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ASGCT: Gene Therapy Shows Promise for Patients with Pompe Disease

New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

Milwaukee (Vocus) January 30, 2010 -- New research in gene therapy is bringing doctors and patients closer to a comprehensive treatment for a rare disease portrayed in a new Hollywood film, according to the American Society of Gene & Cell Therapy.

“Extraordinary Measures” is based on the true story of John Crowley, a father struggling to find treatment for his children with Pompe disease, a rare, and often fatal, inherited disease that severely weakens the heart and lungs. Currently, the only approved treatment for Pompe is the enzyme-replacement medication Myozyme, which was ultimately developed by Genzyme Corporation.

In a study released this week in Molecular Therapy, ASGCT’s official journal, researchers at the University of Florida reported that gene therapy targeted at the diaphragms of mice with Pompe disease improved the ability of the mice to breathe independently.

This summer, six infants with the disease will receive the treatment. Although the treatment is not a cure, when used in conjunction with other therapies, like the drugs portrayed in the film, it is another step toward improving the lives of patients with Pompe disease.

“While the availability of Myozyme has been a life-saving therapy and a breakthrough for those living with Pompe disease, it must serve as a catalyst for continued innovation,” said Robert Mattaliano, PhD, group vice president of protein research and development at Genzyme. “Gene and cell-based therapies are at the forefront of such endeavors and bring the potential go to beyond replacing a missing enzyme by further improving the muscle repair process in this disease and others.”

Barry Byrne, MD, PhD, director of the Powell Gene Therapy Center at the University of Florida, is one of multiple doctors on which Harrison Ford’s character, Robert Stonehill, is based. Byrne also served as a technical advisor on the film.

"The movie focuses on John (Crowley) and the scientists he worked with to develop a treatment," Byrne said. "The filmmakers strived to create a story the audience will understand. I think it will resonate with people to see how much a parent will go through for his kids."

“Extraordinary Measures” follows John Crowley through his battle to secure funding for research, which eventually leads him partner with William Canfield, PhD, and Canfield’s pharmaceutical company, Novazyme, to develop a treatment for Pompe disease. Harrison Ford’s character is also based on Canfield. Prior to Novazyme finding a treatment, Novazyme was purchased by Genzyme Corporation. Following the purchase, Genzyme remained committed to developing a treatment for Pompe disease; on April 28, 2006, Myozyme was approved by the U.S Food and Drug Administration.

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website,

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