Advertisement

Alberta to cover treatment for spinal muscular atrophy on case-by-case basis

Click to play video 'Alberta family cautiously optimistic following Zolgensma funding announcement' Alberta family cautiously optimistic following Zolgensma funding announcement
WATCH: An Alberta family is hopeful their two-year-old son will be eligible to receive a potentially life-changing treatment. As Tracy Nagai reports, the province announced on Wednesday that children diagnosed with spinal muscular atrophy would be approved to receive Zolgensma on a “case-by-case” basis – Jan 27, 2021

The Alberta government said Wednesday it had reached an agreement with the manufacturer of Zolgensma, a drug used to treat the rare illness spinal muscular atrophy, or SMA, to fund the treatment for some children in the province.

Health Minister Tyler Shandro said this new agreement will allow the government to provide children and their families the drug, instead of waiting for the final steps in the Canadian approval process.

“I’m very aware of the urgency of making this treatment available and pleased that we are able to provide this funding for children who may slip through the cracks while waiting for the final review and final approval processes,” Shandro said.

Click to play video 'Alberta announces interim access to Zolgensma for children suffering from spinal muscular atrophy' Alberta announces interim access to Zolgensma for children suffering from spinal muscular atrophy
Alberta announces interim access to Zolgensma for children suffering from spinal muscular atrophy – Jan 27, 2021

Read more: Health Canada approves $2.8M treatment for spinal muscular atrophy

Story continues below advertisement

Zolgensma, a gene therapy medication, was approved by Health Canada in December, however, Shandro said the standard drug review and negotiating processes are still not complete, which means families are still waiting to see how they can access it for their children.

Click to play video 'Mighty Max: Update on young Alberta boy fighting extremely rare disease' Mighty Max: Update on young Alberta boy fighting extremely rare disease
Mighty Max: Update on young Alberta boy fighting extremely rare disease – Jan 17, 2021

Zolgensma is a one-dose medication that would replace the faulty gene at the root of the SMA disorder.

Shandro said Alberta is still working with the national health tech agency and the pan-Canadian Pharmaceutical Alliance to come up with a long-term plan to provide access to Zolgensma in Alberta.

For the time being, Shandro said specialists and physicians will be given information to help them and families determine which children are eligible to receive the treatment now and apply for it.

Read more: Alberta family hopes to raise $2.8M to give toddler treatment for spinal muscular atrophy

Story continues below advertisement

SMA is a disease that affects roughly one in 6,000 babies born, and attacks their muscular system, affecting their ability to develop and maintain motor skills like neck and head stability, sitting up, crawling, walking and even swallowing.

Click to play video 'Alberta children with spinal muscular atrophy eligible for interim access to Zolgensma' Alberta children with spinal muscular atrophy eligible for interim access to Zolgensma
Alberta children with spinal muscular atrophy eligible for interim access to Zolgensma – Jan 27, 2021

Shandro said the illness is often diagnosed when children are between three and six months old, and is detected when a baby doesn’t kick as vigorously as they should, or can’t bear weight on their legs without help.

“I can’t imagine the stress and hardship of families with a child diagnosed with SMA. As parents we want what’s best for our children,” Shandro said.

“We want them to have healthy development and have every opportunity available to them.”

For the family of Max Sych, also known as Mighty Max, it’s a glimmer of hope not just for them, but also for the other families they advocate for.

Story continues below advertisement

The family doesn’t yet know if Max will be eligible for the drug, but they’re looking into starting the approval process.

“It’s really, really exciting and it’s a huge win for the SMA families,” Max’s mom Bryarly Parker said Wednesday.

Read more: ‘Support and love totally outweighs’ theft from bottle drive: Mighty Max family

No matter what happens, she says they will keep advocating for treatment for the illness.

“It won’t end with Max. For me as a parent, what I’ve gone through at the NICU and now this — I won’t stop until there’s newborn screening. I won’t stop until every family that wants their child to have access gets this medication,” Parker said.

“We’re not going to stop fundraising, we’re not going to stop our advocacy until literally Max is in the hospital and I just see that Kangaroo pump push that IV into Max.”

Suzi Vander Wyk with Cure SMA Canada said this news will bring hope to families struggling with an SMA diagnosis, adding that the illness doesn’t just impact the child, but the entire family unit.

“These families are going from a mindset of fear of what the future holds for their children and knowing that this clock is ticking, to one of planning the future and making plans for the entire family,” she said.

Story continues below advertisement

Vander Wyk said  because Zolgensma is a new treatment, it’s too early to know the long-term results for children who receive it, but said it does have “an astounding impact on those babies.”

Read more: 2 Alberta toddlers receive life-saving drug thanks to angel donor and pharmaceutical lottery

She said if a child is diagnosed with SMA during newborn screening, before anyone can tell they have the illness, and they receive the treatment right away, there are no signs of SMA as they grow.

“Children that have been treated a little bit later, we know that there are examples of them not being able to move their arms and legs and suddenly they’re rolling off a couch and sitting on the floor and reaching for things, and with this treatment, we know that it gets better and better with time,” Vander Wyk said.