John Crowley & his daughter, Megan.
ABOUT EXTRAORDINARY MEASURES
The movie is based on the true story of John and Eileen Crowley and their two children, Megan and Patrick, diagnosed with Pompe disease before they were two years old.
Pompe disease is an incurable, systemic disease where sugars build up in the entire body and can't break down causing organs and muscles to become overwhelmed and eventually fail.
When doctors told John Crowley that his children would not survive past their tenth birthday, he didn't accept it and instead, did something else. He found a type of cure. An enzyme treatment that is still used today.
The Crowleys started working with non-profit organizations to raise money for Pompe disease research. By the year 2000, with the help of a scientist friend, John started a small biotech company in the hopes of finding a cure for Pompe disease.
Dr. Robert Stonehill's character is based upon scientist and researcher William Canfield, who founded Novazyme. According to Roger Ebert's review, the character is based on Yuan-Tsong Chen, a scientist and researcher from Duke University who collaborated with Genzyme in producing Myozyme, the drug which received FDA approval. William Canfield is a glycobiologist, chief scientific officer and founder of an Oklahoma City-based biotechnology company, Novazyme, which was acquired by Genzyme in August 2001 and developed, among other things, an enzyme that can stabilize (but not cure) Pompe disease, based on Canfield's ongoing research since 1998. Canfield subsequently left Genzyme and established, with his partner in the Novazyme operation, John Crowley, another research laboratory (Cytovance Biologics), which he still heads. Yuan-Tsong Chen is a Taiwanese physician scientist, notable for his work on human genetic disorders. He is the director emeritus (2001–2010) and distinguished research fellow (2001–present) of the Institute of Biomedical Sciences, Academia Sinica, Taiwan, and also tenured professor of pediatrics of Duke University (1993–present) Chen was a 2019 awardee of Taiwan's Presidential Science Award.
Megan Crowley, the daughter of John and Aileen Crowley, the inspiration of Extraordinary Measures and ultimately, the reason the treatment for Pompe disease exists, has survived. She has since earned a Master's degree in Social Work from the University of North Carolina after achieving a Bachelor's from Notre Dame. She is currently working as a research assistant. Her astonishing story and achievements continue to be an inspiration to ADOM and to so many others.
Glycogen storage disease type II, also called Pompe disease, is an autosomal recessive metabolic disorder which damages muscle and nerve cells throughout the body. It is caused by an accumulation of glycogen in the lysosome due to deficiency of the lysosomal acid alpha-glucosidase enzyme. It is the only glycogen storage disease with a defect in lysosomal metabolism, and the first glycogen storage disease to be identified, in 1932 by the Dutch pathologist J. C. Pompe. Alglucosidase alfa, sold under the brand name Myozyme among others, is an enzyme replacement therapy (ERT) orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD). Chemically speaking, the drug is an analog of the enzyme that is deficient in patients affected by Pompe disease, alpha-glucosidase. It is the first drug available to treat this disease. It was approved for medical use in the United States in April 2006, as Myozyme and in May 2010, as Lumizyme.
THE CURE TODAY
Myozyme and Lumizyme are used in the treatment of Pompe disease to this day.