SUNDRE — A fundraiser to help cover the cost of the world’s most expensive drug for a local business owner's great-nephew who was diagnosed with an extremely rare condition is on until until mid-February.
While the provincial government announced yesterday (Jan. 27) that families of Alberta children suffering from spinal muscular atrophy (SMA) may now be eligible to receive funding for gene replacement therapy treatment, it is not immediately known if Max Sych will qualify.
Max was born in Fairview, Alta. on Jan. 11, 2019 four months prematurely weighing only one pound and 11 ounces. Despite arriving in the world requiring three months of neonatal intensive care at Royal Alexandra Hospital in Edmonton, he eventually began to improve and was even meeting all of the milestones according to his adjusted age.
“He was doing really well, he was standing and so on,” said Max’s great-uncle Derrick Calvert, president of Dirk’s Auto & Marine in Sundre.
But fate took another unexpected turn. At about 14 months, his motor skills began to deteriorate and he could no longer stand or walk with assistance. When an MRI came back negative for cerebral palsy, which is common in preemies, his family searched further for answers.
Last November, Max was seen by a neurologist, who discovered he had the rare condition of SMA type 2, a genetic disease that was not connected with his premature birth. One in 10,000 children are born with the disease. Left unchecked, the disease leads to a progressive loss of muscle strength that detrimentally impacts one’s ability to walk, breathe and swallow.
And although recent medical breakthroughs paved the way to treat the condition, the new gene therapy drug is said to be the most expensive on earth, with a price tag of $2.8 million for just one dose. The silver lining is that single dose is enough to save a life with a one-time treatment. Approved in 2019 by the U.S. Food and Drug Administration, Zolgensma was just given the green light by Health Canada in December.
“Most — because of the cost — don’t make it,” said Calvert, adding there is a secondary treatment option that is more of a temporary stop gap measure.
“It prolongs everything a little. But you don’t have a great quality of life, and not a long one,” he said, explaining that alternate path involves a spinal shot every four months.
But as of late last week, more than $1.1 million had already been raised through a province-wide effort that has included a number of bottle drives in small towns like Sundre and Fairview to large cities like Calgary and Edmonton.
“It is incredible — the people of Alberta have really come together for Max,” said Max's mom Bryarly Parker Sych, who is a town councillor in Fairview, although she does not anticipate seeking re-election in October as a result of the challenging circumstances. She and husband Bowden, a pharmacist, also have a three-year-old daughter named Sawyer.
“The charity has been just incredible. The compassion that’s been pouring in is just wonderful — I’m speechless, I don’t know what to say,” said her uncle Calvert, who felt compelled to pitch in.
Inspired by a successful bottle drive in Calgary that raised more than $40,000, he decided to inquire if the Sundre Bottle Depot would be willing to facilitate a local fundraising effort.
“They were accommodating immediately,” said Calvert, adding the owners and staff were all enthusiastic about helping.
“They’re just a fantastic bunch,” he said.
Anyone who is interested simply needs to drop off a load of bottles and tell the staff the proceeds are for Mighty Max. Alternatively, people can also deliver their deposit-back recyclables at Dirk’s Auto, he said.
“I’ll haul them down. Yesterday, I hauled two heaping pickup trucks full,” he said during an interview on Jan. 20, adding close to $2,000 had been raised locally to date and that the fundraiser was extended until Feb. 15.
For the time being, Max is doing well and receiving the spinal injections of a drug called Spinraza, his mom said, last week.
“Right now, he’s in what’s called his loading doses. He gets four loading doses,” she said.
“He gets an injection every four months for the rest of his life.”
However, this treatment is more “like a band aid” than a cure, she said, explaining the drug feeds a back-up gene that provides proteins for motor neurons.
“With SMA, they’re missing a gene that makes protein to feed your motor neurons,” she said.
But even though Spinraza kick starts a back-up gene called SMN2, that only provides about five per cent of what an average person requires, she said.
“So, it’s quite minimal what it’s doing, but it’s also a huge help. It’s incredible that there’s the ability that they have this, because a few years ago, there wasn’t even access to any of this sort of stuff,” she said.
“Zolgensma will stop the progression, and Spinraza slows it down,” she added.
The provincial government announced on Jan. 28 it is working with Novartis Pharmaceuticals Canada Inc. to provide interim patient access to Zolgensma. Alberta children with SMA and who may become ineligible for the treatment while waiting for the final approval processes to be completed, will be considered for funding on a case-by-case basis.
Due to the progressive nature of the disease, the condition gets a little worse every day. That means the sooner Mighty Max receives Zolgensma, the better, she said.
The drug’s manufacturer says the treatment is most effective for children under the age of two. While Max technically celebrated his second birthday earlier in January, his mom remains optimistic that he’d still qualify.
“We’re hoping that if the age does come into play, that they will take into account that he was born so premature,” she said, adding his birthday would have been in May had Max not been born four months premature.
Mighty Max’s journey can be followed on social media as well as by visiting www.mightymaxsych.org.
Although the last time Calvert saw his great-nephew was the summer before last during a family reunion at Nakusp along the Upper Arrow Lake in B.C., the uncle said, “He is something happy — he’s just a happy, happy, happy baby. It’s just unbelievable with the challenges that he’s been going through, and the spirit that he has is incredible.”
The grateful mother expressed her appreciation “to the Sundre community for coming together for someone they don’t really know. It just shows how great the communities are in Alberta, and how Albertans are just willing to help anyone.”
