Alberta may cover $2.8M cost of gene therapy for babies with rare condition

A temporary move by the Alberta government to cover the cost of a life-changing new gene therapy is a sign of hope for the northern Alberta family of a little boy dubbed Mighty Max.

A Wednesday announcement that the province will foot the bill for a $2.8-million, one-time treatment on a case-by-case basis is no guarantee that two-year-old Max Sych will receive the therapy for his rare condition, spinal muscular atrophy (SMA).

"We're not going to stop what we're doing," said Max's mother, Bryarly Parker. "We're not going to stop fundraising, we're not going to stop pushing, because we don't know if he'll be approved or not."

Family, friends and businesses across Alberta have held silent auctions, made and sold hats and hair scrunchies, collected bottles and peddled doughnuts to raise a collective $1.2 million toward the cost of Zolgensma, an infusion that would give Max a working copy of his defective gene.

Supporters have been hustling to raise money since November, when Max was diagnosed with SMA after his toddling turned to crawling and other developments appeared to regress.

Although Zolgensma needs to be administered before a child is two to be effective, Max was born four months premature, and would be eligible to receive the therapy until the two-year anniversary of his due date this May, his mom says.

For children who will qualify for coverage, Alberta's move is huge news, says Susi Vander Wyk, executive director of Cure SMA Canada.

The progressive disease interferes with muscle function, preventing people from walking or sitting, and even interfering with swallowing and breathing in some cases. About 1 in 6,000 babies are born with the genetic condition.

A handful of Canadian children who have received Zolgensma have made astounding progress, Vander Wyk said in an interview.

One child who couldn't talk or swallow is now sitting up without help. Another baby who couldn't move his arms or legs is now lifting up objects and can roll off the couch, she said.

In December, Health Canada approved Zolgensma for use in young children with SMA. It's the first treatment available with the promise of substantially altering the course of patients' lives.

Ontario was the first provincial government to announce it would pay for the pricey treatment on a case-by-case basis, and on Wednesday, Alberta Health Minister Tyler Shandro said his government would follow suit.

No final decision on drug coverage

It's an interim measure while the Canadian Agency for Drugs and Technologies in Health (CADTH) studies whether provincial and territorial governments should pay for the treatment. The independent agency's recommendation is non-binding, but helps guide provincial decisions.

In an email, a CADTH spokesperson said the organization made a confidential recommendation about coverage in December, and drug manufacturer Novartis has asked for reconsideration. They did not say when they expect to release a final decision on coverage.

Shandro said some children need intervention now and can't wait for administrative reviews.

Alberta Health says they hope to see a CADTH recommendation by spring. The pan-Canadian Pharmaceutical Alliance would then negotiate a purchase price, which could take about another 90 days.

Although Shandro didn't know how much any new coverage would cost, Alberta Health says there are 23 children in the province receiving treatments for SMA.

Vander Wyk, of Cure SMA Canada, says she hopes other Canadian provinces and territories follow Alberta's lead.

"From a patient's perspective, the clock is ticking very, very loud," she said. "The fear of the future and what it holds for their children is not able to be measured."

Nearly two-thirds of children with SMA have type 1, the most severe form of the disease, she said. Untreated, most of those children will not live past age two.

Vander Wyk said covering the drug is a good investment, as SMA patients may require intensive and expensive health care throughout their lives.

Max, who shuffles his little arms and legs to slowly clamber up stairs and move around, has type 2 SMA, but also faces a difficult future without treatment.

His mother says his doctor is in the process of applying to the government for Zolgensma treatment. If the province agrees to cover the cost, the family will talk with donors to see if they want their money back, or whether they could use the funds to help another family in similar straits.

Parker, Max's mom, says it's nice to see recognition that SMA doesn't have to be a terminal disease.

"We're not going to stop until Max is fully dosed," she said. "And even then, I'll be turning my help to other families that still require assistance in this fight."