A 4-year-old girl stricken by a rare immune disease underwent the first official attempt at human gene therapy, ushering in what may be a "revolution in medicine."
The historic procedure went smoothly at a research hospital at the National Institutes of Health Friday on a girl suffering from an immune system defect similar to that which afflicted David, the famed Texas "bubble boy," scientists said."The patient is doing beautifully. Everything went perfectly all day," Dr. W. French Anderson of the National Heart, Lung and Blood Institute, head of the research team, said in a statement.
The girl, whose name and hometown were withheld, talked, played and "put stickers on all of our lab coats" as genetically altered cells were infused into her body for 28 minutes, Anderson said.
Although Friday's experiment focuses on an uncommon disease, Anderson said it marks a "crucial, first small step" towards potentially using genetic techniques to fight everything from cancer to AIDS to heart disease.
"If this works, gene therapy might very well become a major new revolution in medicine. This should provide cures for diseases that are now incurable," Anderson told a Thursday news conference. Scientists were unavailable to reporters Friday, saying they wanted to remain at the child's bedside and monitor her condition.
Researchers said it could take up to a year to see if the therapy reconstitutes the child's immune defenses. "We certainly don't expect to tell you within this week that the treatment is a rousing success," said Dr. R. Michael Blaese of the National Cancer Institute.
Jeremy Rifkin of the non-profit Foundation on Economic Trends in Washington, petitioned the NIH Friday, voicing opposition to the experiment and calling for creation of a committee to review the social and ethical implications of all human gene experiments.
"The NIH has brought us into the Brave New World of human genetic engineering. We are calling for a complete halt on future human gene therapy experiments until and advisory board on eugenics is established, " said Rifkin.