(Circulation. 2000;101:e9052.)
© 2000 American Heart Association, Inc.
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Cardiovascular News
Shaking the World of Gene Therapy
In a move that shook the nascent world of gene therapy, the University of Pennsylvania announced on May 24, 2000 that it was ending clinical trials of gene therapy in its Institute for Human Gene Therapy and that it will redirect its efforts at the molecular, cellular, and animal model level.
The Institute, which is led by gene therapy pioneer James Wilson, MD, had initiated many pioneering clinical trials in the past and was considered a world leader in the field. However, in the wake of the September 17, 1999 death of 18-year-old Jesse Gelsinger, a participant in the phase I clinical trial to treat an inherited disorder called ornithine transcarbamylase deficiency, the actions and procedures of the institute came under increasing scrutiny, both from within the University and from federal agencies. Although no clinical trials will be undertaken at the gene therapy institute, the University said that trials might be undertaken elsewhere in the greater institution.
Dr Wilson released a statement that was quoted in the Washington Post (May 25, 2000:1). In it, he indicated that the Institute would "refocus our efforts in the preclinical area—with the goal of developing a foundation of science necessary to ensure the ultimate success of this field. Although numerous scientific obstacles exist, gene therapy has tremendous potential to omeday prevent or cure life-threatening diseases."
The University’s president, Judith Rodin, took action after receiving
the results of an investigation by an independent review panel. The
panel, which was led by William H. Danforth, MD,