Pompe disease hits screens in "Extraordinary Measures"

BOSTON Wed Jan 20, 2010 9:14pm EST

Cast members Brendan Fraser (L) and Harrison Ford pose together at the premiere of CBS film's ''Extraordinary Measures'' at Grauman's Chinese Theatre in Hollywood, California, January 19, 2010. REUTERS/Danny Moloshok

Cast members Brendan Fraser (L) and Harrison Ford pose together at the premiere of CBS film's ''Extraordinary Measures'' at Grauman's Chinese Theatre in Hollywood, California, January 19, 2010.

Credit: Reuters/Danny Moloshok

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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

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 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.

Genzyme also partnered with a research group from Duke University that was using cells taken from Chinese hamster ovaries. 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.

Finally, Genzyme chief executive Henri Termeer demanded that his researchers stop bickering.

"How many more babies have to die?" he shouted from the balcony of the company's boardroom.

In late November 2001, Genzyme decided to run an experiment to figure out which program to focus on.

A review of the data revealed Genzyme's product was the most efficacious and the easiest to manufacture. The other programs were discontinued and in 2006, Myozyme was approved in the United States and Europe.

But the Pompe story was far from over.

The U.S. Food and Drug Administration still has not approved a scaled-up version of the drug, which will be known in the United States as Lumizyme. That means there is still not enough of the commercial product to go around.

Gorski thinks some employees might be hurt at the way Genzyme (called Zymagen in the film) is portrayed -- as a somewhat cold bureaucracy -- and said the real story is far more dramatic than any movie.

"Putting 20 years of work into 90 minutes is a hard task that can never be put on screen," she said.

(Reporting by Toni Clarke, editing by Maureen Bavdek)

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