Clock ticking on Edmonton family's hope for $2.8M drug treatment for toddler son

·3 min read

Kaysen Martin has a machine to help him breathe while asleep, another to help him cough, and every four months, the wheelchair-bound tyke heads to the hospital to have a drug injected into his spine.

Now almost two, Kaysen was diagnosed at eight months with spinal muscular atrophy (SMA), a motor neuron disease found in one in 6,000 babies that affects the voluntary muscles used for lung support, swallowing, crawling, walking and head control. Most children with the Type 1 version affecting Kaysen don't live past age two.

The lumbar injection of a drug called Spinraza is "the reason why he's here with us today," but his parents are hoping Kaysen can be fast-tracked for a new drug that would be one treatment for the rest of his life, says his mother Lana Bernardin.

But there are catches.

The drug, a gene-therapy called Zolgensma, isn't yet approved in Canada thought the United States' Food and Drug Administration gave it the green light in 2019.

Not eligible after 2nd birthday

The single injection costs $2.8 million, making it the one of the most expensive drugs in the world.

And he needs to get it before he celebrates his second birthday on July 17, Bernardin told CBC Radio's Edmonton AM on Friday.

"It's not a cure. It's the closest thing to a cure that they have so far," she said.

The drug is delivered as a live virus, which means that as a child gets older and heavier, the dosage must be increased to levels that may not be safe.

Supplied by Lana Bernardin
Supplied by Lana Bernardin

"The main thing with SMA is that the earlier babies are caught with the disease, before a lot of damage is done, and get some sort of treatment, the better," Bernardin said.

"There are babies out there that have received Zolgensma within weeks of being born and they are living a normal life. They're walking, they're eating on their own, they're breathing on their own."

Norvartis, the company that makes the drug, has applied to Health Canada for approval but the process can take up to a year, she said.

Bernardin and Kaysen's father Mark Martin say they have exhausted every avenue to get him the treatment.

Kaysen has already been approved under the federal government's Special Access Program, which approves individuals with serious or life-threatening conditions to use drugs that are sold or distributed in Canada, she said.

'Our last option, our only hope'

The access is critical as it allowed him to be entered into a Novartis "lottery," which offers up to 100 compassionate treatments each year to eligible children outside of the U.S. who are under age of two and have spinal muscular atrophy.

"You can enter your kids … and then every two weeks there's two kids drawn. And this is worldwide, though, so it's still a really slim chance," she said.

After trying since January to get Kaysen into the draw, his parents were finally successful on June 19 — giving him just one shot at the lottery before he's too old.

Alberta's Health Minister Tyler Shandro has agreed to meet with the family to talk about the situation. A statement to CBC News said Shandro sympathizes with the family's situation and that the case is being reviewed

The Spinraza drug, which Kaysen would need to continue taking for the rest of his life, costs $375,000 per year, Bernardin said. She is hopeful that Alberta Health Services will approve the funding — and the additional quality of life for her son.

"It's been a lot of uphill battles and, you know, kind of just taking one step in front of the other," she said.

"This now is our last option, our only hope."