Alberta family race to raise $2.8M to treat son 'Mighty' Max's rare disease
Max was diagnosed with type two of spinal muscular atrophy (SMA) in November
Max Sych turns two years old next month, but he's already lived up to the nickname Mighty Max.
Born nearly four months premature, he survived a brain bleed and bedside surgery while living his first three months in a neonatal intensive care unit at the Royal Alexandra Hospital in Edmonton.
Now, a month after he was diagnosed with a rare genetic neuromuscular disorder, his northern Alberta family says he needs more than might.
They are trying to raise $2.8 million to pay for the world's most expensive medicine.
Zolgensma, a gene-therapy treatment for spinal muscular atrophy (SMA), was greenlit by the U.S. Food and Drug Administration in May 2019, with Japan and the European Union following suit. While it's undergoing a priority review, it has not been approved or funded in Canada.
Max was diagnosed with type two of SMA in November. Without effective treatment, he could lose his ability to stand or walk, and possibly develop trouble eating and breathing. The more aggressive type one condition can be fatal before the age of two.
"I don't know how it's possible to charge something like that for a kid to walk, or to breathe or to eat without a tube," said Bowden Sych, Max's father.
"I can't think of a parent that if they had the option to give their child the ability to walk that they'd say no. We're going to do everything we can to make that happen for him."
Max qualifies under Alberta health insurance for the drug Spinraza, the only SMA treatment approved by Health Canada. Studies show the spinal fluid injections delivered every four months are 40 to 50 per cent effective, but the benefits may decrease with age.
He starts his treatment on Tuesday, but the family is hoping to raise enough money to secure a dose of Zolgensma, delivered in a single injection.
The online fundraiser has raised over $455,000 as of Sunday from 2,500 donors. In their town of Fairview, about 115 kilometres north of Grande Prairie, some farmers have offered to harvest a plot toward Max's causes. A number of NHL players have lent their support on social media.
"He's the happiest little guy. He's clever and he's sneaky. Everybody who gets to meet Max, they love him," said Bryarly Parker, Max's mother. "We have to believe that Max is here to show his purpose, and what we can do, and what he can accomplish, and to give people some hope, I think."
But time is working against them. In the European Union, the medication is authorized for children who weigh less than 21 kilograms. In the U.S., it's only authorized for children under the age of two.
Max turns two on Jan. 19.
"We're trying to move a very large mountain for our little boy," said Bryarly, a local town councillor.
'Another huge mountain'
Max first received the Mighty Max nickname from a nurse at the Royal Alex as he recovered from the premature delivery, Bryarly says.
When he returned to Fairview at three months old, he began to hit his developmental milestones and show signs of a healthy recovery. But this spring, Bryarly and Bowden noticed troubling changes. Max stopped standing up without help. The muscles he'd slowly built over the year started to atrophy.
During a routine prematurity checkup at the Glenrose Rehabilitation Hospital, he was examined by a neurologist. A series of tests confirmed the type two SMA diagnosis.
"I think the first couple days, I don't want to say grieving, but we had to hear that news and our son had already been through so much," Bowden said. "To find out that we had to go through this with him, another huge mountain."
SMA affects one out of every 8,000 to 10,000 people worldwide, according to the U.S. National Institutes of Health.
As the owner and operator of the local pharmacy, Bowden says it pains him to know the medication he wants most for his son is one he can't provide.
"It's extremely frustrating that that's what I do every day for a living and there's absolutely nothing I can do about it," he said.
The family has looked into options for U.S. treatment, a plan the family says is complicated by the pandemic and costs for a hospital stay, on top of the drug price. They are trying to find a neurologist willing to deliver Zolgensma through a special access program, which sees Health Canada grant the use of non-marketed drugs in exceptional circumstances.
They don't have to look far for a glimmer of hope. Kaysen Martin, a two-year old Edmonton boy with the more aggressive type one SMA was able to secure a dose earlier this month. Kaysen's family raised over $1 million through an online fundraiser, and then a mystery donor paid for the rest, according to the family's social media account.
"Miracles do happen," the family wrote.