The father of a teenager who died after being injected with experimental genes said he and his son were not informed of the treatment's risks and potential side effects.
Paul L. Gelsinger of Tucson, Ariz., said that before he let his son participate in a gene therapy experiment, he was not told that a monkey had died in a similar experiment and that another patient had had serious side effects.
Gelsinger's son, Jesse, 18, died at the University of Pennsylvania in September after being injected with genes designed to correct an inherited liver disease. Jesse was recruited for the experiment because he was born with ornithine transcarbamylase deficiency syndrome, a liver disorder.
"It looked safe. It was presented as being safe," Gelsinger testified Wednesday at a Senate subcommittee hearing. "Since it would benefit everybody, I encouraged my son to do this.
"I was misled," he added. "That's what hurt the most. This was not as it was presented."
Jesse Gelsinger went into a coma and died within hours, becoming the first patient whose death was directly linked to experimental gene therapy.
The testimony came in a hearing by the Senate Health, Education, Labor and Pensions Committee's panel on public health, which is investigating patient safety in gene therapy clinical trials and whether more vigorous government supervision is needed. The hearing follows reports that some researchers have failed to tell the National Institutes of Health about participants' illnesses.
Sen. Bill Frist, R-Tenn., a physician and chairman of the subcommittee, said the death of Gelsinger "has sobered us all" about gene therapy.