This week, FightSMA was proud to be an NINDS Invited Participant of The 2014 NINDS Nonprofit Forum. The Forum provides an opportunity for nonprofit leaders to engage in discussions with NINDS staff and program directors. FightSMA Collaborating Scientists Dr. Thomas Crawford (MDA, Johns Hopkins University) and Dr. Kurt Fischbeck (NIH/NINDS) helped to lead the Clinical Outcomes Panel, where Spinal Muscular Atrophy (SMA) was distinguished many times as a unique disease example currently reflecting great progress towards clinical treatments as a result of collaboration by all sides, and with its strong advocacy backing being able to provide all the difference for success.
We also join with the NIH/NINDS and colleagues in thanking Dr. Story Landis her many years of service and faithful support of SMA as she leaves her position as NINDS Director to enter retirement. As Dr. Jane Paulsen (University of Iowa) stated, “it is so critical for representatives of rare diseases – of ALL diseases – to work together.” The ability to share data and truly collaborate across a wide spectrum is the surest path to scientific progress, especially for a rare disease like SMA, and is why FightSMA is so dedicated to this endeavor through programs like our Annual Conference and our NPRNA legislation. We thank you for your support of our ongoing advocacy efforts and will continue to keep you updated on our progresses!
FightSMA and the Coalition for Pediatric Medical Research met with House Energy and Commerce committee Chairman Fred Upton (R-MI) on September 10, 2014, to discuss NIH’s pending implementation of the National Pediatric Research Network Act that was signed into law last November. Chairman Upton is a champion for pediatric research and continues to work closely with FightSMA to help provide additional federal support for SMA clinical trials. In addition to shepherding the NPRNA through Congress, Chairman Upton is leading the new 21st Century Cures Initiative on a bipartisan basis with his House colleagues. After a series of roundtables and hearings exploring every aspect of the process for discovery and development of new treatments, the initiative is expected to result in legislation in the coming months that would accelerate the pace of cures and expand resources available for medical research. The NPRNA was featured by the initiative as an example of the kind of collaborative approach that will improve research and save lives. We thank Chairman Upton for helping advance the fight against SMA and we look forward to working with him to secure the resources to support a robust and timely implementation of the NPRNA law.
“It began with a 1990 solicitation letter, which Joe Slay wrote to friends on a second-hand McIntosh computer purchased for that purpose. A helpful woman at the Muscular Dystrophy Association had clued him into research being undertaken by a Columbia University Medical Center (CUMC) geneticist, Conrad Gilliam, who was trying to identify the SMA gene. The Slays had sent Columbia a $1,000 check, earmarking it for Gilliam’s work. More was needed, obviously, hence the fundraising letter. Friends gave money; strangers, too. In six months, they raised $25,000 for Gilliam’s lab. What ensued over the next 24 years is a long story… Today, (that) organization is called Fight SMA.”
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FightSMA wishes to extend our heartfelt thanks to Congressman Eric Cantor (R-VA), who announced Friday, August 1, that he will be stepping down from his seat in Congress later this month. A longtime advocate for expanded pediatric research, Cantor was instrumental in the 2013 passage of the National Pediatric Research Network Act (NPRNA).
Congressman Cantor and his dedicated staff began working with FightSMA more than a decade ago to advance spinal muscular atrophy research. He was the first Member of Congress to introduce legislation to support a clinical trials network for SMA, becoming the lead Republican sponsor of the SMA Treatment Acceleration Act in 2007. Since that time, the Congressman has met with SMA families, heard our concerns and wishes, advocated for increased pediatric research funding, and made the NPRNA a top priority as House Majority Leader since 2011.
As he prepares for his departure from Congress this summer, FightSMA would like to thank Congressman Cantor for his tireless dedication to our mission of finding a treatment or cure for SMA and other devastating pediatric disorders, and we hope the future brings opportunities to work with him again.
On Wednesday, July 9th, the House Energy and Commerce Committee continued its series of hearings on the 21st Century Cures initiative, which was launched earlier this year by Reps. Fred Upton (R-MI) and Diana DeGette (D-CO). The topic of Wednesday’s hearing was steps that the federal government can take to modernize clinical trials so that they become more cost-efficient and produce results that are quicker to reach the market.
In their remarks, members of the committee and witnesses to the hearing agreed that the size, duration, costs and failure rates of clinical trials are higher than ever and that this curve must be bent in the other direction. In her opening statement, Rep. Lois Capps (D-CA) offered the National Pediatric Research Network Act (NPRNA) as an example of legislation that is taking a step in the right direction. Several of the other speakers proposed standardized clinical trials as a way to reduce redundancy and maximize the data available from studies across the globe. Another popular suggestion was the creation of a national patient registry database, which would have the potential to greatly reduce the length of time needed by researchers to find volunteers for clinical trials. One witness to the hearing suggested that a database of this kind could reduce the process of finding volunteers from several years to just weeks in some cases.
The focus of this hearing was critically important to the mission of FightSMA as we continue our efforts to find a treatment. As FightSMA continues its work with the National Institutes of Health (NIH) to implement the NPRNA, we thank the Energy and Commerce committee for its ongoing focus on the importance of modernizing and improving our nation’s health delivery systems. FightSMA will continue to monitor future hearings on this important initiative, and we again thank Chairman Upton, Ranking Member Waxman, and Reps. McMorris Rodgers and Capps for their leadership in enacting the National Pediatric Research Network Act.