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
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.