Genzyme role in Pompe disease struggle hits theaters

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

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

The race for a cure was fought out among scientists at four companies, including Genzyme. Between 1998 and 2002, Genzyme teamed up with a trio of companies and had acquired rights to their experimental drugs.

The company''s first partnership was with Pharming Group NV, a company based in the Netherlands that was developing an enzyme using milk from hundreds of transgenic rabbits. But rabbits are hard to milk, presenting production hurdles.

"We figured we would have needed a herd of 70,000 rabbits to produce enough enzyme to meet demand," Robert Mattaliano, who was in charge of Genzyme''s internally developed Pompe disease product, said in an interview.

Genzyme also partnered with a research group from Duke University that was using cells taken from Chinese hamster ovaries (CHO), and in 2001 it acquired Novazyme, a company that John Crowley -- played by Brendan Fraser in the movie -- helped form based on the work of Dr. William Canfield, the scientist who is played by Harrison Ford. Genzyme also had its own product.