Published:
BY SARAH O’DONNELL, EDMONTON JOURNAL AUGUST 13, 2013 7:34 AM
EDMONTON - St. Albert father Dane Sadownyk picked up his three-year-old daughter Aleena and “just hugged her” Monday morning when the family learned the Alberta government will fund a crucial treatment for her rare medical condition.
“It was an extremely emotional moment,” Sadownyk said. “I was so elated. It felt like I could come up for a breath of air. That’s what it felt like, that I can breathe again.”
Aleena’s family and their supporters have been lobbying Alberta Health for a month to approve treatment for Maroteaux-Lamy Syndrome, a rare disease that means she lacks glycosaminoglycan, an enzyme that helps break down cellular waste.
Instead, the waste builds up, restricting movements, damaging organs and clouding eyesight, among other serious health complications. Without treatment, sufferers see their life expectancy cut short. Naglazyme, a synthetic enzyme approved in the U.S. but not Canada, can help break down that cellular buildup.
Though not a cure, weekly infusions could help prevent Aleena’s symptoms from getting worse, the family’s supporters say. Four other provinces have agreed to fund the treatment for seven children with the syndrome — also known as mucopolysaccharidosis type VI or MPS VI — and Aleena’s family was pushing Alberta to quickly approve the expensive but critical treatment, which is expected to initially cost about $300,000 a year.
Sadownyk, who was in Connecticut Monday with his family attending a conference on MPS VI when they heard the news, said they are feeling immense relief. Aleena was diagnosed with MPS VI in April.
“Today is definitely a day we are joyful for her and look forward to the future,” he said.
Andrew McFayden, director of the Isaac Foundation, went through a similar struggle in Ontario when his son Isaac was diagnosed with MPS VI as a toddler and was the first to receive treatment in Canada. He stepped in to assist the Sadownyks with their case and said he shared their feeling of elation Monday.
But McFayden also said the happiness is mixed with frustration that it took so long and that the family’s supporters felt they had to mount a public campaign with the support of MLAs such as Wildrose health critic Heather Forsyth, after the family’s original funding application through the Alberta Rare Disease Program was denied.
“We’ve gone through this numerous times,” McFayden said. “To me, that’s a big problem there still hasn’t been a process put in place by Health Canada for provinces to deal with funding these rare diseases.”
Health Minister Fred Horne said Monday he signed off on the funding for Aleena’s treatment through Alberta’s Short Term Evaluative Drug Therapy program, instead of the Rare Disease program, because Aleena’s case involves a drug not licensed for sale in Canada.
Horne said he weighed several factors, including the clinical evidence, the rarity of the disease, affordability of the drug and the best interest of the patient. Public pressure was not one of those factors, he said.
“I think Albertans would expect their minister and government to make these decisions based on evidence and looking at each case individually,” Horne said. “I’m pleased it’s able to be a positive outcome in this particular case. But these kinds of situations are becoming more common in Canada and it’s because we have more drugs coming out every day and more and more of these drugs are geared to rare diseases.”
Horne said he plans to talk about the need for an orphan drug program — a term used to describe medications for rare diseases — with other provincial health ministers and federal Health Minister Rona Ambrose.
“It’s an issue where we really need to collaborate,” Horne said. “There are only going to be more of these situations in the future.”
Forsyth, MLA for Calgary-Fish Creek, said Alberta Health must work to make the provincial system easier to navigate for families who suddenly find themselves seeking help with a rare condition. “The whole thing is just convoluted,” said Forsyth, who said she was overwhelmed to hear Aleena will receive treatment. “I think they have to simplify things and make it easier for the public to understand.”
NDP health critic Dave Eggen said Alberta Health also needs to speed up the process. “I’m glad something moved,” the Edmonton-Calder MLA said. “But in the future I don’t want to see people’s health compromised by being run through the wringer again.”
With Aleena’s funding approved, the Sadownyks’ next learning curve will be tied to her treatment.
“This is something that is new territory for us,” her father said. “We’ll learn.”
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