Inside Duke Medicine
Media Spotlight: News 14 features Duke’s Pompe research
posted January 25th, 2010
A new film called “Extraordinary Measures,” starring Harrison Ford and Brendan Fraser, centers around a medical treatment developed at Duke University.
The movie features a father's desperate search for a drug to treat his child's Pompe disease, which is caused by the lack of an enzyme that breaks down glycogen for energy. That breakdown can lead to anything from weakened muscles to an enlarged heart.
In the movie, the dad starts a biotech company to develop a therapy. But in real life, the only life-saving treatment was developed at Duke University Medical Center.
“Similar to a patient with diabetes who's missing insulin, in patients with Pompe disease, they are missing this enzyme called acid alpha-glucosidase. And so what's being done is providing this missing enzyme to a patient with Pompe disease to allow the body to be able to do the work it should have been able to do if it were making the enzyme on its own,” Dr. Priya Kishnani, a Duke pediatric geneticist, said.
Watch the video.