CBS Films this weekend releases “Extraordinary Measures,” a movie that tells the true story “of one man’s quest to find a cure for his two children, who suffer from a rare genetic disorder that would leave them unable to walk, eat or even breathe on their own.”
The story focuses of the life of John Crowley, a son of a
According to one article, the deadly Pompe disease, named after a Dutch pathologist who first described it, afflicts an estimated 1 in every 40,000 births, according to the National Institute of Neurological Disorders and Stroke. Victims rarely lived past 2.
When
He then launched a biotechnology company in 1999, Novazyme Pharmaceuticals Inc. of
His inspiration to save his children’s lives led him through the difficult and complex workings “of the pharmaceutical industry to lead the development of a drug called Myozyme that eventually shrank Megan and Patrick's hearts to normal size, and saved their lives.” The FDA approved the therapy drug, Myozyme, in April 2006.
Crowley was so dedicated to his cause that in March 2003, his daughter “Megan became one of the first to participate in clinical trials of an enzyme replacement therapy.”
Dr. Dave Dickens, pediatric hematologist and oncologist with Helen DeVos Children's Hospital, noted that although “enzyme replacement therapy does not cure Pompe disease, it keeps the effects of the missing enzyme at bay.” Dr. Dickens further emphasized that “Pompe disease is no longer a terminal illness but a chronic disorder that can be managed similarly to how diabetes is managed through insulin.”
Dr. Robert Mattaliano, a vice president at Cambridge-based Genzyme who spent years trying to find a treatment for the disease, applauded the movie for its ability to “increase awareness and give more hope for people with rare diseases.”
Crowley commented on his experience by noting the impact his family's story might have on health care reform. He emphasized the "importance of innovation in medicine,” and how far we have come “with these great new medical technologies.” When I was a kid, 30 years ago, a classmate was diagnosed with leukemia. And he died. And now most kids diagnosed with leukemia don't die. What I'm really concerned about is that we don't come to the point where we say we've done enough."
Mr. Crowley’s family story should serve as an inspiration that with the right kind of dedication, support and collaboration, the odds can be beaten, and adversity can be overcome.
His unwavering passion for the private sector to start a biotechnology company to save the lives of his children is symbolic of the efforts that almost every employee at pharmaceutical companies strive to make, whether finding a cure for cancer or diabetes.
This film helps remind us that saving lives and helping patients depends on collaboration, drive and resources to make it happen.
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