BROOKE AND BRIELLE ARE CHAIRMAN UPTON'S INSPIRATION for the 21st Century Cures LEGISLATION!

Meet the Two Little Michigan Girls Who Helped Inspire a Big Initiative in Congress

Chairman Fred Upton with the Kennedy girls

Chairman Fred Upton with the Kennedy girls

“This is what hope looks like. We should all have their courage to believe in a brighter future, and their fortitude to fight for it every single day. 21st Century Cures looks at disease through the eyes of those who believe it can be beat. Let’s prove them right."

-Chairman Fred Upton (R-MI)

“I can and I will.” This is the motto of two brave little angels in Mattawan, Michigan, who are battling Spinal Muscular Atrophy (SMA), a rare disease that destroys the nerves controlling voluntary muscle movement. Their names are Brooke and Brielle Kennedy and they are an inspiration for Chairman Upton’s bipartisan 21st Century Cures initiative.

Brooke and Brielle were both diagnosed with Spinal Muscular Atrophy Type II shortly after their births. Their whole lives have been a constant battle with trial treatments and drugs, physical therapy, rehabilitation, and the many daily struggles SMA brings. But Brooke and Brielle have faced these challenges with bravery, optimism, and smiles. 

The Kennedy girls’ bright smiles are infectious. Brooke’s sweet and loving nature alongside Brielle’s enthusiasm for life touches everyone around them. They have a presence that is indescribable. Their cheerfulness and determination compelled Chairman Upton to do all he could to help.

Brooke and Brielle, self-introduced as Cinderella and Sleeping Beauty, first met with Upton in 2011 and told him about their story battling SMA. With the help of their parents, Sarah and Eric, Upton gained a first hand look at the challenges facing families with loved ones suffering from rare diseases. Clinical trials take time and money, research funding is low, the approval process for treatments is slow and inefficient, and the rarity of these diseases means there are very few people to test potential cures. But mostly, there is a lot of frustration and not a lot of hope.

That’s where Brooke and Brielle come in. They were powerful voices that helped Upton and the Energy and Commerce Committee get the National Pediatric Research Network Act signed into law in November 2013 following a multi-year and multi-Congress effort. That was just the beginning. 

Brooke and Brielle visit the U.S. Capitol in the summer of 2014.

Brooke and Brielle visit the U.S. Capitol in the summer of 2014.

With the help of the Kennedys and many other patient advocates, researchers, and health care innovators, the 21st Century Cures initiative continues to bring hope to those dealing with the thousands of diseases without cures. For the past year, the committee has been gathering information on how Congress can speed up the approval process for treatments and cures, expand medical research, encourage innovation, and ultimately save more lives.    

Brooke and Brielle draw the “key to the cure” for Chairman Upton following the April 30 hearing on the Cures discussion draft.

Brooke and Brielle draw the “key to the cure” for Chairman Upton following the April 30 hearing on the Cures discussion draft.

Upton plans to have legislation to accelerate cures and treatments for diseases on the president’s desk by the end of this year. This legislation will have the potential to change not only Brooke and Brielle’s lives, but also the lives of many other patients and families across America. Brooke and Brielle may be little, but they have had a big impact in Congress and have inspired hope for so many.

As Chairman Upton said at the April 30 hearing on the Cures discussion draft, Brooke and Brielle “are two of the brightest stars I know. We have a chance to do something big, and this is our time. And it is Brooke and Brielle’s time.”

To learn more about the 21st Century Cures effort, click here


Sarah's thoughts: 

When my daughters were diagnosed with SMA, we were told there is no treatment or cure for the disease. I remember feeling completely heart broken and helpless against this monster of a disease. I googled every SMA article I could find to try to learn all I could about the disease, but I was really looking for a glimmer of hope. I found these words, "The National Institutes of Heath selected SMA as the disease closest to a treatment of nearly 600 neurological disorders. Researchers believe that a treatment will be found within the next 5 years with the proper resources." That was my spark, that was the glimmer of hope that I needed.

Fast forward to today - and the video I've attached to this post. U. S. Representative Fred Upton, the Chairman of the House Committee on Energy and Commerce, is fighting for that increased Federal funding to find a cure not only for SMA but for all diseases without cures.

With the incredible leadership of Fight SMA, we've worked closely with Chairman Fred Upton to secure mandatory language in the 21st Century Cures bill that would require the National Institutes of Health to implement the National Pediatric Research Network Act (NPRNA). After years of advocacy by SMA families and researchers across the country, the NPRNA was passed by Congress and signed by the President in November 2013. The law would establish a pediatric research network that includes consortia focused on clinical trials for rare pediatric disorders like SMA. The 21st Century Cures Act language will help to drive implementation of the NPRNA, and also authorizes much-needed additional funding for the NIH over the next five years.

