Originally published April 11 2005
Genetic therapies slow to produce breakthrough treatments
by Mike Adams, the Health Ranger, NaturalNews Editor
Genetic therapy, the use of genetic material as a targeted drug for a variety of conditions, is an idea whose boosters promised quick cures for deadly diseases. However, after more than 900 clinical trials of gene therapies, there have been few real successes, and supporters of the therapies acknowledge that gene therapy successes have been slower to materialize than they anticipated. For example, in one trial, children who were born without a working immune system had been injected with working copies of a gene that helps the immune system grow. Though most of the children developed functional immune systems, as many as thirty percent later died or developed leukemia-like conditions.
Fifteen years ago, scientists heralded gene therapy as a medical revolution that would quickly bring cures for crippling and deadly diseases.
"Everybody realizes that using a gene as a drug is a good idea," says Ronald Crystal, chairman of genetic medicine at Cornell University's Weill Medical College.
Experts say the story of the treatment of 10 sick children at a Paris hospital, which made news in March, illustrates the technology's promise as well as its challenges.
The boys were born with a rare and lethal genetic disorder that left them without working immune systems.
Although bone marrow transplants can cure the disease, many children are unable to find matched donors, says Donald Kohn, a professor of pediatrics at Keck School of Medicine at the University of Southern California.
Instead of treating the symptoms, French doctors hoped to correct the source of the illness by providing the boys with working copies of a gene that helps the immune system develop.
"They're out with their playmates rolling around in the dirt," says Theodore Friedmann, a professor of pediatrics and biomedical ethics at the University of California San Diego School of Medicine.
Many new medical treatments --- from heart transplants to cancer drugs --- took decades to perfect, doctors say.
Scientific companies are shifting efforts away from rare disorders and toward more common maladies such as cancer, blindness and Parkinson's disease because of the larger potential markets, says Mark Kay, a researcher at Stanford University and president-elect of the gene therapy society.
Only 10% of gene therapy trials in the USA now involve single-gene disorders, says Sonia Skarlatos, gene therapy coordinator for the National Heart, Lung, and Blood Institute.
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