FDA to defend closure of gene therapy trials to Senate subcommittee

February 2, 2000
Web posted at: 10:20 a.m. EST (1520 GMT)

(CNN) -- A Senate subcommittee will be hearing testimony Wednesday into whether the Food and Drug Administration did the right thing in shutting down gene therapy trials at the University of Pennsylvania after a patient's death.

Eighteen-year-old Jesse Gelsinger of Tucson, Arizona, died in September after receiving an experimental gene therapy at the University of Pennsylvania for an inherited liver disease.

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 is a great disappointment to many of the participants with the same incurable disease Gelsinger had, known as OTC deficiency.

In gene therapy, researchers actually replace or fix the 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.

At Wednesday's hearing on Capitol Hill, Gelsinger's father and officials from the FDA and the National Institutes of Health will be testifying on whether Gelsinger's death was a tragic accident or a sign that something was truly wrong with these gene therapy experiments.