31 March 2013

Easter 2013

Happy Easter!

A family photo before our church Easter Egg hunt!

Getting ready to hunt for some eggs!

Although there were 400 eggs at the park spread out for 40 children, within minutes, the bigger kids had gathered up much of the eggs.  The eggs were gobbled up so fast, our little guys didn't get many.  Fortunately, they weren't too concerned.  I think they were just satisfied to get to sit down and eat their little pieces of chocolate that they didn't even notice their baskets were near empty.

One of the most meaningful moments for me yesterday happened spontaneously while I sat on the ground with them taking pictures of their opening their eggs.  Oliver opened one of the eggs to find nothing was inside.  To my surprise, instead of being upset (which I had fully expected since he's 5), he excitedly said, "Look, Mom, nothing inside!!  It's like the cave when Jesus wasn't there!"  It was a really touching moment for me.  Oliver saw his empty egg, not as the loss of candy but as an empty tomb.  (I had no idea how powerful our annual tradition of telling the Easter Story through Resurrection Eggs would be for Oliver.  In that moment, I realized, "he gets it.")

He is Risen!  Happy Easter!

26 March 2013

VLC 2013 Science Update

{EDIT: I apologize in advance that there is a lot of technical information in this post.  I have tried to make it as easy to understand as possible.  But if I can clarify something, please let me know.  Also, if you were at the VLC meeting and heard this talk and noticed I have an error, please let me know, as well!}

I learned a ton during the Cystic Fibrosis Foundation's Volunteer Leadership Conference I recently attended.  So, I thought I'd share what I learned in several smaller blogposts rather than in one large one.  Here's what I learned...

1.) The CFF believes Kalydeco may help all of us and up to 95% of those with CF could benefit within 4 years!

Bob Beall started out his talk to our group by sharing a few really encouraging words.  One quote I loved that he said was: “our vision has never been clearer or more focused...It’s not when we get there but what’s it going to be like when we do.”

Below are several slides shown as a part of the update on the Science presentation by Dr. Preston Campbell...

This slide shows the benefit of Kalydeco (also known as "ivacaftor").  As you can see, Kalydeco shows huge improvement over the current medications available for improving lung function.

To me, this slide is probably the most impressive slide of all the Conference.  This is what the CFF predicts for the expansion and benefits of the current CFTR Modulation drugs.  Let me explain it...
2013 lists 4% of those with Cystic Fibrosis (mainly those with the mutation G551D and the like) who are currently benefiting from Kalydeco.  This number is expected to increase to about 8% by 2014 because the Cystic Fibrosis Foundation is currently looking for other mutations who might benefit from Kalydeco.  This number has the potential to go up to 65% by 2015 because it is hoped that the VX-809 drug will be available at that point and will be effective when given along with Kalydeco to those with double Delta F508 mutations.  By 2017, the CFF hopes to increase this number to 95%.  That's right, the CFF is predicting that by 2017, up to 95% of the Cystic Fibrosis population may benefit from Kalydeco as the additional 30% of the population which has Delta F508 + one other mutation will also be able see benefits from Kalydeco and VX-809.

Based on this information, I am assuming that it is expected that, by 2015, Phase III trials will end for VX-809 and it will be placed on the market...and that, for those with with Delta F508 and another mutation, they will see their medication succeed through Phase II and Phase III trials by 2017.

For Bennett, this means, there is a very real possibility that he will see a reasonably effective medication benefitting him by 2017.  SO EXCITING!!!

This slide shows The Effect of a Potentiator and Corrector on CFTR Activity in the lab.  As you can see,  the G551D mutation demonstrates less than 10% CFTR activity in vitro (this is why those who have it G551D, along with another CF mutation, have Cystic Fibrosis - CFTR activity is very important to the body).  The slide also shows, however, when given Kalydeco, those with G551D see significant improvement in the CFTR activity as the cells in the lab show CFTR activity reaches 50%.

