Progress!

Hello blogosphere.  Yes, it’s been awhile.   Seeing that I haven’t posted since the half marathon, perhaps you’ve wondered if I survived.  Well, I did live to tell about it and shall do so soon!  But today, I have some updates to share from the CF world.

Progress in the Lab

Last Monday, May 7 Vertex Pharmaceuticals announced interim results from a Phase 2 clinical trial of Kalydeco™ and VX-809, a CF drug in development.  The results showed a significant improvement in lung function in people with two copies of the most common CF mutation (Delta F508) who received the two drugs in combination.  Both Kalydeco™ and VX-809 are designed to treat the underlying cause of CF.   While these drugs would not be cures for CF, it appears they have the potential to control certain symptoms of CF to a great degree.  Click here to read more about the Phase 2 interim results.

Related articles: http://bit.ly/K76iUX and http://onforb.es/JMy4ah

Progress on the Hill

In this post, I explained that one of the key items on the CFF’s public policy agenda is to get the EXPERRT Act  included in the House and Senate versions of the Prescription Drug User Fee Act (PDUFA).  In short, the EXPERRT Act would require the FDA to ensure that opportunities exist for consultation with external experts on products for CF and other rare diseases.

I am thrilled to share that representatives in both the Senate and the House heard our voices!  The EXPERRT Act has been included in the final versions of PDUFA for both the Senate and the House.  The bills will now proceed to the Senate and House floors for final vote in the near future.   The CFF, my fellow advocates, and I are cautiously optimistic that the core of the EXPERRT act will remain in the final PDUFA bill which will be signed into law.  My State Advocacy co-chair Marissa and I especially appreciate the outreach made by our fellow constituents to Representative Marsha Blackburn.  As a member of the Health Subcommittee of the House’s Energy and Commerce Committee, Congresswoman Blackburn’s support was paramount!

I think this goes to show that we should all follow William James‘ advice:

Act as if what you do makes a difference.  It does. 

Running Out of Love and Gratitude

Time is a precious commodity.  It is defined as the measurable period during which an action, process or condition exists or continues.  We measure time in intervals such as seconds, minutes, hours, years.  We measure time by important events that occur in our lives – anniversaries of births, weddings, graduations, deaths.

On April 24, 2002 an event occurred that forever changed my life; I lost my younger brother and only sibling to cystic fibrosis (CF).  It is difficult to believe that April 24, 2012 is the tenth anniversary of Andy’s passing.  This is not an anniversary to celebrate, but it is one that I feel is important to acknowledge.

For nearly ten years, family, friends, and many who never even knew Andy have honored his memory by supporting me and my walk team, Roo’s Crew, in the Cystic Fibrosis Foundation’s annual Nashville Great Strides Walk.  While Roo’s Crew will once again participate in the Nashville Great Strides Walk (*see event details below), I will be doing something different to acknowledge this anniversary and the incredible support we’ve received over all these years.  On Saturday, April 28, 2012, in tribute to Andy and as thanks to all who contribute to the CFF through Roo’s Crew, I will be running the half marathon portion of the Country Music Marathon.

One would think that, over time, it would get easier for me to ask for your support.  In fact, it is quite the opposite – especially in these tough economic times.  Through these many years, some have asked why I continue to be involved when CF is no longer something my family and I have to face every day.   The answer is simple:  I carry Andy’s legacy of hope and faith that we will see a cure for CF in our lifetime.  And I am overjoyed to say that this desire, this mission was tremendously validated in late January when the FDA approved the new drug Kalydeco®.    Please read more about Kalydeco® at www.cff.org

There are myriad reasons why you can feel good about contributing to the Cystic Fibrosis Foundation; one being that nearly 90 cents of every dollar goes directly to research, education and care programs.  Whatever your reason and whatever the amount – for no amount is too big or too small - I would be tremendously honored by your support.  To make an easy online tax-deductible donation, please click the GREAT STRIDES widget in the right sidebar, then choose “CLICK TO DONATE”.

If you would like to join Roo’s Crew at the Nashville Great Strides walk, just follow that same link and click “JOIN MY TEAM”.

With love and gratitude,

Blake

* WHAT:  2012 Nashville Great Strides 

*WHEN:  Sunday, May 20, 2012 at 1:00 PM

*WHERE:  Bicentennial Capitol Mall State Park, Nashville, TN

Your Voice Counts!

A Message of Hope and Proof of Concept

On March 8, 2012, my friend Marissa and I joined dozens of other advocates from across the country in Washington D.C. to talk with our nation’s Senators and House Representatives about issues important to the cystic fibrosis (CF) community.  What a thrill it was to go to Capitol Hill on the heels of the FDA’s approval of Kalydeco®; the first medicine to treat the underlying cause of CF for 4% of the CF population (those who have a certain mutatation in their CF gene called the G551D mutation).   Additionally, Kalydeco® has created a road map that will extend to treatment for 100% of CF patients.  Dr. Margaret Hamburg, FDA Commissioner, and Dr. Robert Beall, President and CEO of the Cystic Fibrosis Foundation, hail this as a huge step forward for both personalized medicine and for CF treatment.

