Titles can be deceiving, in a way. Take the titles of a new film and book about Princeton’s Crowley family, “Extraordinary Measures” and “Chasing Miracles,” respectively. While both chronicle this family’s struggle with a devastating genetic disorder, Pompe disease, which afflicts two of the Crowley’s three children, both titles divert attention from what really characterizes this family — a determination to maintain normality and treasure the ordinary.

When Megan Crowley, now a seventh grader at the John Witherspoon Middle School, was a year old, her parents noticed that their very bright little girl was not developing physically as she should. So the doctors did a muscle biopsy, which revealed that the 15-month-old girl had a rare and fatal genetic disorder, Pompe disease, that causes a deficiency in the enzyme that breaks down glycogen. Its buildup in the body causes extreme muscle weakness. Pompe is related to muscular dystrophies and to other neuromuscular diseases like Lou Gehrig’s disease, or ALS. Megan’s infant brother, Patrick, now a sixth grader at John Witherspoon, also turned out to be afflicted. And big brother, three-year-old John, had attention-deficit hyperactivity disorder.

But what might have been a recipe for disaster in many families was viewed as an opportunity by the Crowleys. They saw it as a chance to create normality and maintain it with great determination despite children who could neither eat, walk, nor breathe on their own; to raise money and start a company to work on treatments for Pompe; and to create happy memories together every single day.

One person fascinated by this story was movie actor Harrison Ford who, with producers Michael Shamberg, Stacey Sher, and Carla Santos Shamberg, was looking for compelling material for a film project. After reading an article in the Wall Street Journal and later a book, “The Cure: How a Father Raised $100 Million — and Bucked the Medical Establishment — in a Quest to Save His Children,” both by Geeta Anand, about John Crowley’s quest to save his children, Ford recognized the power of their multifaceted story.

The film he ultimately brought to the screen, “Extraordinary Measures,” which opens on Friday, January 22, is a somewhat fictionalized, foreshortened version of the Crowley’s odyssey. In the film John Crowley (Brendan Fraser), the newly minted MBA who drops his corporate job to save his children by finding a cure for Pompe disease, is one of two central characters. Playing opposite him is Dr. Robert Stonehill (Harrison Ford), a brilliant but unappreciated and unconventional scientist and a composite of the different scientists and researchers who ultimately came up with a treatment for Pompe. To further focus the story’s dramatic tension and shape the film, its director, Tom Vaughan, shortened the span of time covered by Anand in “The Cure.”

Having a movie made about their family is probably much easier to assimilate for the Crowley’s, who face huge challenges on a daily basis — from siphoning mucus from their children’s lungs several times a day to lugging around their equipment-ridden children (respirator, wheelchair) to having nurses around 24/7 to emergency hospital visits.

Nonetheless being on a set where hundreds of extras were recreating events from his family’s life was an incredible experience for the family, Crowley writes in his introduction to “Chasing Miracles.” The 300-pound wheelchair used by actress Meredith Droeger was an exact replica of the one that his daughter Megan uses — except that it has a hole in the seat so that Droeger could actually stand in it so that she did not lose circulation. What struck Crowley powerfully one day on the set was the contrast between the actress, who was perfectly normal physically, and his daughter, Megan. “I watched them film a long scene,” he writes, “then the actress playing Megan jumped up to stretch her legs. I had to look away. It was so very bittersweet.”

Crowley will appear at Barnes & Noble MarketFair on Saturday, January 23, at 3 p.m. He and his wife, Aileen, will also introduce the 1 p.m. screening of “Extraordinary Measures” at MarketFair. In his book Crowley describes the process of taking the whole family out to Oregon to watch the movie being filmed. After describing the authenticity of the set and health paraphernalia, he talks about a scene where his wife, Aileen, is loading the children into their van. “Something Aileen and I accomplish on a daily basis without a second thought really does look like an ordeal when acted out by others,” he writes. “We watched that one scene being filmed over and over for four hours, and it was very strange for the kids to see in such stark relief how difficult it can be simply to get moving in the morning.”

