Family hopes lottery for $2.8 million treatment will give son a normal life
Published Monday, January 13, 2020 5:53PM EST
KITCHENER -- Families who have babies with a rare genetic disorder may soon have access to a near-cure, but only if they're lucky enough to win a lottery.
A family from Milton is hoping for just that.
The Vasey family is eagerly awaiting a draw that would give their one-year-old son, Wyatt, access to treatment for his spinal muscular atrophy type one, or SMA.
SMA is a neuromuscular disorder that attacks the motor nerves, causing a loss in muscle growth and strength.
Baby Wyatt can't stand on his own, crawl or sit—everything he does needs help, his parents explain.
Now a new drug is available to treat his disorder, but only by way of lottery: otherwise, it costs $2.8 million.
"It's the closest thing to a cure, they say now. So I guess, every parent wants the best and if they're saying this is the best right now, why not fight for this as parents?" explains Christine Vasey.
The family submitted their name into a worldwide lottery that a pharmaceutical company is offering.
The drug, Zolgensma, was recently approved in the United States. It's a gene replacement therapy and only needs to be taken once.
"You're almost curing these kids, so you're stopping the nerve loss at such a level that they're going to have pretty normal function, or at least converting a kid whose going to die at two years of age to someone who has a pretty much normal, if not normal life span," explains McMaster University professor Mark Tarnopolsky.
It's not available in Canada, though, which is why it's being offered by lottery: it offers a free dosage to kids under two in countries where Zolgensma hasn't been approved.
There is another drug that treats SMA that is available in Canada called Spinraza. It's a drug that's administered by spinal tap to help slow the progression of the disease.
It helps expand the life expectancy for those living with SMA, but it carries its own risks.
"We have to subject him to further lumbar punctures, which you do four times a year until you're 18," Wyatt's father Brandon explains. "That's a lot of risk."
There's also a chance that Wyatt could end up wheelchair-bound for his whole life.
Tarnopolsky says the Zolgensma is given through an intravenous infusion rather than those punctures. The fact that it's only taken once—as opposed to 72 times for Spinraza—is another bonus.
The Vasey family has started an online fundraiser to help pay for the treatment. They say they'll pay out of pocket if they're forced to in order to help give Wyatt a normal life.