The movie Extraordinary Measures tells the story of parents who formed a biotechnology company to develop a drug that could save the lives of children who have the rare life-threatening disease known as Pompe.
In the movie, which is based on the book The Cure, Harrison Ford plays research scientist Robert Stonehill, who was instrumental in finding the cure, while Brendan Fraser plays John Crowley, the man who raised US$100 million to buck the medical establishment.
In reality, John Crowley is the name of the person who started the biomedical company, but the real Dr Stonehill who developed the cure is in fact Chen Yuan-tsong (陳垣崇), director of Academia Sinica’s Institute of Biomedical Sciences.
PHOTO: CNA
At an event held earlier this week by the Taiwan Foundation for Rare Disorders to mark the debut in Taiwan of the movie’s DVD, Chen said he had mixed feelings watching the movie.
He said he began researching a cure for Pompe disease in 1991 after being saddened by the deaths of so many children 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 the second movie about rare diseases and patients after Lorenzo’s Oil.”
Movie critic Roger Ebert said that Harrison Ford, as the film’s executive producer, probably saw Stonehill as a good role for himself and ordered a rewrite of the script because he could not play Chen.
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 research and development was mostly done in the US, but Chen conducted his clinical trials for the cure — later named Myozyme — at National Taiwan University Hospital.
Myozyme, which took Chen and his team 15 years to research and develop, was introduced in Taiwan by US pharmaceutical company Genzyme and has been covered by the National Health Insurance (NHI) program since 2005, the foundation said.
Myozyme was sold in Europe and the US after it was approved by the US Food and Drug Administration and the EU health authority in 2006. It has contributed to saving the lives of more than 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 die before they reach two years old. There is also a juvenile and adult form of the disease, which can appear at almost any age, the foundation said.
Currently, Pompe disease patients in Taiwan receive NT$7.9 million (US$245,577) per patient per year in Myozyme and related medical care 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 more than 70 percent of them do not receive effective drugs or therapies, the foundation said.
STATS: Taiwan’s average life expectancy of 80.77 years was lower than that of Japan, Singapore and South Korea, but higher than in China, Malaysia and Indonesia Taiwan’s average life expectancy last year increased to 80.77 years, but was still not back to its pre-COVID-19 pandemic peak of 81.32 years in 2020, the Ministry of the Interior said yesterday. The average life expectancy last year increased the 0.54 years from 2023, the ministry said in a statement. For men and women, the average life expectancy last year was 77.42 years and 84.30 years respectively, up 0.48 years and 0.56 years from the previous year. Taiwan’s average life expectancy peaked at 81.32 years in 2020, as the nation was relatively unaffected by the pandemic that year. The metric
Taiwan High Speed Rail Corp. (THSRC) plans to ease strained capacity during peak hours by introducing new fare rules restricting passengers traveling without reserved seats in 2026, company Chairman Shih Che (史哲) said Wednesday. THSRC needs to tackle its capacity issue because there have been several occasions where passengers holding tickets with reserved seats did not make it onto their train in stations packed with individuals traveling without a reserved seat, Shih told reporters in a joint interview in Taipei. Non-reserved seats allow travelers maximum flexibility, but it has led to issues relating to quality of service and safety concerns, especially during
A magnitude 5.1 earthquake struck Chiayi County at 4:37pm today, the Central Weather Administration (CWA) said. The hypocenter was 36.3km southeast of Chiayi County Hall at a depth of 10.4km, CWA data showed. There were no immediate reports of damage resulting from the quake. The intensity of the quake, which gauges the actual effect of a seismic event, measured 4 in Chiayi County, Tainan and Kaohsiung on Taiwan's seven-tier intensity scale, the data showed. The quake had an intensity of 3 in Chiayi City and Yunlin County, while it was measured as 2 in Pingtung, Taitung, Hualien, Changhua, Nantou and Penghu counties, the data
The Supreme Court today rejected an appeal filed by former Air Force officer Shih Chun-cheng (史濬程), convicted of Chinese Communist Party (CCP) espionage, finalizing his sentence at two years and two months for contravening the National Security Act (國家安全法). His other ruling, a ten-month sentence for an additional contravention, was meanwhile overturned and sent to the Taichung branch of the High Court for retrial, the Supreme Court said today. Prosecutors have been notified as Shih is considered a flight risk. Shih was recruited by Chinese Communist Party (CCP) intelligence officials after his retirement in 2008 and appointed as a supervisor