care, accreditation of care centers, education of providers and advancement of research in all aspects of CF."

The Committee meets twice per year, typically in May and in December for 1.5 to 2 days each time. During these meetings, the Committee evaluates those CF Centers visited during the previous months.  All CF Care Centers are visited by the Committee and are evaluated for accreditation every 3-5 years.

The Accreditation Process
There are two core processes of the Accreditation Process.
They are:
1.) site visits are made by Center Committee members and
2.) annual updates submitted by centers.

The Site Visit Process
When a CF Care Center is visited by the CF Care Center Accreditation Committee, the in-person visit typically last a day or day and a half.  Typically there are two visitors who attend the site visit (usually a pediatric and an adult CF doctor).

During this meeting, the Committee representatives meet with institutional leaders (aka the CF Clinic director, the hospital directors, etc.).  The Committee does a comprehensive assessment of the center.  They look at the clinic, the personnel, the facilities, key labs to CF care (such as the sweat test and microbiology labs).  They do a chart audit and hear report outs of teaching and research, as well as quality improvement.  Sometimes, but not always, the Committee members will meet with the CF Patient Advisory Board.

Once the site visit is complete, the Committee members will get together to share and review their Site Visit findings.  During that meeting, the Committee will go through all centers considered in that 6-month cycle.  A decision is made on accreditation status of each Clinic evaluated.

Accreditation possibilities include:
* Accredit
* Accredit with contingencies (pending immediate action)
* Accredit with provisional status
* Disaccredit

Once a decision is made, critiques based on the site visit are written down and are sent in a letter form back to the CF Care Center, at which time the CF Care Center is notified of their accreditation status.

After revealing the accreditation status of each center, the Committee requests a response from the CF Center.  CF Centers are given a short amount of time to respond via letter regarding the Committee's findings. (aka, if the Committee states the CF Care Center is losing their accreditation, the Committee lists in the letter why the CF Clinic is losing their accreditation.  The CF Clinic then has some time to write a letter back to the Committee stating their challenges and how the Committee can expect the CF Clinic to respond.

Annual Updates
Since not all CF Clinics are visited yearly, there is an important aspect of accreditation that continues. This is done through "Annual updates." Annual updates are sort of like progress reports from the CF Center to the CF Foundation each year.  Questions are asked regarding the CF Clinic's strengths, weaknesses and challenges.

If the CF Foundation sees a problem with one of these annual updates, the institution will be flagged for an early site visit and the Center Committee is notified.  If there are no problems, the CFF will provide accreditation approval and they stay on their 3-5 year cycle of Accreditation.

So, once a CF Center is Accredited, then what?
So, center sites visited in the previous year are eligible for the "Quality Care Award" based on evidence of "sustained quality improvement resulting in improved outcomes.  The CF Foundation provides recognition of the Center/s who receive this award at the NACF Conference. 

Benefits of Accreditation
The benefits of a CF Care Center being accredited includes:
* Accountability - ensures standards are met

* Ideas for improvement - host centers and site visitors benefit from visits to other CF Care Centers
* Securing resources from institution - when a CF Care Center must meet requirements set forth by the accreditation process, this engaged the institution to support the CF Care Center in meeting these requirements for fear that otherwise, the CF Care Center will not longer be accredited (something that the host institution such as University or hospital would not want to see is the CF Care Center brings patients to its facilities)/
* Public recognition of excellent performers - CF Care Centers can be the pride and joy for awards and their institutions.  They can be model for all other centers.

Here's an example of how well CF Care Centers work for CF patients.  The picture below shows data from CF Care Centers with regard to their patient's FEV1 vs BMI percentile.  In 2002, CF Care Centers were scattered in all four quadtrants.  Some were doing amazing in one area or in the other.  Some were doing amazing in both.  Others were not.  But, as CF Care Centers worked to find solutions, they began to figure out better ways of caring for CF patients.  In 2012, the same data shows that most CF Care Centers were amazing in both areas.  This is the power of working together and fighting for a goal of better health outcomes for CF patients:

Current New Initiatives for Care Center Accreditation
The CF Foundation recently indicated there are several new initiatives for Care Center Accreditation.  They include:
* The Patient and Member Experience Care Survey - already 107 (about 40%) of the 265 CF Care Center programs have participated
* Revising accreditation criteria and bolstering struggling centers (such as allowing for independent pediatric and adult programs - as long as transition in place, relaxing the teaching criteria and allowing referrals for clinical trials to count towards research criteria
* Facilitation greater CF Foundation and core center oversight of affiliates

Slide is from presentation from the CF Foundation's Volunteer Leadership Conference 2015.

