These are exciting times for SMA research!
A FightSMA-funded gene therapy program led by Dr. Brian Kaspar at Nationwide Children’s Hospital is gearing up to commence phase I human clinical trials. This is a single-site, dose escalation study to evaluate the safety and efficacy of gene transfer for SMA type I. They will be looking for nine infants with SMA type I between the ages of 0 and 9 months.
Keep your eye on http://ClinicalTrial.gov/ for more information about this promising program as enrollment begins.
And for more information about this clinical trial, click here.
The Muscular Dystrophy Association (MDA) and the National Institute of Neurological Disorders and Stroke (NINDS) are co-sponsoring a workshop on April 22, 2014 8:00AM ET entitled, “Joint MDA-NINDS Workshop on Best Practices for Gene Therapy Programs.” The focus will be on core challenges in the development of gene therapy for neuromuscular diseases and develop recommendations to help guide such efforts. All are encouraged to join via webcast!
April 22nd 8:00AM ET
The workshop will be broadcasted live at:
WASHINGTON, D.C. (April 10, 2014) — Two organizations dedicated to defeating the childhood neuromuscular disorder spinal muscular atrophy (SMA) announced today three winners of the “FightSMA and Gwendolyn Strong Foundation Emerging Investigator Awards.”
The awards, conceived jointly by the two organizations, are designed to draw strong, innovative talent into SMA research, according to FightSMA Chair Mike Calise. The winners were chosen from a field of promising young investigators ranging from recent PhD graduates engaged in post-doctoral fellowships up to assistant professors within the first two years of their appointment.
“We’re excited to make these awards for a second year,” said Bill Strong, co-founder of the Gwendolyn Strong Foundation. “Our goal was to bolster the existing SMA research landscape by ensuring that emerging talent and fresh approaches are encouraged and supported, and we continue to be inspired by the quality of grant applications received from major SMA research institutions.”
The three winners were announced at the annual FightSMA research conference held April 10-11, 2014 in Washington, D.C., each of whom will receive a $27,500 award. They are:
Barrington G. Burnett, Ph.D.
Department of Anatomy, Physiology, and Genetics
Uniformed Services University of the Health Sciences
Sara Custer, Ph.D.
Department of Dermatology
Indiana University School of Medicine
Faraz Farooq, Ph.D.
Apoptosis Research Center
Research Institute II
Children’s Hospital of Eastern Ontario
FightSMA (www.fightsma.org ) is a 23-year-old nonprofit organization dedicated to finding a treatment for spinal muscular atrophy (SMA), the leading genetic cause of infant death. The group has awarded research grants at more than 40 universities and research institutions in the United States, Canada, the United Kingdom, France, and Italy.
The Gwendolyn Strong Foundation (www.thegsf.org) is an all-volunteer, nonprofit organization dedicated to increasing global awareness of spinal muscular atrophy (SMA), accelerating research, and supporting families impacted by SMA and other life-altering conditions. The GSF motto: “NEVER GIVE UP.”
After a decade of advocacy on Capitol Hill, the fight against SMA passed a remarkable legislative milestone this past November with the enactment of the National Pediatric Research Network Act (NPRNA). Once implemented, the NPRNA will support the establishment of pediatric research consortia focused on basic, translational and clinical research, including multi-site clinical trials for rare pediatric diseases including SMA. Accordingly, FightSMA’s focus in Washington now turns to ensuring that the NPRNA is fully and expeditiously implemented by the National Institutes of Health (NIH).
As has become all too clear in recent years, bipartisan comity and legislative accomplishments are in short supply in Washington. That reality is part of what made passage of the NPRNA all the more remarkable. Resulting from the hard work of our champion lawmakers on Capitol Hill and the tireless efforts of families across the nation, the NPRNA was one of a small number of bills to make it to the president’s desk this past year with such overwhelming support.
The NPRNA began its legislative journey in the 113th Congress by passing the House of Representatives in February 2013 with the leadership of our sponsors, Representatives Lois Capps (D-CA) and Cathy McMorris Rodgers (R-WA), and the strong backing of Majority Leader Eric Cantor (R-VA), Energy and Commerce Committee Chairman Fred Upton (R-MI), and Energy and Commerce Committee Ranking Member Henry Waxman (D-CA). Each of these members of the House publicly fought for the NPRNA and all were instrumental in gaining the support of their colleagues.
On the Senate side of the Capitol, our lead sponsors, Sens. Sherrod Brown (D-OH) and Roger Wicker (R-MS), once again stepped up to the plate to reintroduce the NPRNA and build a bipartisan coalition of support, including the critical backing of Senate Health, Education, Labor and Pensions (HELP) Committee Chairman Tom Harkin (D-IA) and HELP Committee Ranking Member Lamar Alexander (R-TN). With that support in place, House and Senate leaders were able to include the NPRNA in a must-pass health package last fall, which proved to be the key to getting the bill to the White House.
On November 27, 2013, the NPRNA was signed into law in the Oval Office by President Obama.
Since the New Year, FightSMA has worked with our D.C. consultant team, Public Strategies Washington, to formalize a strategy for the next stage of our fight. Recognizing that NIH leadership will ultimately play the deciding role in how the NPRNA is implemented, FightSMA has refocused its efforts in recent weeks on collaborating with the research community and connecting with NIH staff to ensure that the benefits of the NPRNA are clear.
In mid-January, FightSMA Science Director Dr. Chris Lorson circulated a letter to the SMA research community sharing the news of the NPRNA’s passage. This letter was intended to give a full explanation of the law and to alert stakeholders of the potential for expanded research opportunities in the coming months and years. The letter was followed by a collaborative conversation within the community about the need to actively engage NIH leadership.
In February, FightSMA arranged a critical conference call with senior staff of the legislative and public policy office at the National Institute of Child Health and Human Development (NICHD). NICHD officials expressed the need for the SMA community to highlight the obstacles we face in developing a clinical trials network for SMA.
FightSMA worked with our partners in the research community throughout the month of March to formulate a response to NICHD’s inquiry. We are confident NIH will agree that providing adequate funding to establish the research networks called for by the NPRNA will be the most effective use of our federal resources to combat rare pediatric diseases like SMA.
We are also working with all of our Capitol Hill champions to generate direct congressional outreach to NIH. Senators Brown and Wicker are circulating a letter to their fellow NPRNA cosponsors urging NIH to implement the NPRNA in a timely manner, to engage the rare disease stakeholder community, and to provide robust funding for new pediatric research networks.
In mid-March, Congress passed the Gabriella Miller Kids First Research Act. This important law promises to devote resources (approximately $126 million over ten years) previously used to finance political conventions towards pediatric research at NIH. The enactment of the Kids First law provides us with an opportunity to take advantage of a potential new funding source. Although the Act was only recently signed into law by President Obama, we are already working hard to ensure that SMA is on the priority list as NIH and Congress decide how to appropriate and allocate this new revenue stream.
The 2014 Annual FightSMA Webcast will broadcast live at 6:30PM ET on April 11th.
Go to www.fightsma.org to watch the webcast live!
SUBMIT YOUR QUESTIONS NOW via secure email form
SUBMIT YOUR QUESTIONS LIVE via upcoming link on fightsma.org homepage
SUBMIT YOUR QUESTIONS ANYTIME via Twitter @FightSMA
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