If we look at the Delta F508 mutation, we see that VX-809 by itself provides 20% CFTR activity.  While that is some activity, it is still too low to show significant improvement in the disease.  But, if Kalydeco is added to the V-809, we see about 35% CFTR activity.  This is better, but still not the improvement that those with G551D and Kalydeco see.

The goal is that the body see 50% CFTR Activity in order not to show signs of Cystic Fibrosis.  The CFF believes that if we can give patients with Delta F508 the medications VX-809, Kalydeco and one other effective drug (here its called VRT-XXXXXXX), patients should be able to get 50% activity.  The drug VRT-XXXXXXX has not been identified yet.  This is why it is listed as this.  But the CFF is working hard to identify this drug.

The point is that we're close...but not there yet.  The Cystic Fibrosis Foundation will continue working with the pharmaceutical companies to identify the drug that should help bring CFTR activity in the Delta F508 mutation up to 50% like we see in the G551D mutations.

For those who haven't followed the big news in the world of CF, it was announced at the end of last month that Vertex is beginning Phase III trials for the combination if drugs Kalydeco and VX-809 for those who have two copies of DF508 (about 50% of the CF population).  As evidenced in the slide above, this looks very promising in the lab for those with Delta F508.  We hope that Phase III trials will meet or accede our expectations.

For those like Bennett who have have one copy of DF508 and a copy of another CF mutation (which is   40% of the Cystic Fibrosis population), Vertex is just now beginning Phase II trials on the combination drugs Kalydeco and VX-661.  This is a very important milestone for those with one Delta F508 mutation, including Bennett.

But the CFF is not stopping there.  They want to make sure all who can benefit from Kalydeco can.  The CFF is working to identify more genetic mutations who can benefit: there are already ongoing efforts to identify the next generation of DeltaF508 CFTR correctors.

In addition, the CFF is looking in to how 
Gentamicin may work for those who have "stop-codon" mutations.  They said they don't know if it will be good enough for the stop codon mutations but they are going to find out.

For those with Bennett's mutation (621+1G>T), I had originally thought that this mutation of Bennett's was a "premature stop-codon."  However, Dr. Preston Campbell did an great job helping me understand that it's, in fact, not.  Instead, it's a "missense mutation."  (Therefore, gentamicin should not work on Bennett's mutation.)

Dr. Campbell said that it is unknown whether or not 621+1G>T is responsive to Kalydeco and "testing in the laboratory will be difficult due to technical difficulties" but "they will soon sorting this out."  

Other good news I heard at the conference is that those with CF who are pancreatic sufficient may have residual CFTR function.  Not only does this mean their disease is likely less severe but it also may mean they do not need as much benefit from a drug to see significant improvement in their disease.  Although this is not Bennett's situation, it makes me very hopeful for those who are pancreatic sufficient as they may not need much in the way of effectiveness from a drug to see significant benefits.

The question remains, of course, for the 5% of those with CF who have what is called “X” mutations.  These mutations have not found their miracle drug yet.  The Cystic Fibrosis Foundation continued to say over and over and over again during the conference that they would not stop until 100% of those with Cystic Fibrosis are cured.  

Unfortunately, I did not hear of any trials for drugs to help those with these mutation as they are incredibly rare.  But that does not mean that the CFF does not know about them or doesn't want to help.  They are just more difficult and the CFF is working as quickly as a possible to get medications to help them as well.

The great thing is that the CF Foundation is it is working very hard for a cure.  Just this year alone, the CF Foundation will spend $120 million this year on its medical program (research and efforts to find a cure) which is funded mainly with private funds (most from our Great Strides efforts) in addition to the royalties the CF Foundation gets from Kalydeco.

Bob Beall shared that the Foundation has taken the money they would get from royalties from Kalydeco and already sold it to a broker (he said they simply did not want to waste time).  This broker has given this money to the Foundation which the CFF has already turned around and used to invest in research for the next generation of drugs.  Bob shared something to the effect: "we don't have time to sit around and wait for the money to build up in an account so we can reinvest it in research.  We needed to do it now."  

