VANCOUVER -- A family hoping to fundraise the $3 million cost of a new drug to treat their baby daughter has seen donations skyrocket over the past few days: $800,000 has been donated in just one week, pushing the total to just over $1 million.
Two-month-old Lucy van Doormaal has a neuromuscular disorder called Spinal Muscular Atrophy 1, or SMA1. The rare genetic disorder affects Lucy’s motor neurons, “reducing her ability for activities such as sitting, crawling, head and neck control, and swallowing,” her parents wrote on a GoFundMe page they set up.
If left untreated, the baby may not reach her second birthday.
Lucy’s father, Scott van Doormaal, said he and his wife have been using every social media platform available to spread the message, and it’s starting to pay off.
“We’re optimistic with one week’s worth of fundraising,” van Doormaal said. “It’s literally all we’ve been doing, all day, all night.”
The effort isn’t just for Lucy, but for other families who are affected by the genetic disorder, van Doormaal said. That includes Edmonton toddler Kaysen Martin, whose family is also fundraising to cover the cost of the new drug, called Zolgensma.
Since media stories first appeared about Lucy and Kaysen, the issue has caught the eye of celebrities like Ryan Reynolds. The Vancouver-raised actor donated $5,000 each to fundraisers for the two children.
B.C.'s health ministry currently covers one treatment for SMA called Spinraza. The other treatment the Van Doormaals are considering, called Zolgensma, was submitted to the Common Drug Review on June 25.
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Both drugs are expensive: a single dose of Zolgensma costs around $3 million, and it’s often referred to as the most expensive drug in the world.
But Spinraza costs about $750,000 for the first year and about $350,000 after that, said Dr. Durhane Wong-Rieger of the Canadian Organization for Rare Disorders.
That makes the eye-popping cost of Zolgensma actually cheaper than a decade of similar treatment on Spinraza, she said.
Children who are able to take Zolgensma early in their lives have better outcomes than those on Spinraza, according to van Doormaal, such as not being as reliant on feeding tubes to help them eat and ventilators to help them breathe.
Lucy is on both a feeding tube and a bipap ventilator at night, said van Doormaal. Since taking Spinraza, the baby girl has shown improvement in being able to move her arms and legs.
However, Zolgensma is not covered in Canada because it is still being reviewed by the national Common Drug Review. B.C.’s health ministry says it needs to wait for that review to be completed before the Van Doormaals can request PharmaCare coverage for it.
Van Doormaal said he has every faith that the drug will eventually be approved in Canada, just like Spinraza was, and will help other families like his.
But in the meantime, the young father is focused on “the pricetag they’ve set on Lucy’s life.”.
With files from CTV News Vancouver’s Alyse Kotyk, Jon Woodward and CTV News Edmonton
