CAMPBELLFORD, Ont. – An Ontario family is lobbying for provincial funding so their two-year-old boy can get treatment for his rare and debilitating enzyme deficiency disease.
And they’re doing it with the help of another Ontario family who went through the same fight five years ago.
Seven-year-old Isaac McFadyen from Campbellford, Ont., is one of eight people Canada diagnosed with Maroteaux Lamy Syndrome (MPS VI). Only 1,100 worldwide have the progressive disease. Symptoms include stiffening joints, stunted growth, heart and airway disease, spinal cord compression and a shortened life span.
The McFadyens also know how much treatment has helped Isaac – a treatment he gets only because of provincial funding, which they had to lobby to get.
About five years after Isaac’s diagnosis, a Palmerston, Ont., family is going through a similar experience. Darren and Pam More’s two-year-old son Jasper was diagnosed with MPS VI six weeks ago. They learned Monday that Jasper was denied funding to receive the treatment.
“It’s shocking in our province right now that this is taking place. We do have precedent set with Isaac and it’s virtually an identical case,” said Andrew McFadyen, Issac’s father. “To me, it’s a travesty and it’s not representing equal access to health care for everybody.”
However, Health Minister Deb Matthews noted the laws in Ontario changed in 2006 to prevent politicians from choosing which drugs do or don’t get covered. Now, the decision rests with an executive officer who relies on a panel of experts that weights medical evidence.
“However, I do want to learn more about this and I need to understand the basis of the decision,” Matthews said.
While there’s no cure for MPS VI, there is the life-sustaining treatment available called Naglazyme, which provides patients with a synthetic version of the enzyme they are lacking by infusing small doses of it into the bloodstream.
The treatment can cost anywhere from $300,000 for a small individual to $1 million for a young adult each year.
“Studies are showing right now that kids that get on this treatment at a young age, basically where Jasper is, it gives them a better opportunity to prevent some of the side effects that come with the disease,” McFadyen said.
“Children diagnosed before age of three are thought to have a severe case and need to be treated immediately in order to prevent a lot of difficult things disease can inhibit in children.”
Naglazyme is only available to Canadian patients through the federal government’s Special Access Program. The only provinces funding the treatment are Ontario, British Columbia and Quebec.
Isaac has been receiving the treatment once a week for five years at the Hospital for Sick Children in Toronto.
Told of the More family’s circumstances, Ontario Premier Dalton McGuinty said his heart goes out to the family in their difficulties, but that he had no first-hand knowledge of the case and could not comment. He suggested getting in touch with Health Minister Deb Matthews.
Matthews echoed the premier’s sentiments.
“I certainly certainly understand why this doesn’t sit right with Ontario families. I am following up to learn more about this drug and this particular situation,” she told QMI Agency on Wednesday. “I do know how important this is, and we want to do the right thing.”
Matthews said she will urge the ministry to examine the latest evidence on naglazyme.
The Mores issued an appeal to the rejection of funding on Tuesday.
The McFadyens established The Isaac Foundation in 2006 to raise money for innovative research projects that aim to find a cure for MPS VI. They also help families touched by the disease.
– With files from Sheena Goodyear