I have more updates to include but these might just be my most exciting of all my updates for it shows the CF Foundation is absolutely making progress and Bennett may end up benefiting from a significantly effective drug within the next 4 years!!  Yaaaaahoooo!!

17 March 2013

CFF's Volunteer Leadership Conference 2013 in Pictures

This is the first of several posts about my time at VLC 2013.  I thought I would start out with a few pictures first...

For the past two years, I've had the privilege to attend the Cystic Fibrosis Foundation's Volunteer Leadership Conference.  This year's theme "Mission:Possible."  I loved this theme because it's SO true at this point in history with the disease Cystic Fibrosis.  We are really, seriously, honestly, totally facing a POSSIBLE mission: to give every person with CF a cure.

I tried to take pictures along the weekend to document our time.  Unfortunately, Brian wasn't able to attend the conference with me this year because he had his own conference for which he was a presenter. So, this blog post is as much for him as for anybody else! :)

This year's conference was held in Reston, Virginia with about 400 people in attendance.  Those who attend this conference are hand-selected by their CFF chapters and are some of the most dedicated volunteers from their area.  I always find being invited a huge honor and one I do not take for granted.

One of my favorite things about this conference is getting to hear Bob Beall give the "State of the Foundation" address.  As a fundraiser, volunteer and beneficiary of the Cystic Fibrosis Foundation, I am always very eager to know how the Foundation is doing and its plan for the future.

I also really appreciated hearing the "Science Update" by Dr. Preston Campbell (right; Bob Beall is to the left) as I am always highly interested to know the money we raise for the CF Foundation is going to the right place.  In the next post, I will share some really exciting parts of Dr. Campbell's science update and how our hard-earned money is benefitting Bennett and those like him.

As a fun play on the "Mission:Possible" theme, my friend Paige (from Texas) and I had to dress up as secret agents.

One of the most disappointing part of this year's conference, and it's a pretty big one for those of us with family members with CF, is that this event is one of the first in which the Cystic Fibrosis Foundation decided to enforce it's new infection policy for those with Cystic Fibrosis.  Continued scientific research has demonstrated that those with CF are even more a risk to each other than we first thought.  So, the CF Foundation is on the cusp of releasing its new infection policy which will change the "3-feet-rule" to the "6-feet-rule" and will dictate that only one Cystic Fibrosis patient can be at any indoor Cystic Fibrosis Foundation function (this does *not* apply to outdoor events, such as Great Strides).  To keep Cystic Fibrosis patients involved since they cannot be physically present, the CF Foundation web-streamed the conference.

The CF Foundation shared their fear this infection policy risks isolating those with CF.  But the CFF also communicated they are concerned first and foremost for the health of those with CF (no one wants to raise money to save someone with CF while simultaneously causing them to be sicker).   I am impressed with the CFF's use of technology as a way to help those with CF to feel a part.  

Another great use of technology at this year's VLC Conference was Twitter.  Not only were we encouraged to tweet about our time at VLC but the CFF offered us a laptop and computer screen in the lobby so we could tweet using the hashtag #VLC2013 and the handle @CF_Foundation.  I decided that maybe twitter could be good for Bennett's Brigade so I am now on Twitter: @breckgamel and will post under #bennettsbrigade.

Although I will share in greater detail about what we learned at the VLC Conference in the next post, here is a brief recap via Cystic Fibrosis Foundation's webpage:

13 March 2013

What we spoke to Congress about...

I will post one more time about March On The Hill 2013 before I move on to sharing about my time at the CFF's VLC 2013 Conference within the next few days.  I wanted to document some of the specific things I learned about the Cystic Fibrosis Foundation with regards our public policy agenda and our message to Congress this year.

March On The Hill is part of the Cystic Fibrosis Foundation's Advocacy Program.  The goals of the CFF Advocacy program are to advance research, to promote access to care and to raise awareness of the disease.  Most of the program is made up of volunteers who offer to stay informed via email about changes in the CFF's public policy agenda and other volunteers who have indicated they want to stay in touch with their local government to communicate our needs to our lawmakers. (There is a video of how you can be CFF Advocate on the bottom of this post.)