Me, Congressman Jim Cooper, and Marissa - March 8, 2012

CFF Agenda

Like last year, we met with our legislators to explain how the National Institutes of Health (NIH) and Food and Drug Administration (FDA) play critical roles in the search for the cure for CF, and why it is so important that funding to these two institutions continues.  Since our meetings, a record 150 co-signers signed onto Congressman Ed Markey’s letter supporting funding for the NIH in Fiscal Year 2013.

Additionally, the CFF’s public policy agenda for 2012 seeks to expand rare drug expertise during the FDA review process.  On March 7, the day before our advocacy meetings, Congressional CF Caucus Co-Chairs Representatives Edward Markey (D-MA), Cliff Stearns (R-FL) and Tom Marino (R-PA) introduced the “Expanding and Promoting Expertise in the Review of Rare Treatments (EXPERRT) Act,” in the House of Representatives. This legislation aims to enhance the FDA’s rare disease drug review process by including systematic consultation with experts and patient advocates.  Consulting with rare disease experts will ensure that the FDA has complete information, allowing drugs to move through the pipeline quickly.  Kalydeco® is an excellent example of this process.  It was granted approval in approximately three months, making it one of the fastest FDA approvals ever.  Simply put, the EXPERRT Act will make best practice standard.

You can help!

Marissa and me at Rep. Blackburn's office

One of the most enlightening things I learned while in D.C. was that every phone call, every letter, every email gets counted and noticed by our Representatives.  The information gathered in those communications is the baseline those offices use to know what is going on.  Please join Marissa and me in helping CFers and countless others with rare diseases.  Tell your lawmakers to support the Expanding and Promoting Expertise in Review of Rare Treatments (EXPERRT) Act of 2012! Your members of Congress will support the bill if they hear that it is important to you, your friends and your family. Email your legislators today and urge them to support the EXPERRT Act!

Please click here for the quickest, easiest way to make your voice count.

Playing Catch Up

Hello blogosphere.  I have missed you!  It’s past time for updates.  I am anxious to share about my experience on Capitol Hill, but for today, let’s get up to speed on my half marathon training (have you missed my puns?).

In January, at the first group run, our training coordinator noted that this journey would require sacrifice.  The further into this journey I have traveled, the more I appreciate her statement.  My greatest sacrifice is time.  As the weekly training miles increase, so does my time commitment.  Squeezing in a four to six mile run three times between Monday and Thursday each week is challenging.  I sacrifice time with my family and friends.  I put off tasks that have a tendency to become daunting when left undone for too long.  I’m so very thankful to my husband, kids, parents, and friends for being gracious, supportive, and forgiving of this sacrifice of time.

Mileage-wise, I now have ten consecutive miles under my water belt (Admit it.  You missed my puns).  I’ve learned that every run is different; some are relatively smooth, others are a battle to the finish.  Instead of thinking about how many miles and hills I have ahead of me, I’ve realized that I have to focus on the present and take it one step at a time.  As one of our trainers said to me last week, “You’re not a speed runner, you’re an endurance runner.” In the end, whether I’m running, walking, or crawling, I will get there.  Yes, this is a physical journey, but even more so, it is one of the mind and spirit.  In the end, it will be worth it.

 

Nike image borrowed from http://kristensbirthdaywish.com/kristens-blog/

Tribute to Andy

Cystic Fibrosis

It’s just two words strung together.  It’s a disease with a definition.  A diagnosis.  It’s all too common for CF to be relegated to mere terminology, allowing for discernible emotional distance.

Cystic Fibrosis

It’s something that happens to other people.  Not you.  Not your sister.  Not your spouse.  Not your brother.

But it happened to my brother, Andy.  He was diagnosed at age three.  With that diagnosis came the revelation that cystic fibrosis wasn’t just a bit of medical terminology.  It was real.  It was solid.  It wore my brother’s face.

Andy, however, knew better.  He did not let the disease define him.  He was a warrior, not a victim.  It is my dream that we find a cure for CF, but it is in my brother’s memory – in deference to the full life he lived in such a short time – that I strive to promote awareness and acquire funding for vital research.

Since Andy cannot tell his story in words, I will share it with you in pictures.

My deepest, heartfelt thanks to Antigone Rising for permission to use Borrowed Time,  to Ali Trotta for offering just the right words, and to each of you who join me and Roo’s Crew in the fight to eradicate CF once and for all.

