Movie tells story of development of treatment
Print
Email
Save
Reprints
Twitter
Share
Del.icio.us
Digg
Facebook
Reddit
There was no medical treatment available for Pompe disease in 1998, when John and Aileen Crowley, then living in San Francisco, learned their two youngest children had the fatal disease.
The story of how John threw himself into starting a biotechnology company that ultimately led to the development of a treatment for Pompe has been made into a movie, “Extraordinary Measures,” opening Friday.
Brendan Fraser plays the part of Crowley, and Harrison Ford plays the scientist, Dr. Robert Stonehill, who, together, set out to find an answer to Pompe.
The movie is based on the book “The Cure: How a Father Raised $100 Million — and Bucked the Medical Establishment — In a Quest to Save His Children,” written by Wall Street Journal reporter Geeta Anand.
Today, children diagnosed with Pompe disease, a progressive disease that impairs muscles from functioning normally, are being treated with an enzyme replacement medicine called Myozyme.
The medicine is manufactured by the company Genzyme, which purchased the startup biotechnology company that Crowley founded.
Currently, two children in the Baton Rouge area are receiving the enzyme replacement therapy at Our Lady of the Lake Children’s Hospital, according to Dr. Michael Brumund, a pediatric cardiologist at the Children’s Hospital.
Dr. Duane Superneau, a local geneticist with Genetics Services of Louisiana, a subsidiary of the Pathology Group of Louisiana, said that the medicine is difficult and expensive to make and is reserved for persons who most need it.
Those are usually infants, said Superneau, who helped confirm the diagnosis of Pompe in one of the children being treated here.
Without treatment, the disease is lethal for infants, he said. A late onset form of the disease affects people in childhood, adolescence or adulthood, with symptoms that are usually milder, he said.
The treatment of the disease is so new that long-term outcomes are still being learned, he said
Still, it can be a life-saving treatment, Superneau said.
“We know what’s going to happen if we don’t treat” the illness in infants, he said.
Click "Report Abuse" to notify our moderators that a comment may contain objectionable content.
Your comment appears to contain objectionable content and must be reviewed by a site moderator. If your comment is deemed objectionable, it will not appear on the site.
 | ||||
| Most Popular | Most Emailed | Hot Topics | ||
