GENE THERAPY: Successful Treatment Aids Battered Field
Gene therapy has "unequivocally succeeded," according to French researchers who used the treatment in several infants suffering from a rare, immune disorder, the New York Times reports. The recent success may be a boost for the beleaguered field of gene therapy research, which has been plagued by a decade of unfulfilled expectations and most recently became the target of an extensive investigation after last year's death of a University of Pennsylvania clinical trial participant (Kolata, 4/28). Published in today's issue of the journal Science, Dr. Alain Fischer and colleagues at the Necker children's hospital in Paris report that they used the treatment in two infants suffering from severe combined immunodeficiency disease (SCID). Also known as the "bubble boy disease," SCID prevents infants from developing the immune cells necessary to fight off infections. Researchers infused the infants' stem cells with a normal version of the immune system gene and after 11 months, both showed immune system cell levels equivalent to healthy children of the same age, and neither showed any adverse reactions to the treatment.
Hope and Caution
Despite the apparent success, researchers cautioned that it was still too soon to determine if the infants had been cured ( AP/Richmond Times-Dispatch, 4/28). Fischer said, "It may be that after some time there will be a decline in these cells and that sooner or later we will have to treat them again." Still, researchers hope that the finding will move the field in a positive direction. Dr. R. Michael Blaese, a member of the first medical team to attempt gene therapy in humans 10 years ago, said, "This would probably be the first example in any disease where gene therapy could be a fully successful treatment" (New York Times, 4/28).