Fabry’s disease sufferers rally at Queen’s Park

“TORONTO — Patients suffering from a rare and fatal genetic disorder that ravages vital internal organs said Thursday the Ontario government will leave them to die if it does not immediately start paying for treatment they believe would save their lives.
Fabry‘s disease sufferer Darren Nesbit pleaded with Health Minister George Smitherman to approve funding of enzyme-replacement therapy.
“I’m not ready to leave this world yet,” the Sarnia, Ont., man wrote in a heart-wrenching letter he handed to the minister on Thursday.
But Smitherman told him the earliest he and Ontario’s 30 other estimated Fabry’s patients can expect a decision would be in September, when provincial health ministers meet with the federal government to discuss drug funding.
“We think it’s incredibly important that the federal government stand in its place and play a role in helping to offer appropriate funding for these orphan drug challenges,” Smitherman said.
However, he reiterated expert opinion advising against public funding of such treatment _ which includes the drug Fabrazyme _ citing a lack of scientific evidence that it works.
The latest recommendation came last month from an independent panel called the Common Drug Review, which provides advice to federal and provincial governments.
But Nesbit, who is believed to be the first in Canada to undergo experimental trials of the drug, has no such doubts.
“Whether you believe in the science or not, I will bet my life on Fabrazyme before I bet my life on anything else,” said Nesbit, who has felt crippling pain in his hands and feet since ceasing treatment about two months ago.
“I’ll die knowing that it works.”
Nesbit, diagnosed 10 years ago, said he believes cost is the real reason the provincial government won’t approve funding for the treatment. It costs $300,000 annually for a single patient.
“I said, `What’s my life worth to you?”‘
Australia, Japan, the United States and some countries in Europe pay for enzyme-replacement therapy.
“Every genetic doctor around the world is laughing at Canada right now. They think we’re a joke because we won’t pay for this,” Nesbit said.
Genzyme Canada, which produces Fabrazyme, recently stopped providing the drug to patients for free. It wants provincial governments to start picking up the tab because the drug was approved for use last year by Health Canada.
But ministry officials say that approval only ensures the drug’s safety to enter the marketplace and doesn’t test its clinical efficacy.
In Fabry’s patients, an enzyme deficiency causes fats to accumulate in blood vessels, leading to damage of internal organs such as the heart and the kidneys. It also causes acute pain, often in the hands and feet.
It’s estimated between 150 and 300 Canadians have been diagnosed with Fabry’s disease.