(Circulation. 2000;101:e9001.)
© 2000 American Heart Association, Inc.
Cardiovascular News |
Gene Therapy at a Crossroads
No one who presented a protocol before a meeting of the Recombinant DNA Advisory Committee (RAC) before 1996 ever enjoyed it. It was, indeed, trial by fire as basic scientists, clinicians, bureaucrats, attorneys, ethicists, and patient advocates chewed through the proposed protocols, questioned the validity and extent of the experiments, rewrote the informed consent forms, and debated the population of patients who would be permitted to enter the trial. To add further insult, the investigators involved in gene therapy then had to submit reams of similar paperwork to the US Food and Drug Administration (FDA) for further review. Many in the field complained loud and long about the process, which was eventually streamlined and softened by Harold Varmus, MD, who has just left his post as head of the National Institutes of Health.
However, the power of the RAC—now part of the Office of Biotechnology
Assessment—may be resurgent in the wake of the furor over the death of
18-year-old Jesse Gelsinger, who was involved in a phase trial using a
modified adenovirus to correct ornithine transcarbamylase deficiency,
an inherited disorder that can cause death in affected newborn males
because of their inability to properly process nitrogen in food
proteins due to a genetic defect in the liver. In a statement on the
University of Pennsylvania’s Web page, the researchers involved
stated that "The findings suggest that the experimental drug used in
the trial—a modified cold virus, or vector, incorporating a
potentially corrective gene for Mr Gelsinger’s genetic
disease—initiated an unusual