By Kevin Davies
February 3, 2010 | SAN FRANCISCO -- Biotech entrepreneur John Crowley, whose astonishing personal quest to develop a treatment for Pompe disease, a rare genetic disorder that affects two of his young children, is captured in the new Harrison Ford movie Extraordinary Measures, spoke movingly about his family’s experiences and hopes in the opening keynote at CHI’s Molecular Medicine Tri-Conference (MMTC) here today.
“We’ve learned more about life and love from our two kids than we’ve ever taught them,” Crowley told the MMTC audience, many of whom were visibly moved. But he shared many inspiring and humorous stories as well.
In 1998, on a Friday 13th no less, Crowley and his wife received the devastating diagnosis that his daughter Megan, just 18 months old, had the rare recessive condition Pompe disease, a failure of glycogen metabolism, causing the compound to accumulate to toxic levels in the body’s muscles. “I’m sorry, there’s nothing that can be done,” the doctors told Crowley. His son Patrick was diagnosed with the same disorder shortly afterwards. The typical life expectancy at that time was about two years of age.
Of all the emotions the Crowleys experienced in the weeks that followed, the overpowering feeling was determination – in particular to find a treatment for the incurable disease, and to live life to the fullest every day. But he had not interest in being cast as a hero. “I did my job as a dad,” he said modestly.
Crowley founded a biotech company called Novazyme, drawing personal inspiration from a lecture he heard at the BIO convention in 2000 from wheelchair-bound Christopher Reeve. He quickly raised money and sold the company in 2001 to Genzyme Corporation, which specialized in developing treatments for orphan diseases, for more than $100 million. Genzyme’s drug Myozyme was approved by the FDA in April 2006 and remains the standard of treatment.
Before then, however, Crowley’s daughter, Megan, became very sick. At one point, she was on a ventilator and her heart stopped three times. When she started receiving the drug, Crowley admits he had high hopes. “I expected trumpets,” he said, “but I realized we weren’t done.” Still, thanks to the drug treatment, which she receives every couple of weeks, she has become a precocious teenager who talks with her parents of her hopes of going to college and getting married. Although confined to a wheelchair, the dangerous enlargement of the heart has been halted.
Hollywood Calling
In 2006, a Wall Street Journal reporter, Geeta Anand, published a book on the Crowleys’ quest called The Cure, which caught the attention of one of the most powerful figures in Hollywood. Crowley relived the day that Harrison Ford called him and expressed interest in making a film about Crowley’s family. His reaction? “Which one of my idiot friends was playing a practical joke on me?” he joked.
Crowley is played in Extraordinary Measures by Brendan Fraser, best known for his swashbuckling roles in adventure romps. Crowley remarked that Fraser had done a great job. “He’s 6’5” and I’m 5’6”,” he said. “I think someone in the casting office must have been dyslexic.”
Crowley hopes that Extraordinary Measures raises public awareness of the plight of children and families with all sorts of orphan diseases besides Pompe disease. His latest business venture is Princeton-based Amicus Therapeutics, which develops chaperone drugs to help misfolded enzymes and proteins reach their appropriate cellular levels and locations. Such an approach could help treat dozens of genetic diseases, including cystic fibrosis.
It’s a small contribution among the biotechnology and pharmaceutical ecosystem to find treatments for the thousands of genetic disorders and millions of Americans suffering from various diseases, with the ultimate goal of preventing other parents from having to have a doctor tell them that nothing can be done for their child.
Crowley spoke passionately about his daughter’s strength of spirit and fierce determination. On one occasion, receiving an infusion of a bone-strengthening drug in a pediatric oncology ward at Columbia University medical center, she remarked to her dad that all the other children had cancer, refusing to feel sorry for herself.
And she has a sense of humor too. After a grueling IPO road show for Amicus a few years ago, Crowley took his staff to New York to ring the closing bell on Wall Street. When he got home, his daughter told him that she’d seen her father on television. “Well, what did you think?” Crowley asked. “Dad, you looked really, really… short,” she replied. “But I loved your tie.”
Make a Wish
Crowley paid tribute to the story of Abbey Meyers, whose son was suffering from Tourette syndrome in the early 1980s. After a newspaper story, the plight of orphan diseases was featured on episodes of the medical drama Quincy. Just a few months later, President Reagan signed the Orphan Drug Act into law.
The Crowleys have just published their own account of their family’s story, called Chasing Miracles, with all proceeds going to the Make-A-Wish Foundation. But despite all the glamour of movie premieres in Los Angeles and New York, Crowley’s daughter still has one fervent wish that her father has not been able to grant just yet – an audience with the Jonas brothers.