On December 23, 2016, the FDA approved a drug treatment for Spinal Muscular Atrophy (SMA). Two days before Christmas. I wished the timing was different, a month earlier or later would have been better. So close to Christmas, the approval felt more like a gift to a well-behaved child rather than the culmination of decades of research funded by hundreds of millions of dollars of private and public capital. “A Christmas miracle!” exclaimed so many mothers wrote on Facebook. Families and patients rejoiced.
SMA is a degenerative neuromuscular disease that affects the strength and endurance of an individual’s muscle groups. In the most severe cases, diagnoses are accompanied by cruelly short life expectancies. SMA is the number one genetic killer of babies and children under two years old. I was diagnosed when I was two and am now twenty-three. I am part of a modest cadre of adults with SMA, and an even smaller cadre of adults with SMA living independently.
Fortunately, more individuals than ever with SMA are reaching adulthood. The approval of Spinraza (nusinersen) in December 2016 announced the beginning of a new hopeful chapter. Unlike other available drugs, Spinraza treats the underlying cause of SMA, rather than the symptoms. In clinical trials, children who shouldn’t have lived past one year are now celebrating their third and fourth birthdays. Babies are breathing unassisted. Toddlers are walking. The drug does appear to be a modern miracle.
A miracle with a $750,000 price tag. For the first year. And only $350,000 for the following years.
Spinraza, developed by Biogen, is administered through spinal injection multiple times a year. Last summer, The Washington Post investigated the nexus between Biogen and insurance providers and the economic potential of developing drugs for so-called “orphan diseases.” The article detailed one family’s experience battling their insurance provider and navigating Biogen’s own “zero-dollar co-pay program.” Their son, Max, has shown marked improvement and the family has yet to pay out of pocket for treatment. While I would like to hope insurance and other federal programs will continue to cover Max’s treatment, I am not optimistic.
Because the drug was fast-tracked and most test subjects were very young, there is no data on how long patients will need Spinraza injections. As more families rely on Spinraza, more children with SMA with reach adulthood. It is imperative for the parents of these children to concern themselves not only with their child’s immediate medical needs, but also the political and social futures their children will inherit.
In January 2017, one month after Spinraza’s approval, the current administration began dismantling the federal programs and protections that people with disabilities rely upon to live healthy, full and independent lives. The Affordable Care Act. The Americans with Disabilities Act. Medicare. Medicaid. The Supplemental Nutrition Assistance Program. For many adults with disabilities, these programs are indispensable. Only 24.8% of individuals with ambulatory disabilities were employed in 2016. Over 20% of individuals with disabilities live below the poverty line and rely on the government programs currently under attack.
No specific data is available on what those percentages look like amongst the adult SMA community. Nevertheless, it is reasonable to speculate that, as a result of Spinraza’s success, more individuals with SMA will reach adulthood and need to take advantage of government programs in order to pay for medical treatment, housing, and other essential costs of living. I hope, in twenty-odd years, when Max is twenty-six and forced off his parent’s insurance, he can rely on government assistance when a $350,000 bill arrives with his name on it.
In May 2018, Cure SMA, the leading SMA research and family support organization in the country, conducted its yearly D.C. lobbying session. Cure SMA receives funding from Spinraza and is primarily populated by parents of children with SMA. Cure SMA lobbied for increased federal funding for research and pre-natal screening. There was no publicized effort to save federal programs that will be vital to their children’s success later in life.
In truth, Spinraza is not a miracle. It is lifesaving science born out of research funding by private and federal actors. The success of children, teens and adults with SMA is not and will not be a miracle either. I have been living independently for two years and attended a four-year university across the country from my home city. The past six years have been rewarding beyond measure and challenging in ways neither I nor my parents could have predicted. And there will be more barriers, but I, my family and friends are fighting to protect the rights of persons with disabilities and programs upon which we rely.
The parents of children with SMA cannot champion immediate medical advances while ignoring a future that will threaten their children, or the miracle of Spinraza may begin to feel more like a curse.
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