Pompe disease hits screens in "Extraordinary Measures"

Wed Jan 20, 2010 2:02pm EST
 
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By Toni Clarke

BOSTON (Reuters) - "Extraordinary Measures", starring Harrison Ford and Brendan Fraser, opens on Friday and 101 employees from Genzyme Corp will gather in a Boston movie theater to see themselves portrayed in what some fear may be an unflattering light.

The movie chronicles the story of John Crowley, currently the chief executive of Amicus Pharmaceuticals In, in his race to find a cure for Pompe disease, a rare muscle disorder that threatened to kill two of his three children.

For the staff at Cambridge, Massachusetts-based Genzyme, which eventually developed a treatment, the movie has special meaning and opens as the biotechnology company struggles to emerge from its worst year ever.

Last year, a manufacturing crisis caused it to close its Boston-area manufacturing plant after it strained capacity to breaking point. Ironically, it was the company's rush to ramp up production of the Pompe drug, called Myozyme, that led to the crisis.

The movie is loosely based on the book "The Cure" by Wall Street Journal reporter Geeta Anand. It describes the emotional drama of the Crowleys' personal struggle to save their children, and the scientific drama of the drug's development.

"This is our lives played out on screen, and we want to experience it together," said Lori Gorski, a Genzyme spokeswoman.

Pompe disease affects some 5,000 to 10,000 people worldwide, and often kills babies before they reach the age of 2. People with the disease are deficient in an enzyme known as acid alpha-glucosidase, which is responsible for breaking down glycogen, a form of sugar stored in muscle cells. When glycogen builds up in these cells it can cause swelling of the heart and other organs and lead to disability and death.

SCIENTIFIC BATTLE   Continued...