Lottery Underway for One Hundred Doses of the Most Expensivist...,

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.”