Heroes, Hope and Hollywood

How a father led a quest to discover a treatment for his critically ill children, and ended up the subject of a major motion picture

Article Highlights:
  • John Crowley, whose two children have Pompe disease (acid maltase deficiency), lead a quest to develop a treatment for the life-threatening disease.
  • The Crowley family story has been adapted into a major motion picture, Extraordinary Measures, starring Harrison Ford, Brendan Fraser and Keri Russell.
by Tara Wood on December 31, 2009 - 1:22am

QUEST Vol. 17, No. 1

Most anybody who hears John Crowley’s story will call him a hero.

It’s a logical label for a man who led a quest to develop a treatment to stop the disease killing two of his young children.

The Crowley family’s incredible story has been chronicled in the media, a book and now a major Hollywood feature film. “Extraordinary Measures” will be released Jan. 22 with an A-list cast that includes Harrison Ford, Brendan Fraser and Keri Russell.

But even when Crowley pauses to look back at the experience, he balks at the word “hero.”

Grab a sneak peek of the movie with the official trailer from CBS Films.

Read more of John Crowley's thoughts about his quest to save his children and the ongoing drug development process. 

“As a business person, I did what I was asked to do, and as a dad I did what I had to do. That certainly doesn’t make you a hero,” he says. “The kids and Aileen are the heroes.”

From desperate to determined

Aileen and John Crowley with their kids in a recent family portrait: Megan, left, and Patrick, right, six years after their Myozyme treatments began, are active sixth and seventh graders in a New Jersey public school. The oldest child, John III, does not have Pompe disease. (Photo: Brian Doben)

In 1998, when Megan Crowley was 15 months old and her brother Patrick just 7 days old, John and Aileen learned the babies had Pompe disease, also called acid maltase deficiency. Pompe is caused by a deficiency in the enzyme that breaks down glycogen, a type of sugar.

The resulting buildup of glycogen causes profound muscle weakness throughout the body. The couple struggled to grasp the grim reality: There was no treatment and their babies were at great risk of dying before the age of 2. Thankfully, their oldest son, John III, did not have the disease.

Frustration and desperation mounted as the Crowleys also learned there was limited — although promising — research into Pompe, an “orphan” disease affecting very few people that offered little financial incentive for pharmaceutical companies to pursue a cure. (See “MDA’s role“ below.)

The disease took a growing toll on the children’s health. Within a few years, they both were on ventilators, and Megan had experienced a near-fatal bout with pneumonia. The Crowleys’ desperation turned to determination and they decided to take the lead in the quest to find a cure.

Armed only with the support of Aileen and the assurance that his children wanted to continue to fight their disease, John Crowley left the security of his job at Bristol-Myers Squibb and started a biotech company to develop a treatment for Pompe disease.

Making their own miracle

While his family remained on the East Coast, Crowley became CEO of Oklahoma City-based Novazyme. Under his leadership, the company made significant advances in enzyme replacement therapy for Pompe disease. Novazyme's research led to its purchase by pharmaceutical giant Genzyme. Genzyme then developed and brought to market the lifesaving Pompe drug, Myozyme, which received FDA approval in 2006. (Myozyme is not based on Novazyme's drug therapy candidate, however, says Genzyme spokesperson Maureen Stellwag.)

Reviewing a scene from the film are, from left, producer Michael Shamberg, Crowley, lab technical adviser Dr. Hung Do and producer Stacey Sher

Meagan and Patrick began receiving Myozyme infusions in clinical trials in January 2003. After just 12 weeks, tests showed the drug transformed their enlarged hearts and livers back to normal size. Myozyme saved their lives.


Almost six years later, Patrick is now 11 and Megan is 13. They’re sixth and seventh graders at public school, use wheelchairs and ventilators, and enjoy very full, upbeat lives. They receive Myozyme infusions every other week.

“It’s a lifelong infusion. It’s extended and enhanced their lives,” Crowley says.

They’ll join their parents and big brother on the red carpet at premieres in Los Angeles and New York this January, as Hollywood tells the story of how their dad led the effort that saved their lives.

