The national nonprofit spinal muscular atrophy (SMA) support and advocacy organization Cure SMA has launched a grassroots campaign to have required newborn-screening for SMA in every state.
The campaign was announced at the 2017 Annual SMA Conference held June 29-July 2 in Orlando, Florida. It was attended by more than 2,200 researchers, clinicians, and individuals from families affected by SMA.
At the conference, SMA families were introduced to the screening campaign and how they can engage with political representatives and healthcare authorities to advocate for SMA screening in every newborn.
“For many years, families whose children were diagnosed with SMA were told that there was no approved treatment and little that could be done to help them. After decades of work by our community, that has changed,” Cure SMA President Kenneth Hobby said in a press release.
“We now have an approved treatment, multiple other promising treatments being tested in trials, and strong clinical evidence that beginning treatment as early as possible is of the utmost importance. Newborn screening is the best opportunity we have to change the course of SMA for the next generation and beyond.”
The launch of the SMA screening campaign coincides with the recent passage by the Missouri State House and Senate of a bill authorizing statewide SMA screening, signed into law by Gov. Eric Greitens on July 10, making Missouri the first state in the country to require screening of all newborns for SMA.
The legislation was sponsored by Missouri state Rep. Becky Ruth, who was on hand at the SMA conference to share her experience with SMA advocates.
“As a grandmother who lost a grandson to a rare disease, I know how important it is to give these kids every chance to be happy and healthy, and potentially save their lives,” Ruth said. “I was proud to sponsor legislation in Missouri that will make us the first state to start screening for SMA. I can’t wait to support and cheer on these families as they work to bring screening to their states, too.”
Hobby said Cure SMA is “thrilled that Missouri, through Representative Ruth’s leadership, has taken this important step. We envision Missouri becoming the model that other states can follow as we work to move SMA newborn screening forward.”
The SMA newborn screening campaign follows in the wake of the U.S. Food and Drug Administration (FDA)’s December 2016 approval of Spinraza (nusinersen), marketed by Biogen and developed by Ionis Pharmaceuticals for use in a broad range of SMA patients.
In clinical trials of the drug — the first approved treatment in the U.S. for SMA — infants treated before showing symptoms achieved more motor milestones such as head control, sitting, ability to kick in supine position, rolling, crawling, standing, and walking, than those who began treatment after systems developed, supporting the early initiation of the drug.
Cure SMA notes that the most severe forms of SMA cause rapid motor neuron loss, thus providing only a small window for optimal treatment — a frequently lost opportunity due to delays in diagnosis that newborn screening could help avoid.
And Cure SMA said that while each state screens newborns according to its own list of tests, they receive guidance from the federal Recommended Uniform Screening Panel (RUSP), a list of disorders screened for at birth and recommended by the Secretary of the Department of Health and Human Services (HHS) as part of their state universal newborn screening (NBS) programs.
In May, Cure SMA reported an advisory committee of the HHS is reviewing SMA for inclusion to the RUSP.
Cure SMA says it intends its grassroots advocacy campaign to run alongside the RUSP evidence review process, maximizing the opportunity to move SMA screening forward on both state and federal levels.
Grassroots campaign advocates in several states will focus on their state and federal legislators to ensure that appropriate funding is maintained for newborn screening. Cure SMA says that while a successful RUSP application will strongly support its case for required screening, it is not necessary to wait for a federal decision to continue advocating on the state level.