Although last year, advocacy at the CFF involved pushing for the passage of the EXPERRT Act, this year's efforts were not based on a bill that needs passage.  This year, we are just asking our representatives to protect funding of several vital federal programs:

If you've heard of "sequestration" on the topic of the federal government, let me explain how it's going to affect Cystic Fibrosis.  Sequestration is a series of automatic, across the board cuts to government agencies in an effort to minimize the growth of the national debt.  Unfortunately, those federal programs in health care have received an 5% cut in funding.  That's a pretty significant cut to some really important programs that could impact Cystic Fibrosis research.

The CF Foundation works very closely with the National Institutes of Health (NIH) and the Food and Drug Administration (FDA).  Both of these agencies are now having to figure out how to cut 5% out of their budgets.  So, we are asking our Congressmen and Senators to please help protect funding of the NIH and FDA.

Let me explain how important the NIH and FDA are to our efforts to a cure.

The NIH is great at supporting basic human research.  The way the NIH is set up, they give small grants to up and coming researchers (many are PhD students or professors at universities across the nation) and employ researchers at their own laboratories in Bethesda, Maryland.  These researchers, in a variety of health care fields, do basic research on how the human body works, things like, "how does the sodium chloride channel work" or "how does infection affect the immune system."  This type of research is that which we can all benefit from.  But it's particularly of interest to the Cystic Fibrosis Foundation because we know we must truly understand what is going wrong in the body, with regard to CF, before we can fix it.

For example, the basic science behind Cystic Fibrosis (research on the human genome) was funded in part by the federal government through the National Institutes of Health.

When something important in basic human research has been discovered to affect those with the disease of Cystic Fibrosis, this is when the Cystic Fibrosis Foundation comes in.  The CF Foundation, using private funds, will often partner with those scientists and their basic human research to further their studies.  Research for a cure for Cystic Fibrosis is jointly funded by the National Institutes of Health (NIH) and private funds.  The CF Foundation does not receive or accept any government funds but does use already funded projects at the NIH to help further find a cure.

This is what happened with the development with the medication Kalydeco.  Once the CF Foundation better understood the basic human research behind the sodium chloride channel, the CFF took private money they had raised and helped pay for the furtherance of that research.  So, if we knew what went wrong in the sodium chloride channel, the CFF offered the financing to have the researchers now move forward to see how we could fix the sodium chloride channel.

At this stage in research, the CFF has now taken private money to further the basic human research already completed by the NIH.  This is why the NIH is so important to the CFF.  The CFF is directly benefiting from research at the NIH.  If the NIH has to cut down on staff or research grants or programs, it will slow down the CFF's efforts to further research in the world of CF.

Once basic human research from the NIH is furthered with private funds from the CFF and research has now been discovered where a drug could be beneficial, this is when industry/the pharmaceutical  pharmaceutical companies will get involved.  But pharmaceutical companies don't want to waste their time or money on discovering a drug.  They want to be involved when they smell success on the potential of marking a very real opportunity for a drug to fix the human body.

This is the point in which Vertex became involved in the process of discovering Kalydeco.  We need the pharmaceutical companies.  But it is difficult to get them involved in our basic research, which is why we need the NIH.  Once industry is involved in the drug-development process, then, we now have three parties involved: the NIH, the CFF and now the drug companies.

Once the drug companies have gotten the drug to place where they feel the drug is worthy to be marketed, the drug companies, along with the CF Foundation, will submit the drug for approval from the FDA.  This is the second federal government program the CFF works very closely with.  The CFF follows the drug throughout the FDA approval process.  In fact, CFF has a great relationship with the FDA.  The FDA knows we submit only those drugs that are well-researched data-driven.

