Canadians living with a rare genetic disease are counting on a new Hollywood film opening Friday to draw attention to their debilitating and often deadly condition.
Extraordinary Measures, starring Harrison Ford and Brendan Fraser, is based on the true story of a determined American father named John Crowley, who set out to develop a drug to treat Pompe disease after two of his children were diagnosed with it.
People with Pompe disease are missing the enzyme that helps the body break down sugar. Without that enzyme, glycogen accumulates in the muscles, damaging them until they no longer work, and eventually the person can't move or breathe on their own.
When Ian MacPherson, a Hamilton, Ont., resident was diagnosed with Pompe disease at 2 1/2 years old, his family was told to enjoy their time with him because he likely wouldn't live to see his 30th birthday. He's 29.
Every other week, MacPherson, who uses an electric wheelchair and breathes with a tracheotomy tube, receives a drug called Myozyme through an intravenous line.
It truly is a lifeline for him, MacPherson said.
"If it was not for Myozyme I would not be here. That is the plain truth," he said in an interview. "Before Myozyme I was pretty much waiting to die."
Myozyme is made by a company called Genzyme, which acquired the company formed by Crowley and a scientist named William Canfield, played by Ford in the film.
Crowley and Canfield developed an experimental drug for Pompe disease but Genzyme ended up manufacturing a different one that its own researchers developed in collaboration with other scientists.
MacPherson has been on Myozyme for five years and, while it's not a cure for the disease, it helps stop the progression, and in some cases it can reverse some of the damage.
MacPherson and his family attended the Toronto premiere of Extraordinary Measures last week.
"It's a very uplifting movie and it depicts Pompe very well," he said.
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