Word: cystic
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Dates: during 1990-1999
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...target cells and sometimes insert themselves in the wrong place in the cellular DNA. Then too, once in place, the new genes sometimes fail to express themselves. "There are still a few walls to get over," Anderson concedes. He points out that in the initial trial, viruses used for cystic fibrosis, for example, produced an inflammatory response: "So the trials were halted, and another generation of viral vectors was developed. Now we're going to restart the clinical trials with these new-generation vectors," which he thinks will...
...months in 1987. In fact, the FDA's responsiveness to aids and certain life-threatening diseases has surprised some early skeptics. Five of the six antiviral drugs used to treat AIDS were first approved in the U.S. The anticancer drug Taxol, the first multiple sclerosis drug and the first cystic fibrosis drug all got the FDA go-ahead long before they were accepted for use in Europe...
Quick to put the best face on the discouraging results, investigators pointed out that they have other research paths to pursue. "You don't usually hit a home run the first time," says Dr. Michael Knowles, the University of North Carolina researcher who led the cystic fibrosis trial. "You usually make incremental steps forward, and that's what this study...
...comes word of major technical snags in two areas of gene therapy that had been regarded as among the farthest along. Reporting in separate articles in the New England Journal of Medicine last week, researchers concluded that the most commonly used genetic treatments for cystic fibrosis and muscular dystrophy had run into a dead end. In both cases, scientists inserting normal genes into patients with defective ones were not able to elicit corrective changes in their patients' bodies...
...basic research and clinical trials mutually exclusive goals. "Humans are not big mice," says Dr. Ronald Crystal, who is working on gene therapy for cystic fibrosis at New York Hospital-Cornell Medical Center. "Unless we do clinical trials, we're never going to learn. You have to test it in the lab on animals, try it in humans and then go back to the lab. It's a cyclic process...