Once the FDA approves a drug, it can then be placed on the market for patients to use.  This process was expedited recently for the CF drug Kalydeco (the third fastest drug to pass through the approval process with the FDA due to effeciency of data and the need for the drug to save lives).  Slowing down the FDA process could have dire consequences for those with CF.  But if the FDA continues to have to absorb large cuts to its funding, we may see exactly that happen.

So, as you can see, this year we are asking our Congressmen and Senators to protect NIH and FDA cuts to help them to continue to be efficient with regards to Cystic Fibrosis research.

Let me put this in another smaller chart form.  This is basically a repeat of what I just wrote:

NIH (federal funding=research) works with the CFF (private funding=research). Together, that collaboration offers new research. ---->  Once enough research has been done, industry/private sector (aka pharmaceutical companies) get involved, bringing in more financial backing.  ---> Once a drug has been shown to work in the lab, the FDA gets involved to oversee the trial process and approve the drug for human use.
The CFF is an example of excellent and effective drug development and care.  I am very proud of the way the Cystic Fibrosis Foundation uses its money as well as the already present research out there.  I am blown away by the efficiency of money and research to find a cure.  As a mother of a child with CF who regularly solicits private giving to help my child benefit from a cure, I am very proud to be a part of a process that IS working and is working well.  Hearing more about how we help in the development of CF drugs makes me very confident in the Cystic Fibrosis Foundation and their efforts to get a cure for all those with CF.  It's working - the partnership with the NIH, the FDA, the Foundation and the drug comanpies.  I hope it will continue to work smoothly.  Protecting the NIH and FDA's budgets is crucial to our need for a cure.

The other thing we discussed as a part of our public policy agenda, something that will become more relevant on a state government level than on a national level (but one we still mentioned to our federal representatives) is the importance of "Cystic Fibrosis Care Centers."

Cystic Fibrosis, being a rare chronic disease, needs data-driven highly specialized specialized care.  This is why we have "CF Centers."  CF Care Centers are those multi-faceted hospital departments and clinics that treat only Cystic Fibrosis patients.  Bennett visits a CF Care Center every three months.  His CF Care Center doctors (a pulmonologist, a GI doc, a ENT doc, etc.) are specifically trained in his disease.

As Bob Beall recently said, "We are the poster child for personalized medicine."  We have learned that children and adults with CF need care that can specifically address their disease on many levels (remember, CF affects the entire body, not just the lungs and digestive system).  CF Care Centers effectively treat the patient and use data to do that.  CF Care Centers are accredited by the Cystic Fibrosis Foundation and are held to very high standards in the way of CF Care.

However, with the new laws from ObamaCare/the Affordable Care Act, we may see children and adults with CF having less access to this type of care - as efforts to minimize costs are made.  This will be very determintal to those with Cystic Fibrosis.  We want our governmental representatives to know how important our CF Care Centers are to those with CF and how we must protect access to these centers for those with the disease, despite upcoming changes in our healthcare system.

That's the basic gist of what we spoke to our represetnatives about.  I found learning and later articulating this information to be very helpful for me as a mother and advocate for my son.  I have a very strong desire to keep a pulse on what the CF Foundation is doing and making sure they are using our hard-earned money well.  I am very pleased to hear they are not only doing that but they are advocating well on our behalf in other areas, as well.

Before I end this point, let me add that we need many more people to advocate for those with Cystic Fibrosis.  Here's a video of what Advocates do and, if you're interested, how you can sign up to help:

11 March 2013

March On The Hill 2013: Day 2, Evening

After a full day at the Capitol and a few hours of rest, our group gathered Thursday evening for a reception dinner at the hotel where we shared stories of our day.

Chris and Noreen, a great couple who has been at this for years.  They have a son in high school with Cystic Fibrosis.

Noreen, me, Mary and Angie - Noreen is a CF mom and all around wonderful person.  She does everything she does with such grace.  Mary is the VP of Governmental Affairs for the Cystic Fibrosis Foundation.  She is incredibly smart, talented and does an amazing job with regards to Cystic Fibrosis and the development of public policy.  We are so lucky to have her at the CFF.  Angie is a CF mom of two teenage girls, a veteran volunteer and someone I absolutely admire.  Angie's got bounds of energy and passion for advocacy that I love and want to soak up!