The 21st Century Cures Act is a remarkable piece of legislation with the much-needed goal of accelerating the pace of cures in America. This is a big week for the legislation and I truly believe it is history in the making.

We are beyond excited that Chairman Upton announced Brooke and Brielle are the INSPIRATION for this legislation and it brings us so much HOPE for the future.


In May 2014 Brooke and Brielle were featured in the National Organization for Rare Disorders (NORD) Portraits of Courage gallery representing the spirit of the rare disease community.  They had the honor of presenting the Leadership Award to Chairman Fred Upton and also presented their own homemade medal to him as a thank you for all the hard work he has done for the rare disease community.

 

WASHINGTON, May 9, 2014 /PRNewswire-USNewswire/ -- The National Organization for Rare Disorders (NORD) focused a spotlight on individuals and families who display great courage as they live with rare diseases at its Portraits of Courage Celebration last night..

During the program, Brooke and Brielle Kennedy of Michigan, ages 5 and 6, who have a rare disease known as spinal muscular atrophy, put a medal around the neck of Representative Fred Upton of Michigan for sponsoring legislation to promote and expand pediatric medical research.

A Portraits of Courage gallery displayed photos and stories of 18 individuals and families representing the spirit of the rare disease community. These included Sam and Alex Bode, two sisters from Connecticut living with Friedreich's ataxia; Joe Ellenberger of Nebraska, living with paroxysmal nocturnal hemoglobinuria (PNH); and Sam Berns, who died earlier this year and who helped promote worldwide awareness of progeria.

"This was a celebration of the spirit of the community," said NORD President and CEO Peter L Saltonstall.  "We meet extraordinary people every day in our work at NORD, and we chose this way of honoring the courage that our patients and families display in the face of great challenges."

The celebration took place at the National Building Museum in Washington, DC. The program included remarks by Sean Hepburn Ferrer, son of actress Audrey Hepburn and Mel Ferrer, who shared the story of how a rare disease affected his family. Audrey Hepburn died of a rare cancer known as pseudomyxoma peritonei.

In addition to the Portraits of Courage patients and families, honorees included:

PUBLIC HEALTH LEADERSHIP AWARDS:

  • Senator Sherrod Brown (OH)
  • Representative Fred Upton (MI)

ABBEY MEYERS LEADERSHIP AWARD:

  • MPN Research Foundation 

LIFETIME ACHIEVEMENT AWARD:

  • John Walsh, co-founder of the Alpha-1 Foundation and AlphaNet Inc.

PARTNERS IN PROGRESS AWARDS FOR ORPHAN PRODUCTS APPROVED IN 2013:

  • Actelion Pharmaceuticals for Opsumit
  • Bayer HealthCare for Adempas
  • Genentech Inc. for Gazyva
  • Genzyme, a Sanofi Company and Isis Pharmaceuticals for Kynamro
  • GlaxoSmithKline Plc for Tafinlar and Mekinist
  • Pharmacyclics Inc. for Imbruvica  

Photo - http://photos.prnewswire.com/prnh/20140509/86217 

Logo - http://photos.prnewswire.com/prnh/20110719/DC37656LOGO-b

SOURCE National Organization for Rare Disorders (NORD)

http://news.yahoo.com/nord-hosts-portraits-courage-celebration-160600330.html

 

In 2011 we met with our U.S. Congressman for the first time to tell him about our two special constituents who live in his district that have Spinal Muscular Atrophy. We wanted to gain his support for the National Pediatric Research Network Act (NPRNA) - legislation created and championed by Fight SMA. The legislation authorizes the National Institutes of Health (NIH) to establish a network of research consortia that will conduct multi-site clinical research on a variety of pediatric disorders. The NPRNA requires that an appropriate number of consortia to focus on research conducted on pediatric rare diseases such as spinal muscular atrophy (SMA), including clinical trials evaluating potential therapies and treatments. It will help create the collaborative infrastructure necessary to conduct leading-edge pediatric research today and well into the future, including support for promising young investigators beginning their careers in pediatrics. 

The National Pediatric Research Network Act (NPRNA) PASSED Congress and was signed into Law on November 27th 2013 by President Obama.

Chairman Fred Upton continues to push for accelerated cures in America by recently unveiling the 21st Century Cures initiative. The Energy and Commerce Committee released a video highlighting the merits of the program and cited the passage of the NPRNA as a model for Congress to use as it considers ways to support medical research in the future. The video can be found at: http://bit.ly/about21stCC