"Meanwhile, journalists and federal health officials discovered several troubling lapses in the conduct of the study. For example, the researchers had earlier told the FDA they would tighten up the trial’s eligibility criteria, but they never followed through. When two patients suffered serious side effects, the scientists did not immediately inform the agency or put the study on hold as required. It turned out Jesse’s pretrial test results showed he had poor liver function, indicating he arguably shouldn’t have received the OTC gene injection.
But perhaps most damning were failures in the informed-consent process. Researchers hadn’t told Jesse about the earlier patients’ side effects or about two lab monkeys killed by high doses of adenoviruses. If he had been properly briefed about these previous issues, he might have dropped out of the study and still be alive today. Wilson was also accused of a conflict of interest: he had a stake in the company that owned the gene-transfer technology and stood to benefit if the trial succeeded.
...
The investigations drew attention to wider problems in oversight of gene-therapy experiments and human research generally. For example, the FDA and NIH revealed that 691 volunteers in gene-therapy experiments had either died or fallen ill in the seven years before Jesse’s death; only 39 of these incidents had been reported promptly as required. The agencies tightened monitoring of trials, increased inspections, and created a new system for reporting serious side effects, among other steps. Penn responded to the crisis by strengthening the institutional review boards that oversee its trials, putting in new protections for patients, and prohibiting researchers from having financial stakes in their trials."
Thanks for this.
"Meanwhile, journalists and federal health officials discovered several troubling lapses in the conduct of the study. For example, the researchers had earlier told the FDA they would tighten up the trial’s eligibility criteria, but they never followed through. When two patients suffered serious side effects, the scientists did not immediately inform the agency or put the study on hold as required. It turned out Jesse’s pretrial test results showed he had poor liver function, indicating he arguably shouldn’t have received the OTC gene injection.
But perhaps most damning were failures in the informed-consent process. Researchers hadn’t told Jesse about the earlier patients’ side effects or about two lab monkeys killed by high doses of adenoviruses. If he had been properly briefed about these previous issues, he might have dropped out of the study and still be alive today. Wilson was also accused of a conflict of interest: he had a stake in the company that owned the gene-transfer technology and stood to benefit if the trial succeeded.
...
The investigations drew attention to wider problems in oversight of gene-therapy experiments and human research generally. For example, the FDA and NIH revealed that 691 volunteers in gene-therapy experiments had either died or fallen ill in the seven years before Jesse’s death; only 39 of these incidents had been reported promptly as required. The agencies tightened monitoring of trials, increased inspections, and created a new system for reporting serious side effects, among other steps. Penn responded to the crisis by strengthening the institutional review boards that oversee its trials, putting in new protections for patients, and prohibiting researchers from having financial stakes in their trials."
Truly nothing new under the sun.