Congressional Representative John Fleming (R-La) came to speak at our event Thursday night.  He has his own very special and very personal connection to Cystic Fibrosis: Bennett is his grandson.  I am super proud of my father for his work in Washington DC as well as how he is a champion for those with Cystic Fibrosis.  My mother, also pictured, is a loving grandmother to the boys and a fabulous mother to me.

This is Peter (left) - such a great guy - fun to be around and passionate about finding a cure for Cystic Fibrosis.  His teenage daughter, who suffers from CF, has been on the life-changing drug Kalydeco for a short time and is showing amazing improvement in her lung function.  Peter became emotional this evening as he shared with us all how lucky he is not to have to worry about losing his daughter and how he wants that for all of us.  Peter is also the volunteer chair of the Cystic Fibrosis Foundation's Annual Fund and does a phenomenal job raising money for a cure.

I must add something about this picture (above).  Oliver (who adores Peter, by the way) is in the picture because he had a chance to come with me to Thursday's dinner (since this year's dinner was relatively low-key due to the weather and our having a smaller-than-normal group at March On The Hill).  I chose to bring Oliver to the dinner because I wanted him to get to watch his Congressman grandfather speak as well as get to see what the meetings I to go to are all about and how what Brian and I do relates to Bennett.  So often, Oliver sees me do things for CF but has no idea what I'm really doing.

At the dinner, each person in the room shared informal introductions.  When it was Oliver's turn to introduce himself, I asked him if he wanted to share (since he was just a kid and most everyone else were adults).  He indicated he did so on his turn, he stood up on his chair and said in a strong voice, "My name is Oliver.  My three-year-old brother, Bennett, has Cystic Fibrosis."  And then, completely unsolicited and to my surprise, he added, "And we need money for a cure."

The crowd of mothers, fathers and friends of those with Cystic Fibrosis broke into laughter and applause.  Oliver knew we were there because we were working towards asking people for money for a cure.  I hadn't realized that he would feel the need to ask for himself!

I was incredibly proud of him in that moment.  I realized, right there, that, on some level, he gets it - he got why we were there and is beginning to really understand why I do the things I do.  To my surprise, he is beginning to see he too can be a part of this process, of helping his brother.

Clearly, Oliver had not just seen his coming along with me to an adult dinner as an opportunity for his Mama to advocate for Bennett but for him to do so, as well.  What a gift Oliver is to his brother!  And, more importantly, what a gift he is to me! :)

This dinner concluded our March On The Hill conference until we convene again next year.  I can't wait to come back and do it all again!!

09 March 2013

March On The Hill: Day 2, Morning

Boy, what a day!  Our day started very early Thursday morning with a 7:30AM breakfast and 8AM visit with Bob Beall, CEO and president of the CF Foundation.  Although we had received the bulk of our training Wednesday, we were given more pointers on things our Representatives' and Senators' offices might ask us during our meetings that afternoon.

Unfortunately, due to the weather, many of our members of Congress were not in the office Thursday (many flew back to their districts/homes Wednesday).  So, instead of meeting with our Represenatives, we were told we would likely meet with their staff members.  Although initially disappointing, I had to remind myself that the staffers we would speak to are people who keep the members of Congress abreast of healthcare issues.  So, while we may not have been able to speak with the decision maker, we certainly were going to get a chance to speak with a valuable voice in the office.

After breakfast and a quick presentation by Bob Beall, we were given our packet (of which wish I had taken a picture).  Each of our packets listed our scheduled meetings for the day (the CFF arranged all meetings) and provided us handouts for each meeting.  This was awesome.  It was so organized and made it incredibly easy for us!

