MPP demands help for ailing boy

LISA PRIEST
Thursday, May 11, 2006
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Two-year-old Isaac McFadyen sat quietly in a gallery of the Ontario Legislature yesterday as the Progressive Conservative health critic demanded that the provincial government pay for a costly drug to treat the toddler's rare disease.

Elizabeth Witmer, MPP for Kitchener-Waterloo, asked Health Minister George Smitherman to ensure bridge funding for the enzyme replacement therapy, used to treat Maroteaux-Lamy syndrome, until a national policy is in place.

Without it, the McFadyen family from Campbellford, Ont., will have to move to England where the government covers the drug Naglazyme, which costs a staggering $300,000 to $1-million annually.

"This [drug] can reverse some of the problems of this disease and may even prevent them altogether," Ms. Witmer said.

But Mr. Smitherman said he was in no position to say right then and there that Ontario would fund the drug.

"This is one of the more difficult circumstances that can be encountered by a family, of course, and by the challenges that it presents for a public health care system," Mr. Smitherman said. ". . .The case is obviously an individual case that we have to treat as such."

People like young Isaac are missing an enzyme that is needed to break down carbohydrates, which build up in the body's cells and affect many organs.

Signs of the disease, known as MPS VI for short, include stunted growth, enlarged tonsils and adenoids that cause breathing problems, poor mobility and dramatic changes in facial features, including a flat nose and large head. Once sufferers reach their teens, they often require heart-valve surgery.

Isaac has already suffered some of the effects of the disease: a piece of his skull and a portion of vertebrae in his neck were surgically removed last month after they began compressing his spinal cord. His corneas are clouding, his forehead protrudes and he has an umbilical hernia.

But in many ways, he is an average rambunctious toddler who, in a high-spirited moment, managed to give Mr. Smitherman a high five yesterday after Question Period.

In an interview, Isaac's father, Andrew McFadyen, said he was disappointed by Mr. Smitherman's response in the legislature. But he said that the Health Minister later told him he was going to work "vigorously on the file."

The drug Isaac requires, Naglazyme, is so expensive that even its U.S.-based developer and manufacturer, BioMarin Pharmaceutical Inc., has acknowledged no patient can afford it. It is the only drug available to treat the disease, which is so rare that it's estimated only three to 10 Canadians have it.

In most countries where Naglazyme is licensed, such as those in the European Union, governments cover the drug's cost. In the United States, it is largely funded through private insurance.

In Canada, there is no policy for "orphan drugs" like Naglazyme -- medications for rare diseases, the incidence of which varies by country -- and no way for people like the McFadyens to afford such costly therapies.

Because Isaac's parents cannot afford the drug, the boy is going without the treatment. The McFadyen family is planning to move to England to obtain the costly therapy. There, Isaac would be treated by Dr. Ed Wraith, at Royal Manchester Children's Hospital, who has said that at a minimum he hopes Naglazyme will halt the boy's disease process.

"We don't want to have to sell everything and go but we will," Mr. McFadyen said. "We just can't sit on this."

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