Link to my Great Strides page:  http://www.cff.org/great_strides/BlakeLeyers

Storming the Hill – CF Advocacy Day in D.C.

Soon, I will be heading to our nation’s capital to participate in the Cystic Fibrosis Foundation’s annual advocacy event, “March on the Hill”.  What an honor it will be to help deliver information critical to our cause to our elected officials.  With the recent approval of Kalydeco, it will be especially exciting to share our views on drug development and approval policy.

The following is a letter I wrote to friends and family last year, after my first experience on Capitol Hill:

In the nine years since Andy’s passing, much has changed in the fight against cystic fibrosis (CF).  The diagnosis of CF still causes fear, but thanks to research, doctors have a clearer vision of the root cause of the disease.  This clearer vision gives us hope and fuels the progress toward a cure.  One thing that has not changed is my family’s commitment to Andy’s memory through our participation in the Cystic Fibrosis Foundation’s annual Great Strides Walk in Nashville, TN with our walk team Roo’s Crew.

Today, the Cystic Fibrosis Foundation (CFF) is supporting approximately 30 potential new treatments currently in development – that’s more than in the entire history of the disease.  The CFF is also expanding its medical research program by $100 million over the next five years to focus on developing therapies known as CFTR modulators, which address the underlying defect that causes CF.  Researchers are optimistic that the CFF is on the right path to fundamentally change the treatment of CF by targeting the cause of the disease.

Andy, my husband, and me - Hawaii 1998

My heart swells when I think of how happy Andy would be to hear this news.  Though we experienced the worst outcome of CF, there are many reasons why the broad-view CF story is one of hope.  It is a story of success, but we haven’t crossed the finish line.

On March 17, 2011, I joined my friend and fellow CF advocate Marissa to share this story with several of Tennessee’s legislators at the CFF’s annual March on the Hill event in Washington D.C.   The CFF receives no federal funding, but as lawmakers look at making significant budget cuts in response to our country’s current financial crisis, we feel it is especially important they understand the role federal investment plays in the development of the drugs that treat CF.

We described how basic research funded by the National Institutes of Health (NIH) accelerates the development and testing of treatments supported by the CFF.  We also explained that the Food and Drug Administration (FDA) plays a critical role at the other end of the research spectrum.  Cutting the jobs of professionals who understand the nuances of rare diseases could essentially halt the process of moving newly developed treatments through to approval.  This would result in those treatments sitting on the bench instead of being utilized to change, improve, and save lives.  The plea to our legislators was to sustain funding to the NIH and FDA.

Meeting with Congressman Jim Cooper, March 2011

In order to cross the finish line with a cure, the CFF also needs sustained funding.  As mentioned above, they receive no federal funding.  Nor do they receive backing from major drug companies, because these companies do not see supporting rare diseases a profitable endeavor.  Contributions from folks like you and me are what make CF research possible.  Words cannot express the depth of gratitude I feel for the support you continue to provide in memory of Andy.

************************************************

Here’s to hoping for another successful March on the Hill!

No One Truly Knows How Hard You Try

It’s been a couple of weeks since I posted about my half marathon training.  It’s past time for an update!

Today marks the beginning of training week number seven and I can’t believe how far I have come.  Physically, I can now do a six mile run and, starting this week, will be averaging 20 miles a week.  I’m sure my 12 minute mile isn’t fast by “runner standards”, but considering that I’m new at this (and my routes include hills), I’ll take it!

What I want to share with you today is the breakdown of my Week Five; the week I mentally turned the curve:

That Monday I found myself thinking strange thoughts; as I drove past a pair of runners I thought “Yeah, the weather’s great for a run!  I wish I could join them.” —-WHAT?  Where did that come from?

As I explained here, I will be running the half marathon in memory of my brother and in support of CF research.  So when the news of the FDA’s approval of Kalydeco broke on Tuesday, I was walking on air.  I was so elated that I figured I would rock my 4 mile training run.  Ah, but that was not the case.  Instead, it ended up being a brutal, ugly, fight-for-every-mile kind of run.  It left me deflated, frustrated, and glad that cross training was on Wednesday’s agenda.

When I strapped on my running shoes that Thursday, I was plagued by negative thoughts:  What if I have another crappy run?  What if I get injured?  What if can’t see this commitment through?  Then, about midway through my run, I decided there was no room for negative thinking.  I pictured Andy, thought of Kevin Dwyer and so many CF friends, and was reminded of the courage and determination CFers need just to survive.  Remembering why I signed up for this, I shed the last scale of doubt and embraced it – I’m gonna do this!   I AM A RUNNER!

A friend of mine shared this picture during my Week Five.  Its sentiment certainly rings true for runners.  When you simply replace “running” with “living”, it rings true for all.  

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