My first meeting at 9:30AM was with my own Representative Bill Flores (R-TX).  I had fully expected to meet with his staffers.  But I was surprised when I learned Rep. Flores himself was in the office and would be meeting with me.  It turns out that he couldn't get a flight back to Texas due to the weather, so he just decided to come to his office to work.  I was more than delighted!

What a lucky gal I was to get to meet with Rep. Flores again.  His Legislative Director John Ohemen also joined us for our meeting.  He enjoyed getting an update on Cystic Fibrosis and what we are needing from Congress these days (mainly protection from funding cuts at the National Institutes of Health (NIH) and the FDA).  But I was most incredibly touched when Representative Flores shared with me as a side note that he has been praying for Bennett and that he had put Bennett on his prayer list ever since our last meeting.  Boy, what a sweet thing to do.  And coming from this Texan, I know he means it.

After I met with Rep. Flores, I went down the hall to my next meeting.

I met with Jenny Howell from Rep. Barton's office.  Jenny, Rep. Barton's Legislative Assistant, met with me since Rep. Barton was already back in Texas.  Jenny was knowledgeable, interested and seemed to genuinely appreciate Bennett's story of CF and how the government and the CF Foundation are working together effectively to find a cure.  She asked some great inquisitive questions, which I really appreciated.  I look forward to meeting Rep. Barton sometime in the future but I really enjoyed getting to sit down with Jenny today.

My last meeting at the House of Representatives was with Sarah Johnson, Legislative Assistant, from Representative Burgess' office.  She had worked in biology some so she was somewhat familiar with Cystic Fibrosis, which was great.  I shared with her the same things I had shared in my previous meetings but was able to be a bit more technical as she seemed interested in that area.  I really enjoyed getting to meet the staff from each office because it allowed me to get a feel for what they were interested in.  It certainly gives me confidence with going forward to meeting the Representatives next time I get an opportunity in DC or in the district.

My last meeting was with Senator Cornyn's office on the Senate side of the Capitol.  It's interesting how different this building is from the Representative side.  This building is bigger, more plus and seems more "airy."  I suppose it's because there are much fewer Senators than Representatives.  But I enjoyed walking the halls a bit.  It's *much* easier to get around in the Senate building than trying to navigate the Representative hallways (which all look exactly alike, no matter which way you turn).

Since Senator Cornyn was unable to meet, I met with his Legislative Correspondent, Wes Hambrick.  Wes was very engaging, attentive and caring about our need for a cure.  He, like the others, seemed to find my message refreshing.  I wasn't asking for more money, I was asking for the money that goes to research on rare diseases be protected.

Wes surprised me by one question when he asked, "how much does the Cystic Fibrosis Foundation get from the National Institutes of Health?"  I loved the question because I was able to say: "nothing!  The CFF receives no government funding.  All we want is for the NIH to continue their work on basic human research and we will continue to partner with them to move that research towards a cure."  I was able to explain that I am a part of a larger network of thousands of people who fund raise for Cystic Fibrosis each year so that we can put this money with the federal dollars already spent by the NIH to move research further and eventually get the pharmaceutical companies interested in investing in worthwhile and effective medications."  It felt good to speak free of the need for funding because I know that our funding comes from those who give from their hearts, not from their tax dollars.  As Wes indicated, my message is a bit of a change of message in Washington DC.

Overall, I had a fabulous time.  I was nervous at first.  I wondered if I could do it alone.  But I did and it was actually a lot of fun!  By sharing Bennett's story and what I know of how the Cystic Fibrosis Foundation works with government agencies, I was able to really articulate how proud I am to be a part of this organization and to be speaking on Bennett's behalf.

06 March 2013

March On The Hill: Day 1

When I went to bed last night, March On The Hill (MOH) was still planned.  When I woke up this morning, it had been cancelled.  By the late morning, it was on again.  What's the deal?  Bad Weather! Boo!

The Cystic Fibrosis Foundation had expected about 60 people from around the US to fly in to DC to participate on the annual March On The Hill event today.  However, expectations of up to 12 inches of snow in the area cancelled flights as well as the congressional session today.  Realizing that we wouldn't likely be able to meet with our respective Representatives and Sentaors, the CFF March on the Hill team decided to cancel the event.

