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Gene therapy no quick fix
September 13, 1996
Web posted at: 11:55 p.m. EDTFrom CNN Correspondent Andrew Holtz
PALO ALTO, California (CNN) -- Revolutions take time.
That's what researchers and patients are learning about gene therapy.
Take cystic fibrosis, for instance. The gene that causes the potentially lethal breathing disorder was identified in 1989. But a genetic cure isn't yet in hand.
Hayley Wester knows all too well about the time-intensive practice of translating discoveries into results. Four times a day, she thumps her lungs to clear clogging mucus caused by cystic fibrosis, an ailment that stems from an error in a single gene.
"I remember the gene was discovered when I was in high school," Wester said. "I had the newspaper article and I ran into the lunch room and I went to a really small school so everybody knew what I was doing and I said, 'Hey, they found the gene.'"
The next steps seemed obvious: Fix the gene and cure the patients.
But that's not as easy as it sounds. Since cystic fibrosis was discovered seven years ago, researchers have learned a lot about how difficult it is to turn that discovery into something practical that patients can use.
Wester is part of the latest effort to devise an effective cystic fibrosis gene therapy. A virus carrying the corrected gene was squirted into her sinuses. Previous attempts had disappointing results.
"The virus caused some problems within the cell itself," said Dr. Phyllis Gardner of Stanford University Medical Center. "It caused the disease, and the patient's own body reacted against the virus by producing antibodies."
A more recent experiment used a virus that usually causes no symptoms.
In an effort to avoid the side effects caused in previous experiments, researchers removed almost all of the virus's original genes.
"That makes it a stealthier virus," Dr. John Wagner of the medical center. "It's harder for the immune system of the body to find it and react to it."
Researchers monitor the treatment with a flexible scope. Eventually, they expect gene therapy could be applied with simple aerosol inhalers.
But hopes for gene therapy are tempered by caution. "We let the community believe that a cure was around the corner, and it's not," Gardner said. "We have to work out these strategies that make this work. I believe that ultimately we will."
Practical gene therapy is still years away, researchers say.
And that's not soon enough for Wester. Her lungs are so damaged that she's preparing for a lung transplant. Still, she believes the experiments will help those who follow her.
"I've seen so many advances just in my lifetime," she said. "I think that the prospects are very good for the future."
Related stories:
- Researchers find gene injection slows cancer - August 28, 1996
- Gene therapy proves successful for two children - October 20, 1996
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