Gene Therapy Setback; February 2000; Scientific American Magazine; by Beardsley; 2 Page(s)
Eighteen-year-old Jesse Gelsinger died at the University of Pennsylvania last September 17, four days after receiving a relatively high dose of an experimental gene therapy, a novel and unproved technique that aims to correct genetic diseases and other conditions. Gelsinger's death was apparently the result of an overwhelming immune reaction to the engineered adenovirus that researchers had infused into his liver. He died of acute respiratory distress syndrome and multiple-organ failure.
The trial, led by James M. Wilson, director of Penn's Institute for Human Gene Therapy, had sought to test in patients the safety of a possible treatment for an inherited liver disease, ornithine transcarbamylase deficiency (OTCD). Gelsinger had been healthier than most men with OTCD, which causes ammonia to build up in the blood. His illness was being partly controlled with a lowprotein diet and with a chemical therapy that helps the body eliminate ammonia-co-invented, ironically, by one of his doctors in the fatal experiment.