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Harrison Ford's Extraordinary Measures

In his latest movie, the actor and producer brings to life the story of a father's quest to cure his children of Pompe disease.

Searching for a Pompe Disease Cure continued...

Ford, who in addition to starring in the film served as its executive producer, felt the story was intensely compelling. "A father who gives up so much of his life to devote himself to finding a cure for what ails his kids is something I was attracted to. But I was also attracted to the elements of the story showing the difficulty of bringing a drug to market."

Looking for tips for coping with a child's chronic illness? See "When A Child is Chronically Ill."

To deliver such a drug, Crowley found himself working against a ticking clock. The life expectancy of Pompe patients diagnosed as infants is, at most, nine years, although most children die within the first year of birth from heart or respiratory complications. He knew the secret to creating a treatment in time for Megan and Patrick, who were growing weaker by the day, would be found among academic researchers with cutting-edge -- if seriously underfunded -- theories. "Failure wasn't an option," the real Crowley tells WebMD.

Ford acts opposite Brendan Fraser and Keri Russell, who play the Crowleys, but science is the real star here. "People with Pompe disease are missing an enzyme in their cells that breaks down glycogen [a storage form of glucose, or sugar]," says Hung Do, who, in addition to holding a PhD in medical biochemistry and genetics, also served as the official scientific adviser to the film -- and was one of the original members of the research team on which Crowley gambled. "All that glycogen builds up within the body's cells and leads to the many biochemical and physical problems associated with the disease."

Enzyme replacement -- a therapy that delivers the missing enzyme into the body's cells, thus enabling the drug to break down glycogen and allow cells to function normally -- was the goal. "But getting the enzyme inside the cell to the proper internal location is difficult," says Do. "You've got to deliver the enzyme with the proper packaging -- wrap it with a bow, if you will -- in a way that a cell agrees to accept the enzyme. And that's what our research focused on."

Developing Enzyme Therapy

"I knew nothing about any of this when I first started," Crowley says. "Once the seed money was in place and the science [to develop an effective enzyme therapy] was down, my initial foray into the biotech industry was two steps forward, one step back. But I think my 'never quit' attitude made up for my lack of experience."

Clearly. As the film movingly depicts, developing a therapy from the innovative thinking of scientists -- represented by Ford's character -- was merely step one. Just as progress was being made -- in real and reel life -- Crowley ran out of money. He was forced to sell his fledgling company to a bigger biotech firm, Genzyme, whose leaders welcomed his continued management of his research team.

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