New Program for Affiliate CF Care Centers
To facilitate great CF Foundation and Core Center oversight of affiliates, the CF Foundation has recently begun a program called the Targeted Assistance Program (TAP).  This program provides:
* data-driven approach to selection (registry data, experience of care survey data, site visit reports and annual updates, staffing levels)
* intervention team comprised of peer clinician
* reporting to center committee
* improvement plan with timetable
* investment of resources of CF Foundation and the institution

In the slide below, you can see how CF Care Centers are doing in their measurement of patients (6-17 years old) with their FEV1.  Most CF Care Centers (represented by black dots) fall within the mean (represented by the gold line).  However, a few CF Care Centers are doing much better than the mean (see the green dots) and some much worse than the mean (see the red dots).  Note that 5 of the 6 red dots are those CF Care Centers who are CF Care Center affiliates.  This is just one of the reasons why the CF Foundation has put in to place the Targeted Assistance Program.  Nobody wants to go to a CF Care Center well below the mean.

Slide is from presentation from the CF Foundation's Volunteer Leadership Conference 2015.
This blogpost packs in a TON of information.  But I wanted to blog about this process for two reasons.  One, I wanted to blog because this information - information about the CF Care Center Accreditation Process is hard to find.  The CF Foundation's website does not list much, if any, of this information.  I wanted to share it with other CF families.  The second reason I wanted to share is because, as a part of my work with the CF Care Center Redesign, I have realized that while there are SO many wonderful processes in place to help our CF patients get and stay well, there are many aspects of CF care that need to be improved. The Accreditation Process is one of those.

Here are the things I'd like to see about the Accreditation Process improved:
1.) I'd like CF patients/caregivers involved in the accrediting our CF Care Centers.  The patient/caregiver voice is vital to improving CF care.  We need structured and integrated input from CF patients/caregivers when choosing to accredit CF Care Centers.

2.) I'd like more transparency about our CF Care Centers accreditation process.  When CF Care Centers lose their accreditation, it is simply taken off the website.  Passive communication about a CF Center's accreditation or lack thereof cannot help CF patients make informed decisions.  As much as I absolutely *loved* learning about the ins and outs of the CF Care Center process, I was disappointed that I had to be involved knee-deep in a project that the CF Foundation is supporting before I could get access to this extremely beneficial information.  I want information like this to be more accessible for all CF patients/caregivers, not just those involved in particular projects with the Foundation.

3.) Not only do I want to know how my CF Center is doing in the accreditation process, I'd like to know how other CF Care Centers are doing with their accreditation. The CF Foundation gives out Quality Care Awards at the NACFC.  But, the problem with NACFC is it's nearly closed to CF patients and caregivers attending.  So, I would like to know who is winning awards, why and how. Knowing my clinic or other clinics are doing well only helps me better understand the kind of care my child might be receiving at their CF Care Center.

3.) I appreciate the current Accreditation Process, but I'd like a Process that pushes my CF Care Center to go beyond minimal standards.  I am incredibly grateful for the process we currently have in place.  The CFF Accreditation Process helps to assure Bennett is at a CF Care Center that, at minimal, is up to CF standards in his care.  But I want more for Bennett and for all those with CF.  At this point, there is no direct way for CF patients/caregivers to know how their CF Care Center is doing compared to others around the country.   I'd like to see the Accreditation Process adopt some type of higher recognition of CF Care Centers that are providing care beyond the minimal accreditation standards.

Sunday, April 5, 2015

Easter 2015

"Hoppy" Easter from our little bunnies to yours!

The Texas Bluebonnets are blooming this week.  So, we decided to make our Easter pictures our Bluebonnet pictures, as well!  

While taking pictures of the children playing among gorgeous wild flowers, I couldn't help but feel God's closeness and recognize his faithfulness.  

Every Spring, God sends us flowers.  Every morning, a sunrise.  (Max Lucado)

“Because children have abounding vitality, because they are in spirit fierce and free, therefore they want things repeated and unchanged. They always say, "Do it again"; and the grown-up person does it again until he is nearly dead. 

For grown-up people are not strong enough to exult in monotony.  
But perhaps God is strong enough to exult in monotony.

"It is possible that God says every morning, "Do it again" to the sun; and every evening, "Do it again" to the moon. It may not be automatic necessity that makes all [bluebonnets] alike; it may be that God makes every [bluebonnet] separately, but has never got tired of making them. It may be that He has the eternal appetite of infancy; for we have sinned and grown old, and our Father is younger than we.” 
-- G. K. Chesteron, Orthodoxy

Oliver, 7 years old; Avonlea, 21 months old; Bennett, 5 years old

Saturday, March 28, 2015

"...until there is a cure."

I won't be able to find a cure for Bennett alone.

This is why it is so incredibly meaningful when a friend shares Bennett's video on Facebook, or when a friend retweets Bennett's story on Twitter, or wears our Bennett's Brigade t-shirt, or walks (even leads) a Great Strides Walk or gives a donation to the CF Foundation in honor of Bennett.