However, questions lingered...what about the 15 people already in town for MOH?  And what happens if the expected "12 inches of snow" turns into nothing but "ice-cold rain"?  The answer:  You pull together to make the best of it and you call it "Mini March" (as in mini-March on the Hill.)

Mary Dwight and Eric Chamberlin (see standing in picture) from the CFF's Public Policy department met with those of us in town this afternoon to train us on all things "advocacy" in the event we can meet with our Congressman's office should the federal government continue on as planned tomorrow.  (The congressional session has already let out for the week due to the weather.  Whether we will meet with staff or not is still to be determined.  But I expect we will have a chance to speak with someone from most of the offices we had hoped to meet with.  Unfortunately, we won't get the face-to-face Congressional appointment we were hoping for).

It's late and I need to head to bed so I don't have time to write what I learned today.  But it was wonderful to learn better about how Congress works, what we need from the federal government and how to tell our story effectively.  I am so thankful Bennett has such a wonderful organization advocating on his behalf.

Of course my greatest delight from attending any Cystic Fibrosis Foundation event is meeting the people.  I love it when I get to meet families who can speak CF-jargon and immediately understand the feelings involved with a child whose health is deteriorating, a child who just learned how to swallow pills or a doctor whom is very beloved.

Cystic Fibrosis is an honest disease.  So, I often find honest people behind the disease - honesty with regards to gross things (such as mucus plugs and CF digestive issues), honesty in needing community, honesty in the hope of a cure.  It feels good to not have to say anything to someone and know they just get you in this area of your life.

I look forward to another day with these folks.  I'm very proud of the organization that is helping our son.  And I appreciate so much the opportunity to be a part of it.   I hope that tomorrow I can use the training we were given today.  But, most importantly, I hope tomorrow brings laughter and honesty with a group of families going through raising a child with CF just like us.

05 March 2013

CF in DC

{EDIT: Er, cancel that.  March On The Hill was cancelled early this morning due to snow and the closing down of Congress after 1pm today, what would have been our first day of March On The Hill.}  

This week, I am traveling to Washington DC to attend two Cystic Fibrosis Foundation conferences.  One conference, towards the end of the week, will be the same conference I attended last year called the “Volunteer Leadership Conference.”  I am very excited to attend this conference again this year and to have the opportunity to meet lots of amazing people from around the nation who tirelessly work to help us find a cure for CF.

The earlier conference I will attend this week is called “March On The Hill.”  Last year, I signed up to volunteer as the Cystic Fibrosis Foundation's State Advocate for the Northeast Texas Chapter.  The CFF has volunteer advocates from around the US.  This week, I will join dozens of these advocates on Capitol Hill to help lobby members of Congress for adequate funding of the FDA and legislation that supports getting effective medications into the hands of those with Cystic Fibrosis.  This is a bi-partisan effort.  I am not going to Washington DC to share my political views.  I am going only share about Bennett’s story and our need for a cure.

Advocating in Washington DC is something I am looking very forward to doing, although it’s also the conference I am most nervous about attending.  I'm nobody special - no political science degree here - just a mom with a little boy who needs help.  But I look forward to learning of new ways I can advocate for Bennett.  

Brian would normally join me for these conferences but he must stay back to present at a theological conference in Dallas latter this week.   I'm sad he isn't able to attend but he's always an equal partner in our efforts to help Bennett.

I'll post this week about what I'm learning through both of these experiences.  The awesome thing about CF Foundation is that, since it's an effective advocacy organization, it tends to benefit not just the 30,000 people with CF it represents, but it tends to benefit all those in the United States with orphan diseases.  I look forward to this week and being a part of something that is bigger than just helping our son.  I look forward to representing all of those with CF as well as those who suffer from under-researched little-known diseases that do not yet have a cure.  Finding a cure is half the battle.  The other half is getting it in time into the hands who need.

Will share more details soon...