Soldiering On

superheros

Andy and me, circa 1980. Superheros at heart. Though I was humiliated by the idea, I posed for this picture for my pesky little brother. The things I do for him. Always.

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, I lost my younger brother and only sibling, Andy, to cystic fibrosis (CF).  This is not an anniversary to celebrate, but it is one that I cannot escape; one I feel is important to acknowledge.

Ask anybody and I’m sure they’ll agree that eleven years is along time to support a cause in someone’s memory. Yet for almost eleven 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.

One might think that after many years of doing so, it would get easier for me to ask for support.  In fact it is quite the opposite — especially in light of recent tragedies in our nation and 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.

Now more than ever, this dream is within our grasp.  Last year, the FDA approved Kalydeco® – the first drug to target the underlying cause of CF. This drug treats patients with a certain gene mutation which is present in roughly 4 percent of the CF patient population in the U.S. I am thrilled to report that patients who use this drug are seeing phenomenal results! In addition to helping that 4 percent of our patient population, Kalydeco® is proving to be a key partner to other therapies in the CF Foundation’s research pipeline. Phase 2 and Phase 3 studies of Kalydeco® in combination with two other compounds are  currently underway. The combined therapies are designed to treat the root cause of CF for patients who have the most common gene mutation. At the rate our therapies are progressing, the hope is that 65 percent of CF patients in the U.S. will have some kind of control for the basic defect by 2015. This is fantastic news, but we won’t stop until we have control for ALL of our patients.

Yes, we continue to soldier on. We continue to strive for a cure. But having drugs that will administer control over the symptoms and complications of CF will, in and of itself, be a miracle. When cystic fibrosis is no longer considered a life-threatening disease, our dream will finally be realized!

If you would like to support Roo’s Crew and the CF Foundation in our mission, please follow this link: http://www.cff.org/Great_Strides/BlakeLeyers8622.

To find out more about the Phase 3 and Phase 2 Kalydeco® combination studies, click here and here.

* WHAT:  2013 Nashville Great Strides

*WHEN:  Sunday, May 5, 2013 at 3:00 PM

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

Tribute to Andy 

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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
Image by Cystic Fibrosis Foundation

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.

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