Just last night, my friend Jenny was on Facebook asking to purchase a Bennett's Brigade t-shirt for her son to wear at the St. Charles, Missouri Great Strides Walk.  When I double-checked on the size t-shirt she had ordered for her little one, she explained that she was buying a size up for Alex to "last him a couple of years" because, she said, "we will do this until there is a cure."

I cried.

With those 9 little words, Jenny reminded me how I am not alone in this.  Not now.  Not later.

As Bennett's mama, I know I will do this year and after year.  I will walk.  I will ask for donations.  I will share his story.  But always lingers a little fear inside me: "Will this year be different? Will anybody come?  Will anybody give?  Will anybody care?"

Each year, I fear I am alone in this.  And yet, each year, I am gracefully shown: I am not.

The Great Strides for a Cure for CF Walks that Bennett's Brigades participate in all around the country are about hope.

Our Walks are celebrations of the money raised, to date, for a cure - and celebrations of the support and love that surrounds Bennett as he fights this terrible disease.  I am so touched that there are friends who I haven't seen in more than 15 years who are leading Walks in their areas, asking for participating and donations from their friends for my son.

This weekend we have to finalize our order for our Bennett's Brigade tshirts.  If you haven't already gotten one and want to, please email me at breckgamel at by Monday, 3/30.  It's hard to believe Great Strides 2015 is here!!

For anyone interested in walking or donating in honor of Bennett at any of our CF Great Strides for a Cure Walks across the country, please sign up or donate using the links below:

Thank you to the many of you who remind Brian and my family that we are not alone in our battle with Cystic Fibrosis and our efforts to find our son a cure.  It is the consistent love and support that brings us hope in the midst of despair!

We are so honored to have you join with us in this fight!!  We're #InItForBennett!!

Wednesday, March 25, 2015

Advocating on Capitol Hill for Bennett

This past week, I traveled with all three kids to DC to advocate on Capitol Hill for Bennett and all those with Cystic Fibrosis  (Brian had to stay back to work).  This year, I took Oliver to advocate with me.

This was my third year to participate in the Cystic Fibrosis Foundation's annual "March on the Hill" day in Washington DC.  It is a special day where the CF Foundation arranges meetings with Congressmen and their staff so CF caregivers, friends and family can communicate our need for a cure.  

Those with Cystic Fibrosis are not allowed to come to CF events such as this one, due to infection control issues.  The CF Foundation recommends that no two people with CF be within 6 feet of each other as they can inadvertently get each other sick.  So, while Bennett couldn't be there.  Oliver was there for him instead.  (My mom watched Avonlea and Bennett for the day.)

After a morning of training, more than 100 CF advocates emerged on Capitol Hill to speak on behalf of those with the disease.  One of my first appointments was with Congressman Barton (who happens to share a birthday with me - random!).  I attended this meeting alongside the Northeast Chapter of the CF Foundation's Executive Director, Amy, and Debbie, also from Dallas, whose sister died from CF in 1979.

I just had to mention this sweet lady, Krista (in red), Congressman Barton's Counsel and Legislative Director because she was absolutely fantastic.  She knew so much about CF before we ever came, because she studied up on it.  To my surprise, Krista had already found Bennett's video and was incredibly up to date with the newest information about CF as she had recently attended a meeting put on by the CF Foundation for Congressmen and their staff.  Krista was such a delight to meet, making our first Congressional meeting a really great one!  

As CF advocates, we had several requests when we met with the Congressmen and their staff.  First, we asked them to support the EACT (Ensuring Access to Clinical Trials) which makes permanent that those with rare diseases who participate in clinical trials not have to count as income up to $2,000 in compensation which they receive for participating in those clinical trial.  Doing so has previously jeopardized the health insurance and social security disability benefits of those with rare diseases like Cystic Fibrosis.

This is particularly important issue for those with CF as 1 out of every 2 children with CF and 1 out of every 3 adults with CF are on Medicaid and/or Social Security Disability.  It would be very sad if patients had to face the choice of participating in clinical trials, which helps the entire CF community, OR losing their health insurance because they made too much money due to clinical trial compensation.

Cystic Fibrosis is an orphan disease.  That means the number of CF patients in the US is small. Those who qualify for clinical trials for CF drugs is even smaller.  I'd hate to see that even fewer patients would be available to help us test a cure.

The good thing is the EACT has been the law for the last few years but it had a "sunset" clause on it, which meant it expired at some point.  Now, there is an effort to make EACT in to a law permanently.  It's a bipartisan effort and hopefully should pass.

