thescientist | SMA occurs from having two copies of a mutated version of the survival
motor neuron 1 (SMN1) gene, which is responsible for the proteins that
maintain neurons related to muscle movement. Without proper signals from
the brain to move, muscles
begin to atrophy and cause a host of related problems, such as
decreased mobility and an inability to swallow. Many patients die by age
two, and applicants for the lottery must be under two years old. The
drug, given intravenously, provides the brain with
a functional copy of SMN1 through a viral vector.
Pharmaceutical giant Novartis
has begun accepting applications for a lottery-based program to give
away 100 doses of a gene therapy for spinal muscular atrophy, a
sometimes-deadly muscle-wasting disease that affects about 1 in 10,000
births. The initiative will provide access to children with SMA living
in countries where the intervention, Zolgensma, has not yet been
approved. But there are far more than 100 patients who could be
eligible.
The company has cited production limitations
as the reason for high treatment costs and limited doses for the
lottery. An independent bioethics committee worked with Novartis to
develop the terms of the lottery.
“It’s a difficult situation,” Ricardo Batista, the father of an infant with SMA who lives in Canada, tells The Globe And Mail. “It’s a lottery where we’re leaving children’s lives up to chance. I don’t think it’s a game that any of us want to play.”
