Academia Sinica researcher portrayed in motion picture
TAIPEI, Taiwan -- The movie “Extraordinary Measures” tells the story of parents who formed a biotechnology company to develop a drug that could save the lives of their young children, who have the life-threatening rare Pompe disease. In the movie, based on the book “The Cure,” Harrison Ford plays research scientist Robert Stonehill, who was instrumental in finding the cure, and Brendan Fraser plays John Crowley, the man who raised US$100 million to buck the medical establishment.
John Crowley really is the name of the person who started the biomedical company, but the real Dr. Stonehill who developed the cure is in fact Dr. Yuan-tsong Chen, director of Academia Sinica's Institute of Biomedical Sciences.
Chen was present Monday at a news conference held by the Taiwan Foundation for Rare Disorders to mark the debut in Taiwan of the movie's DVD and indicated he had mixed feelings watching the movie.
Chen said he began researching a cure for Pompe disease in 1991 after being saddened by the passing of many young lives from the affliction.
“Before I knew it, it had been 15 years,” he said.
“It also surprised me that Hollywood would have made a motion picture out of it, making it a second movie about rare diseases and patients, after Lorenzo's Oil,” he said.
Movie critic Roger Ebert suggested that Harrison Ford, as the film's executive producer, perhaps saw Stonehill as a plum role for himself and ordered a rewrite because he could not play Chen very well.
Regardless of how Hollywood decided to recast Chen, his contribution to helping find the cure is well established.
He developed the treatment with colleagues at the Duke University Medical Center. His R&D was mostly done in the United States, but Chen conducted his clinical trials for the cure — later named Myozyme — in Taiwan, at National Taiwan University Hospital.
Myozyme, which took Chen and his team 15 years to research and develop, was introduced in Taiwan by U.S. pharmaceutical company Genzyme and included as a drug covered by Taiwan's national health insurance (NHI) program in 2005, the foundation said.
Myozyme was sold in Europe and the United States after it was approved by the U.S. FDA and the EU health authority in 2006. It has contributed to saving the lives of over 1,000 patients with Pompe disease, or acid maltase deficiency, worldwide each year, including 34 in Taiwan.
Young Pompe disease sufferers have symptoms similar to muscular dystrophy, the foundation said.
Without a cure, most children with Pompe disease would die before age 2. There is also a juvenile and adult form of the disease that can appear at almost any age, according to the foundation.
Currently, the Pompe disease patients in Taiwan are being given Myozyme and related medical care worth NT$7.9 million (US$245,577) per patient per year under the NHI program, greatly reducing their families' financial burden, the foundation said.
The foundation quoted tallies from the Cabinet-level Department of Health as indicating that there are nearly 6,000 families with rare disease patients in Taiwan, but over 70 percent of them do not have effective drugs or therapies that can help them, the foundation said.