By Bio-IT World Staff
January 22, 2010 | “Extraordinary Measures,” starring Harrison Ford and Brendan Fraser, is a film based on Wall Street Journal reporter Geeta Anand's book "The Cure: How a Father Raised $100 Million—and Bucked the Medical Establishment—in a Quest to Save His Children." It is inspired by the story of John Crowley (Brendan Fraser), a father of two children affected by Pompe disease. In the late 1990s Crowley joined with a researcher named William Canfield (Robert Stonehill) to try and develop a treatment for Pompe.
Pompe disease is a rare disorder that affects only about 10,000 patients worldwide. In patients with Pompe, an enzyme known as acid alpha-glucosidase (GAA) is either missing or in short supply. GAA is responsible for the breakdown of glycogen, a form of sugar stored in muscle cells throughout the body. In patients with Pompe, glycogen builds up in cells in the body, weakening and damaging muscles. As it progresses, Pompe can cause a range of serious health problems and can affect breathing and mobility.
Myozyme, the first and only FDA-approved product to treat Pompe disease, was developed by Genzyme Corporation. In the movie, the "Special Medicine," is Myozyme and it was developed based on research conducted by Genzyme in collaboration with researchers at Duke University and the Erasmus Medical Center in The Netherlands. The drug candidate originally developed by Crowley and Canfield failed to achieve data statistically significant to continue development.
The clinical research program to develop Myozyme was a monumental and historic event in medical history. The development effort included 2 global clinical research studies conducted at 7 study sites over 3 years. These were some of the first clinical trials ever conducted involving Pompe patients, and John Crowley's children, Megan and Patrick, were some of the first patients treated with Myozyme.
Genzyme has posted a link on their website, which includes the “real story” behind Myozyme with videos of some of the scientists and patients: http://www.genzyme.com/pompemovie/. The Wall Street Journal’s Review Revue here.