The one thing the family was really concerned about while the movie was being planned and made was that the actors played them be right, and apparently the directors did a good job. Crowley, for example, thinks Brendan Fraser is terrific. “He’s capable of playing such a range of emotions, going from a serious businessman and entrepreneur, to being a dad, to being a husband.” Aileen Crowley had a similarly positive reaction to the actress who portrays her, Keri Russell.

Megan was intensely interested in the process of selecting actresses to play her in the film, and Crowley and his daughter found Droeger very convincing. “Megan has an incredibly dry wit and sarcastic sense of humor,” says Crowley. “Meredith captures that perfectly well. She also convincingly conveys Megan’s strength and determination.” Interestingly, after director Tom Vaughan spent time getting to know the Crowleys at home and experiencing firsthand Megan’s indomitable spirit, he went on to strengthen the role of Megan in “Extraordinary Measures.”

One thing that the Crowley parents have wanted for their children is as normal a life as possible. So when Megan was ready for kindergarten, they took themselves over to Johnson Park School to have a talk with the principal, Robert Ginsberg. He was willing to have Megan but suggested that John Crowley come and talk to the parents so that they could prepare their children for the experience of his gifted, special-needs daughter. Megan, who really comes alive in Crowley’s book as a strong-willed, smart, humorous, sometimes sarcastic, and empathetic child, connected easily with the other kindergarteners.

Although the kindergarteners were accepting, Crowley learned a year later when Megan was about to start first grade that some overprotective kindergarten parents had requested their children not be placed with Megan, however nice the family was. The reality was, of course, much different from these parents’ unfounded worries; rather than being overwhelmed by Megan’s disability, the kindergarteners were totally enriched by the experience of being her friend. And the next year the E-mails that Ginsberg received were pleas that children remain in Megan’s class, not leave it.

When Crowley and his wife got the news of Megan’s illness, along with the prediction that she would only live for a couple of years, they were devastated. “Initially you go through shock, grief, denial, and anger, through all of these in the course of a day,” he says. “But that quickly changed to determination. We didn’t know if we could change the prognosis, but we wanted to do everything we could.”

So they set out on a journey to raise money to fund research and to talk to doctors and researchers worldwide who were investigating Pompe disease, which afflicts only 5,000 to 10,000 people worldwide at any one time, is one of 7,000 rare disorders that affect more than 30 million people. “They’re called orphans for a reason,” says Crowley, “although we’ve come a long way with the Orphan Drug Act in ‘83 that spurred research on these diseases.”

Crowley found researchers working on Pompe in Europe and at Duke University, the University of Florida, and the University of Oklahoma. “They were largely not in touch with each other,” says Crowley. “There is a lot of competition in the academic world.”

At that point Crowley had a plum marketing job at Bristol-Myers Squibb and the family had moved to New Jersey. But by March, 2000, he decided he needed to turn his professional focus entirely to searching out and creating treatments for Pompe disease. “I was frustrated with the pace of research, no company was working on it, and I had tried and couldn’t get the industry focused on it,” he says.

What pushed him into starting his company, Novazyme Pharmaceuticals, were health emergencies with both Patrick and Megan. “They had gotten very sick, very quickly, and we came very close to losing them,” says Crowley. “That’s when I resigned myself to doing everything I could to continue to drive toward treatment and eventually a cure.”

From the beginning the culture at Novazyme up-ended the traditional idea that science is a slow process. “One thing we didn’t have was time,” says Crowley. “We were driving science toward a first-generation treatment in enzyme replacement therapy. We were very focused and what we lacked in experience and financial resources, we made up for in hard work, determination, and a lot of passion.”

In September, 2001, Crowley oversaw the sale of Novazyme Phamaceuticals to Genzyme Therapeutics and became senior vice president at Genzyme.

By January, 2003, Megan and Patrick were able to participate in trials at St. Peter’s Hospital in New Brunswick of a new enzyme replacement therapy treatment that Genzyme developed. The disease in his children had progressed and had devastated muscles in their hearts and respiratory systems as well as skeletal muscles. What was most immediately threatening to their lives was the fact that their hearts were at least twice the normal size. The treatment was, and is, an enzyme infusion that the two children receive every other week at St. Peters Hospital in New Brunswick.

