Cure SMA, a nonprofit organization funding research into new treatments and a possible cure for spinal muscular atrophy (SMA), and providing SMA families with support, reports that the Senate’s Health, Education, Labor and Pensions (HELP) Committee has passed seven bills as part of its “step-by-step” approach to Innovation and 21st Century Cures. Bills approved by the committee now go to the full Senate for a vote.
The advocacy organization explains that the Senate’s bill-by-bill approach to the Innovation legislation contrasts with the comprehensive 21st Century Cures/Innovation legislative package approved in July by the House of Representatives. The various provisions of the 21st Century Cures act will be considered in the Senate as separate bills, with votes spread over February, March, and April.
CureSMA said that among bills passed on Feb. 9 is the Advancing Research for Neurological Diseases Act of 2015 (S.849), sponsored by Senators Johnny Isakson (Republican, Georgia) and Chris Murphy (Democrat, Connecticut), which would expand research on neurological diseases and potentially have a positive impact on SMA research. The advocacy organization applauds the Senate committee’s making headway on this legislation, but notes that the work is not done and it’s important to keep momentum going.
As noted, the next step for these bills will be their presentation to the full Senate. CureSMA requests that people contact their senators and urge them to support this legislation and bring it to a successful Senate vote, affirming the importance 21st Century Cures legislation could have in speeding up the discovery of next-generation SMA cures and treatments.
Cure SMA suggests using www.senate.gov/senators/contact/ to find contact information for the senators representing each state, and to urge them to expedite release and consideration of the Innovations for Healthier Americans Act. They also note that Americans can reach out to their senators through Twitter, by either asking a senator’s office for the lawmaker’s Twitter handle, or finding it using Twitter’s search function.
The group has posted samples of points supporters might address in writing or phoning their senators:
Congress describes the 21st Century Cures act, which has bipartisan support, as an initiative to bring America’s healthcare innovation infrastructure into the 21st Century. The act provides new hope for patients and their loved ones, and the necessary resources for researchers to continue efforts into new treatments and cures, and to speed the advance of healthcare innovation. In a statement, the legislators observe that, from the mapping of the human genome to the rise of personalized therapies linked to advances in molecular medicine, recent decades have witnessed constant breakthroughs that are changing the face of disease treatment, management, and cures.
However, while research is moving quickly, the lawmakers acknowledge that the federal system of drug and device approval hasn’t kept pace, and is in many ways a relic of another era, holding back scientists and leaders at agencies like the National Institutes of Health (NIH) and the Food and Drug Administration (FDA). They note that although some 10,000 diseases or health disorders are known, cures and treatments exist for roughly 500 of them. A House document also observes that despite recent science and technology advancements, designing and conducting clinical trials of new drugs and devices in the United States has become increasingly complex, time-consuming, and expensive, and the clinical trial process must modernize.
Spinal Muscular Atrophy (SMA)
SMA, which can affect any race and either gender, is caused by a mutation in the survival motor neuron gene 1 (SMN1) and robs people of physical strength by affecting the motor nerve cells in the spinal cord, gradually taking away the ability to walk and, eventually, to eat or breathe. SMA affects approximately 1 in 10,000 babies, and is the No. 1 genetic cause of death for infants, with about 1 in every 50 Americans a genetic carrier.
In a healthy person, the SMN1 gene produces a protein that is critical to the functioning of nerves that control muscles. Without it, those nerve cells cannot properly function and eventually die, leading to debilitating muscle weakness.
Cure SMA explains that Congress’s 21st Century Cures bill contains key provisions that could positively affect SMA patients and their families, including requirements for patient input into risk/benefit analysis, changes to the FDA evaluation processes, and $10 billion in mandatory additional funding for the NIH.