John Crowley will be portrayed in the film by Brendan Fraser, and Aileen by Keri Russell. Harrison Ford, whom Crowley calls the “driving force of the film,” optioned the film rights to the story several years ago. He plays a composite character representing researchers and physicians with whom the Crowleys worked over the years.

Crowley on the set with Harrison Ford, the “driving force” of the film. Ford optioned the film rights to the story several years ago.

The Crowleys trusted director Tom Vaughn, and producers Michael Shamberg and Stacey Sher from day one. The team spent countless hours getting to know the Crowleys; screenwriter Bob Jacobs even spent a few weeks living with the family.

“They included us every step of the way. We talked at least weekly for years,” Crowley says.

The actors — who are “very, very down to earth,” Crowley says — also spent personal time to understand their characters.

Russell joined Aileen at home for a day to get a firsthand look at her routine, and Fraser visited several times (“the kids think he’s awesome”), including a day with Crowley at work to see what it’s like to run a biotech company.

Both actors are parents — Fraser has three sons, and Russell has a toddler — and understood the story from a parent’s perspective.

 “It was very important for us and the producers and the studio that it not be a sad, heavy film,” Crowley says. “And it’s not — it’s really very funny in parts. It’s very uplifting.”

Along with laughter and joy, it captures the different realities and challenges that families like theirs experience daily.

“Keri and the nurse come rolling down the ramp, and they have a big goofy handicapped van. It takes 20 minutes to get everybody settled and into the van,” he says. “Most families just hop in the minivan and go.”

Coinciding with the movie release in mid-January is a rerelease of the book detailing their journey, The Cure: How A Father, John Crowley, Raised $100 Million — and Bucked the Medical Establishment — in a Quest to Save His Children, by Pulitzer Prize-winning journalist Geeta Anand. The re-release will contain a new epilogue and cover.

Aileen Crowley, on the set with Brendan Fraser, left, who portrays her husband in the film, and Tom Vaughan, the film’s director. The actors were “very down to earth,” and spent time in the Crowley’s home, getting to know them.

The Crowleys also are writing their personal memoirs about the experience, Chasing Miracles: The Crowley Family Journey of Strength, Hope and Joy, which is due out at the same time.


Hollywood is fond of happy endings, but the Crowleys know that, while their story is dramatic, it’s not over. (Visit their family Web site, www.crowleyfamily5.com.)

The drug the kids take is an effective treatment but not a cure. There’s still much work to be done, and John is excited by the potential.

“After the kids started to make improvements with Myozyme and we realized that wasn’t the final answer, we wanted to keep driving science toward new therapies for human genetic diseases,” he says.

About five years ago, he became CEO of Amicus Therapeutics, then a small start up, now a 100-person, public company in New Jersey.

Amicus focuses on small-molecule pills known as “pharmacological chaperones,” designed to target and enhance patients’ defective enzymes. The company is undertaking studies for Fabry disease and Pompe, as well as work that may translate into discoveries for other neuromuscular diseases. 

He still might not agree, but Crowley likely will have to get used to hearing the word “hero” attached to himself. In fact, Ford — Indiana Jones himself — called him a hero in a recent TV interview.

“It’s very humbling. I told him ‘thank you.’ I know, and I think he knows, I’m not the hero,” Crowley says. “I get up in the morning, shower, shave and I’m ready to go. Megan and Patrick have to get up two hours before school to have their breathing treatments, their physical therapy, get dressed with their nurses and Aileen helping, and get in their wheelchairs, just to get to school. And they want to go to school.

“I mean, that makes you a hero.”

Editor's note: This article was changed on Jan. 6, 2010, to clarify that Genzyme's  Pompe drug Myozyme is not based on the drug therapy technology the company acquired when it purchased Novazyme. For more on the development of Myozyme, see the January-March 2009 Quest article "Rescued Lives," and the March-April 2003 Quest article "A Killer Yields to Modern Medicine."

Watch the official trailer (courtesy of CBS Films)

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