[Natalie Allen, Cnn Anchor:] Experimental gene therapy programs are under scrutiny after the Food and Drug Administration shut down all such studies at a major university. One patient died while taking part in a study of a liver disorder. CNN medical correspondent Elizabeth Cohen spoke with other participants in that study who hope this and other genetic experiments will continue.
[Elizabeth Cohen, Cnn Medical Correspondent:] Eighteen-year-old Jesse Gelsinger died last fall after receiving an experimental treatment called gene therapy. His doctors had hoped the treatment would cure his disorder. Four months later, the Food and Drug Administration shut down the experiment and all gene therapy trials at the University of Pennsylvania, and that makes Tish Simon mad. She has the same disease, called OTC deficiency, for which there is no disease.
[Tish Simon, Gene Therapy Trial Participant:] The people who live with this disease on a daily basis have nothing to hope for. This was the hope, and they're taking it away.
[Cohen:] In gene therapy, researchers actually replace 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 FDA cited several reason for halting the experiments, including the researchers failed the notify the FDA in a timely fashion about the deaths of two monkeys in the study and about some side effects in humans. Janie Sheedy, another participant in the experiment, also says the trials should continue. Her four sons all had the defective gene, all died before they were three days old.
[Janie Sheedy, Gene Therapy Trial Participant:] I'd do it again in a heartbeat. It was they took such good care of me there.
[Cohen:] She doesn't blame the doctors.
[Sheedy:] I don't think that they could have in any way predicted what happened to Jesse Gelsinger, and I think it was a complete shock and a pretty big devastation to them.
[Cohen:] At the Senate hearing, today, Jesse Gelsinger's father says he feels the researchers acted irresponsibly, adding that before his son entered the study researchers down-played possible risks.
[Paul Gelsinger, Jesse Gelsinger's Father:] When lives are at stake, when my son's life was at stake, money and fame should take a back seat. 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.
[Cohen:] Also at the hearing, an official from the National Institutes of Health said that before Gelsinger's death, researchers doing studies similar to the one that the University of Pennsylvania reported that there had been 39 adverse events, in layman's terms, 39 events where something went wrong with patients. But then after Gelsinger's death, researchers said there had in fact been 691 adverse events. The NIH spokeswoman said that's a clear violation of the rules and her agency will be doing an investigation Natalie.
[Allen:] Elizabeth Cohen in Washington. Thanks, Elizabeth.