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Alberta family raising $2.8 million for baby to receive cure for spinal muscular atrophy in U.S.

Click to play video 'Leduc family fighting for treatment for baby’s rare genetic disorder' Leduc family fighting for treatment for baby’s rare genetic disorder
WATCH ABOVE: An Alberta family is fighting for treatment for their baby boy Reign, who has been diagnosed with a rare genetic disorder. The only problem is, the treatment is considered one of the most expensive in the world. Su-Ling Goh has more in this edition of Health Matters – Jan 29, 2020

A family from Leduc, Alta., is fighting for a treatment for their baby who has a rare genetic disorder.

Alex Johnston and Ryan Fengstad are the parents of 10-month-old baby Reign, who was diagnosed with spinal muscular atrophy 1 (SMA1), which is also known as Werdnig Hoffman disease.

The disease essentially causes his muscles to waste away.

“As a parent, you see your child comes into this world, you have all these ideas and thoughts and dreams about what you’re going to do,” said Fengstad.

“That sort of came crashing down when we received the diagnosis.”

Ryan Fengstad, with 10-month-old Reign.
Ryan Fengstad, with 10-month-old Reign.

Most patients die before the age of two when their lungs fail.

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“The amount of pain and stuff that he’s been through… he’s just done so well,” said Johnston.

Reign’s disease progression is currently being slowed by the drug Spinraza, which started to be covered by Alberta Health last year.

READ MORE: Life-changing drug Spinraza still not covered for many Canadians with spinal muscular atrophy

Now, the family has found a new treatment that could help Reign — but there’s one major issue. Not only is it currently not approved in Canada, but the treatment is also considered one of the most expensive therapies in the world.

“We found out it was $2.8 million,” said Fendstad. “It was deemed the world’s most expensive therapy.”

Johnston and Fengstad hope to take Reign to the U.S. so he can be treated with the therapy, called Zolgensma.

It’s a one-time injection that replaces the patient’s missing gene so their body can make the protein it needs. It has to be administered before age two for it to be effective.

READ MORE: Ontario family hoping to raise $2 million for potential life-saving drug for 2-month-old daughter

“I understand that there’s a lot of research and a lot of money that goes into [Zolgensma] to make it possible,” said Johnston.
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“But the whole point of making it possible is to save lives, isn’t it?”

Alex Johnston hopes Reign will be able to receive a $2.8 million treatment that would cure his spinal muscular atrophy 1.
Alex Johnston hopes Reign will be able to receive a $2.8 million treatment that would cure his spinal muscular atrophy 1.
“[Zogensma] was priced cost-effectively in the U.S. at approximately 50 per cent less than the 10-year current cost of chronic SMA therapy,” read a statement from Novartis, the company that produces the drug.

“As we prepare to bring this treatment to Canadians, we are committed to working collaboratively with regulatory, pricing and reimbursement authorities in providing eligible patient access as quickly as possible.”

Novartis is also running a lottery-like program for patients outside of the U.S., giving away 100 doses for free for those in countries it isn’t yet approved in.

However, Reign’s parents are raising money to fund the treatment because they simply don’t have the time to wait for it to be available in Canada, and they can’t guarantee he will receive one of the free doses.

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“The cost of it is overwhelming,” said Johnston. “But there’s hope.”

The family is hoping to raise money for the treatment through a GoFundMe page they launched.