FightSMA, an all-volunteer, parent-led nonprofit, reported that the U.S. Senate Appropriations Committee has, as part of the Fiscal Year 2017 Defense Appropriations bill, included for a first time spinal muscular atrophy (SMA) as a research topic in the Defense Department’s Peer-Reviewed Medical Research Program.
FightSMA calls this welcome development the result of months of advocacy by its researchers and SMA families, and their extensive outreach to key senators on the committee. The Defense Appropriations bill now heads to the full Senate for the next stage in the legislative process.
“Up until today, spinal muscular atrophy was not able to compete for this category of funding,” Martha Slay, a FightSMA co-founder, says in a release. “Now the door has been opened to a whole new level of funding from a completely new source.”
The bill, approved 30-0 with broad bipartisan support, allocates a total of $34 billion to the Defense Health Program, which provides medical services for military personnel and their families, supports continued advancements in medical research, in modernizing and maintaining medical infrastructure, and in the implementation of next-generation electronic health record-keeping and retrieval. That amount includes an additional $915 million for medical research efforts, including $300 million for the competitively awarded, peer-reviewed medical research program and $225.9 million to advance Defense Department medical research priorities.
Should the FY 2017 Defense Appropriations bill be approved, as expected, SMA research projects will be eligible for consideration by the DOD’s Office of Congressionally Directed Medical Research Programs (CDMRP), which has provided more than $9.7 billion in medical research support over the past 25 years.
FightSMA (then called Andrew’s Buddies) was founded in 1991 by Joe and Martha Slay after their son Andrew was diagnosed with SMA (spinal muscular atrophy), with a mission to “strategically accelerate” research to treat or cure SMA, the leading genetic cause of infant death. FightSMA has funded research at more than 50 universities and research institutions in the United States, Canada, the United Kingdom, France, and Italy. Celebrating its 25th year in 2016, FightSMA was an early supporter of efforts to find the SMA gene, and has also funded high-throughput screening for SMN gene upregulation, SMA mouse model research, and SMA gene therapy research, among other projects. In 2013, FightSMA played a leadership role in the successful passage of the National Pediatric Research Network Act.
Spinal muscular atrophy is caused by a missing or defective motor neuron 1 (SMN1) gene, which results in reduced levels of a particular type of protein essential to healthy development of motor neurons, needed to breathe, swallow, walk, and run. Insufficient levels of SMN protein are responsible for the loss of motor neurons within the spinal cord, leaving muscles to atrophy.
It is estimated that SMA affects 1 in every 11,000 children, and currently has few treatment options. Consequently, there is a high unmet need for new therapeutics to address SMA symptoms, and slow or stop the disease’s progression.
FightSMA says its level of optimism is higher than ever. The organization was chosen by the National Institutes of Health (NIH) as a model for translational research because SMA, among all known neurological disorders, is now closest to a treatment or cure breakthrough.
SMA research, the organization notes, also offers invaluable scientific insight of potential benefit to other neurological diseases, such as ALS, Alzheimer’s, Parkinson’s, muscular dystrophies, some forms of cancer, and even spinal cord injury.
“We look forward to working closely with Congress as the Defense Appropriations bill works its way toward enactment and with the Department of Defense as it develops its fiscal year 2017 research agenda,” says Martha Slay, a former mezzo-soprano opera singer. After Andrew Slay was diagnosed as a 1-year-old, Slay shifted her energy and talents to generating funding for SMA research.
As FightSMA president, Martha Slay has reached out to other SMA parents eager to bring the fight to their own communities. With the support of these people, many of whom serve as FightSMA board members, Slay has kept fighting for a quarter-century, and in addition to fundraising has worked for more than 15 years on Capitol Hill as an SMA research advocate.
FightSMA, Inc. a 501c3 tax-exempt organization, relies on charitable contributions — donations and fundraisers — to support its research and advocacy programs. FightSMA also receives money through annual corporate sponsorships and grant partnerships with supportive groups, but does not receive any state or federal funding.
United States Senate