But before his children could begin to participate in the trial Crowley had to first leave Genzyme because of ethical questions about Crowley’s double role as parent and executive of the company developing the drug. He became founding president and chief executive officer of Orexigen Therapeutics. In 2004 he came to Amicus Therapeutics as a director and was advanced to chief executive officer in January, 2005.

Early on the treatment of Crowley’s children brought down the size of their hearts to normal. For a relatively short period, it also strengthened muscles, particularly in Megan’s case, but these improvements, which nurtured Crowley’s dreams of a normal future for her, turned out to be short lived. The treatment did, however, at least stabilize both children.

The good news is that although early predictions of their life spans was two, then five, then 10 years, Megan just turned 13, and Patrick will be 12 in March. “With the efforts of a lot of people, we have extended their lives hopefully a great, great deal and also their quality of life,” says Crowley. “They require ventilators to breathe, and they are in wheelchairs, but they are happy, have lot of friends, are at John Witherspoon (Middle School), are smart, and have the most amazing spirit of life, strength, and joy.”

Crowley’s father was a police officer in Bergen County who died tragically due to leaking carbon monoxide in a faulty police car when Crowley was seven years old. This experience, he says, was an early rerun of what it is like to wake up in the morning, find out something has happened, and say to yourself, “Life isn’t supposed to be this way.” But he, his brother, and his mother did okay. “We got through it, lived, and thrived, only because of the strength and love of my mom and everyone else around us, and the police community.” Also, watching his mother, who worked hard to take care of her family as both a secretary and a waitress, Crowley learned the art of doing what he had to do, also at an early age. Crowley’s Irish and Italian Catholic roots have also been an important influence in his life, which he details in “Chasing Miracles.”

He graduated from Bergen Catholic High School in 1985 and attended the United States Naval Academy in Annapolis, Maryland, from 1986 to 1987. He then transferred to Georgetown University where he earned a bachelor of science in foreign service. He and Aileen, who had been high school sweethearts, married in 1990.

Crowley graduated from the University of Notre Dame Law School in 1992 and worked for the health care practice group of Bingham Summers Welsh & Spilman in Indianapolis. He then continued his education and earned an MBA from Harvard Business School in 1997. His first job in business was with a management consulting firm in San Francisco.

Today Amicus has a Pompe treatment in the most preliminary stages. The company is also working on treatments for two other rare diseases. They are Amigal, for Fabry disease; and Plicera, for Gaucher disease. Amigal is soon to start Phase III testing, and Plicera is in the middle of Phase II, which are the early stages of clinical trials.

In research partly funded by a grant from the Michael J. Fox Foundation for Parkinson’s Research, Amicus scientists have unraveled the biochemical links between Gaucher’s disease and Parkinson’s, following up on a National Institutes of Health public paper reporting that carriers of Gaucher’s disease are at 5 to 10 times the normal risk of developing Parkinson’s disease.

In February, 2009, Amicus had to suspend a Phase II study of its oral drug for Pompe disease after two patients suffered serious side effects, and in October the company’s third-quarter report indicated that a two-year partnership with Wayne-based Shire Pharmaceuticals had ended and will require the company to let go workers. Currently the company has about 100 emplyees, down from a high of 120.

Crowley’s book, “Chasing Miracles,” whose publication is timed to coincide with the movie’s release, expresses his admiration and love for his children and what the family has learned living every day with their special needs and special talents. “In some respects the disease changed us before we could change it,” he says. “Eventually we learned to hope and adapt, to realize that all we have with each other is time.”

Author Event, Barnes & Noble, MarketFair, West Windsor. Saturday, January 23, 3 p.m. John F. Crowley, author of “Chasing Miracles: The Crowley Family Journey of Strength, Hope, and Joy.” A Princeton resident, Crowley left a successful corporate career to co-found a start-up biotech company dedicated to finding a treatment for the rare genetic disorder affecting his two youngest children.

“Extraordinary Measures,” the film based on his story, stars Harrison Ford, Brendan Fraser, and Keri Russell, and opens nationwide on Friday, January 22. John and Aileen Crowley will introduce the Saturday, January 23, 1 p.m., screening at MarketFair. Buy tickets in advance at the movie theater or at www.fandango.com. 609-716-1570 or www.bn.com.

Facebook Comments