NIH Provides Congress an Overview of the Federal Government’s Spinal Muscular Atrophy Research Activities.

Post date: May 17, 2013 2:41:19 PM

February 24, 2012

NIH Provides Congress an Overview of the Federal Government’s Spinal Muscular Atrophy Research Activities.

As part of President Obama’s budget request for fiscal year (FY) 2013, the National Institutes of Health (NIH), the nation’s biomedical research agency, has provided Congress with a summary overview of its SMA-related research activities.  This information is included in the NIH’s budget justification materials and responds to Report Language included last year in the FY 2012 Labor, Health & Human Services, and Education Appropriations bill.

Please click here for the actual Report Language about SMA.

Specifically, the National Institute of Neurological Disorders and Stroke (NINDS) outlines its intention to fund a SMA biomarkers study as part of its NeuroNEXT initiative.  NeuroNEXT is a new government-supported enterprise that provides clinical trials infrastructure for research related to neurological disorders such as SMA.  The initiative closely mirrors, and was partially prompted by, a proposal included in the SMA Treatment Acceleration Act.  The SMA biomarkers study is the first NeuroNEXT initiative – development of a SMA biomarker will facilitate clinical trials for SMA therapies.  NINDS also summarizes its intention to enhance its funding of SMA-related translational research initiatives; such research is a top priority for Families of SMA since it helps to grow the drug pipeline.

Additionally, the National Institute of Child Health and Human Development (NICHD) provides an update on its newborn screening activities.  Implementing newborn screening for SMA is critically important to ensure that new therapies can be delivered presymptomatically.  Families of SMA has worked tirelessly since 2005 to encourage the NIH to support SMA-related newborn screening research and for the federal Advisory Committee responsible for establishing the newborn screening panel to include SMA.  NICHD is funding a study that is analyzing a new and improved SMA newborn blood test as well as working to develop necessary protocols for addressing the needs of infants testing positive for SMA.  Both components will be piloted in two states.  The data gathered from this pilot study will be critical in the effort to add SMA to the federal newborn screening panel – Families of SMA submitted an initial application to the Advisory Committee in 2008.  

NIH Also Releases 2011 Funding Amounts for Spinal Musuclar Atrophy Research.  

The National Institutes of Health (NIH), the federal government’s medical research agency, released its most recent estimate of funding for disease categories which indicates that its base funding levels for SMA-related research has increased from $10 million in fiscal year 2008 to $19 million in fiscal year 2011. 2012 and 13 funding levels are projected to be similar. 

Click here for the NIH report listing 2011 NIH funding levels for SMA Research. 

Part of the increase in federal resources devoted to SMA-related research is the result of the success of a two, long-term strategies at Families of SMA to provide seed funding for critical drug discovery programs in order to leverage federal (and private) resources, and to raise awareness of SMA and SMA-related research among federal policymakers through advocacy and grassroots efforts. 

Several recipients of NIH funding in fiscal year 2011 received their initial seed funding from Families of SMA. 

For instance, NIH supported two projects related to the Quinazoline Program to treat SMA, now being developed by Repligen Corporation and two projects related to the Tetracycline Program to correct SMN2 splicing, including  $1 million in funding in 2011 to Paratek Pharmaceuticals from a 5-year multi-million dollar award. 

The initial seed funding provided by Families of SMA played a critical role in positioning these four projects to receive highly-competitive NIH research dollars for the first time.  Leveraging our initial seed money into greater industry or government investment is a key goal at FSMA. 

Additionally, the NIH funded two grants in fiscal year 2011 to support the Families of SMA Annual Research Conference .

Source Families of SMA