Leduc parents fight to get baby boy treatment in U.S. for rare neuromuscular disease
EDMONTON -- A Leduc couple says there is hope for their baby boy living with a deadly neuromuscular disease—but it comes at an enormous cost.
The journey began when Reign was four months old.
“We noticed he wasn’t moving like other babies and he had an indent in his chest—like it was concaved,” described Alex Johnston.
Eventually, doctors diagnosed Johnston and Ryan Fengstad’s son with spinal muscular atrophy, or SMA 1.
The disease affects the now nine-months-old’s ability to move, swallow, talk and breathe—as well as those abilities of one in 6,000 to 10,000 Canadians.
“Most of them don't live until two years old and they will need a wheelchair, communication boards, a lot of extra assistance,” Johnston explained.
Earlier this year, Alberta expanded coverage one of the drugs Reign needs, Spinraza, to include patients under the age of 18. At a millilitre unit price of $23,600, the treatment helps Reign’s body produce a protein he lacks.
It’s not a cure, but his parents believes a gene replacement drug recently approved in the U.S., called Zologensma, could help.
“It gives them the ability to start moving again, to gain head control, to be able to sit up, to be able to swallow,” said Fengstad.
Zologensma is only given to children under the age of two who still have enough muscle.
“The drug itself has been proven in clinical trials and (is undergoing) further studies to see long-term affects,” said Mina Tadrous, associate professor in the University of Toronto’s pharmacy department.
“As far as we know, it really helps cure the disorder and patients are able to live past what the regular life expectancy for this disorder would be.”
However, the price for the one-time injection is about CAD $2.8 million. Alberta does not offer coverage for it as Zologensma hasn’t been approved by Health Canada.
“We follow national standards when covering drugs and treatments to ensure they provide patients with better health outcomes,” Tom McMIllan, communications person with Alberta Health, told CTV News Edmonton.
“Market approval is the first issue,” Tadrous acknowledged. “The second issue is the price tag, and whether that's going to be paid for or not, even if it's available in Canada, it'll still have a pretty steep price tag.”
Alberta Health added physicians can apply on a patient’s behalf for help in obtaining services that aren’t available at home. However, Reign’s parents told CTV News Edmonton they applied for provincial support to go to the U.S. for the treatment and were denied.
They then set up a GoFundMe page, which had received more than $11,000 as of Sunday evening.
As hard as it has been for them to watch their son struggle, Reign’s parents believe there is potential for him and thousands of others to be helped.
“Gene replacement therapy has the potential to heal so many different ailments and diseases,” Fengstad told CTV News Edmonton.
“This isn't just about our son. We want this gene replacement therapy brought into Canada. It could help thousands of people.”
With a report from CTV News Edmonton’s Sarah Plowman