Father of gene therapy participant says researchers acted 'irresponsibly'

Jesse Gelsinger, 18, was receiving an experimental gene therapy for an incurable inherited liver disease prior to his death in September

February 2, 2000
Web posted at: 3:42 p.m. EST (2042 GMT)

(CNN) -- The father of a young man who died during a gene therapy trial at the University of Pennsylvania told a Senate hearing on Wednesday that researchers acted "irresponsibly" and they downplayed possible risks to his son.

Paul Gelsinger testified during the Senate Health, Education and Labor Subcommittee hearing on whether the Food and Drug Administration did the right thing in shutting down gene therapy trials at the University of Pennsylvania after 18-year-old Jesse Gelsinger of Tucson, Arizona, died in September.

"When lives are at stake, when my son's life was at stake, money and fame should take a backseat," Paul Gelsinger told the senators. "The concern should not be on getting to the finish line first, but making sure no unnecessary risks are taken, no lives filled with potential and promise are lost forever. No more fathers lose their sons."

The young Gelsinger was receiving an experimental gene therapy for an incurable inherited liver disease, OTC deficiency.

Dr. Amy Patterson of the National Institutes of Health also testified at the hearing and said that researchers nationwide had underreported adverse events from studies similar to the University of Pennsylvania's.

After Gelsinger's death the NIH asked researchers conducting similar experiments to ensure all adverse events had been reported. The number then jumped from 39 to 691. Patterson said there were clear rules violations and the NIH will be investigating.

On January 21, the FDA, citing numerous research regulation violations, shut down gene therapy and other clinical trials at the University of Pennsylvania.

The closure of these trials was a great disappointment to many of the participants with the same disease Gelsinger had.

In gene therapy, researchers actually replace or fix a gene that's causing the disease. People with OTC deficiency have a faulty gene that makes ammonia build up in the body, often leading to brain damage and death.

"The people who live with this disease on a daily basis have nothing to hope for," says Tish Simon, a trial participant. "This was the hope, and they took it away."

Simon carries the faulty gene and passed it to her son. She doesn't have severe health problems, but her son died when he was 14.

She sees Gelsinger's death as unfortunate, but believes the trials should continue.

"We've always had trials and patients have died," she says. "We have open heart surgery, we have organ transplants, people died, and ... the research didn't stop."

The FDA cited several reasons for halting the experiments, including that researchers failed to notify the FDA in a timely fashion about the deaths of two monkeys in the study, and about some side effects in humans.

Despite FDA arguments, study participant Janie Sheedy agrees with Simon that the trials should continue. Her four sons all had the defective gene and died before they were three days old.

"If another gene therapy study came up I would without hesitation do it again," Sheedy said.

She does not blame the doctors for Gelsinger's death.

"I don't think they could in any way have predicted what happened to Jesse Gelsinger, and I think it was a complete shock and devastation to them," Sheedy said.