On our second visit of the day, our team met with  Congressman Pete Sessions.  Congressman Sessions was very playful and seemed to really enjoy having Oliver in his office (as seen by the picture above).  Sessions shared he is also a caregiver of a child with special needs as he has a son with Down Syndrome.  It was nice to be able to see Congressman Sessions as a father and caregiver, just as I hope he saw us as desperate parents wanting to make way for a cure.

Outside of asking for the Congressmen to support the EACT, we asked for robust funding for the National Institutes of Health (NIH) and FDA.  Both government departments are crucial to the Cystic Fibrosis Foundation's research for a cure.  The CF Foundation uses basic human research, supported by the NIH, to build upon our own research.  Once a medication is found, the CF Foundation works diligently and swiftly with the FDA to get the drug to market as fast as possible.  The CF Community raises it's own money for a cure but uses help from the NIH and FDA to help expedite a cure.

Another Congressman that we had the privilege to meet was Congressman Bill Flores.  I have met with Congressman Flores for the past 3 years and greatly appreciate his sincerity every time.  Oliver and I are constituents of Congressman Flores and really enjoy having created a positive relationship over the years.  Texas has around 1,744 CF patients and 16 CF Care Centers.  Scott and White, one of Bennett's hospitals, is in Congressman Flores' district.  

We enjoyed meeting with all of the Congressman.  But, Oliver's favorite Congressman is the one best known to him as "Pops."

Congressman Fleming was actually on his way to votes after our meeting with him so he invited his grandson to help him vote on the House floor - something only children get to do. I had to watch from the Gallery.  But here is where Oliver did the most for CF on Capitol Hill and didn't even realize it:

On the floor of the US House of Representatives, Oliver helped his "Pops" vote.  But, between votes, different Congressmen, delighted to see a child on the House floor, came up and spoke with him.  I watched from the Gallery as he shook hands with men and women who represented states near and far.

I knew Oliver wasn't exactly able to capture in his little mind what a cool thing he was getting to do. But I was soaking it up for him, watching from above.  I watched my little first grader speaking with the adults, sitting there in his little khaki's and button up shirt, looking around, listening to the sounds of the gavel being hit and watching the lights brighten up as votes were cast.

At one point, Oliver seemed to unknowingly catch the attention of Congresswoman Debbie Wasserman-Schultz.  This is most interesting because it seems that often the Republican Representatives and the Democratic Representatives tend to mingle together in their respective parties on the House floor during votes.  But Congressman Wasserman-Schultz is a Democrat.  Congressman Fleming is a Republican.

I watched as Congresswoman Wasserman-Schultz came over and began speaking to Oliver, who was standing beside his grandfather.  She struck up a conversation with Oliver and then with Congressman Fleming, who at some point mentioned to Congresswoman Wasserman-Schultz that Oliver was doing something important at the Capitol today - he was advocating on behalf of his little brother, who has Cystic Fibrosis.

I could see there was some sort of agreement going on between the three of them.  But I couldn't tell what.  Later, I learned that during that conversation, Wasserman-Schultz shared with Oliver and Congressman Fleming that she knew what Cystic Fibrosis was.  In fact, she revealed, she has an extended family member with the disease.  And to my surprise, she then turned to Congressman Fleming and said, "Look, I'd love to help.  I'm on Appropriations [Committee].  So, call upon me for any help."

What a powerful moment for me, as a mother, to see my son, on the floor of the US House of Representatives, connecting two legislators who came together, across party lines, in support of those with Cystic Fibrosis.  Oliver had done it.  He had gone to Washington DC, advocated for his brother, and walked away with another fighter, US Congresswoman, in our corner.  I couldn't have been more proud of him.  

Oliver's treat for such a great job: Ice Cream at the US Capitol's Creamery!

I wanted to make sure that I mentioned my friend and fellow CF mom, Rebecca, who became the CF Foundation's newest National Advocacy Chair.  Rebecca and I first new each other only via Facebook through the "CF Mamas" Facebook group.  Now we are real life friends.  It's pretty neat.  I am very proud of having Rebecca in this place.  One of my favorite things Rebecca said during the event was, "CF Advocacy is not political.  It's personal."  This is so true.

At the end of March On The Hill, the CF Foundation invited my dad, Congressman Fleming, to speak to all of the 100+ CF advocates that joined March On The Hill for a dinner.  Senator Markey (not pictured) also spoke.  After dinner, the CF advocates who represented their family members and friends on Capitol Hill shared their stories of the day.  It is always so amazing to hear how receptive our legislators are to our message:  "We are close to a cure.  Help us make that happen faster!"

I am thankful for a father who is not only supportive of his little grandson's daily fight against CF but who is a champion in the United States House of Representatives for all those with Cystic Fibrosis!

And I am thankful for a little 7 year old who advocates for his little brother, not just each day at school and at home, but also in our nations' Capitol.