An Edmonton family is asking the province to fund a life-saving drug for their toddler.
Nearly two-year-old Kaysen Martin was diagnosed with spinal muscular atrophy Type 1 (SMA 1) when he was eight months old. It’s a progressive motor neuron disease that causes his muscles to weaken over time.
“We have a lot of appointments every week — then the therapies, going to the gym to help build muscles,” his mom, Lana Bernardin, explained.
About one in 6,000 babies are born with SMA. Most children with SMA 1 do not live to see their second birthdays.
“After his diagnosis, the first few months were really hard,” Bernardin said.
“We were actually the first family in Alberta to get Spinraza funded through the government.”
Martin goes for those Spinraza treatments every four months.
“He probably would not be here with us today if it wasn’t for that drug,” Bernardin said.
Spinraza is not a cure, however. Martin would need to keep going for those treatments and it costs $375,000 a year.
“Sprinraza is a lumbar puncture,” Bernardin said. “That in itself has a lot of risks, including hitting nerves — then as he gets older and gets scoliosis, it would be a harder procedure to do.”
His family is now trying to get access to a new gene therapy called Zolgensma — a one-time treatment.
“The difference between the gene therapy and what he’s on now is that this gene therapy actually… replaces that dysfunctional gene that causes this neuromuscular disorder,” said Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders.
“It would be a game-changer for Kaysen and his quality of life,” Bernardin said.
The price tag, however, is $2.8 million and time is running out. The drug needs to be administered before Kaysen turns two and he’s only a couple of weeks away from that.
The family is asking Alberta Health to step in and pay for the treatment, even starting a petition to support Kaysen’s fight.
“He got a miracle once he got access to the Spinraza, so what we’re looking for is that second miracle,” Wong-Rieger said.
The Canadian Organization for Rare Diseases said in the long run, the province will save money if it pays for the Zolgensma treatment.
Roughly five years of Spinraza will cost the same as the one-time administration of Zolgensma.
The province said it is reviewing the family’s request alongside Alberta Health Services, but Health Minister Tyler Shandro said options are limited.
“Zolgensma has not yet received Health Canada approval, or completed the required national drug review and approval process,” he said in a statement Thursday.
“I recognize the challenges this family is facing and their wish to explore all available options for their son as they face this terrible disease.”
The family says there’s a Special Access Program granted by Health Canada that allows patients in Canada to access a drug before it’s officially approved in Canada.
Through the program, Martin could get the drug without Health Canada approving it, as long as Alberta Health agrees to fund it.
When asked if the province would consider funding the treatment, a spokesperson with Alberta Health said that was part of its review.
The province noted Zolgensma has been granted priority review status and a decision about whether to approve it is anticipated near the end of 2020.