The US Food and Drug Administration (FDA) shut down eight gene therapy trials at the University of Pennsylvania last week as a result of an ongoing investigation. All the trials have been halted 'indefinitely' after FDA findings of 'serious deficiencies' in the supervision and monitoring of one genetic therapy trial that ended in the death of 18-year old Jesse Gelsinger last September.
The FDA have identified 18 areas in which the university failed to meet protocol and to provide proper supervision and monitoring of the clinical gene therapy trial to treat ornithine transcarbamylase (OTC) deficiency, a rare genetic liver disease. Among the failures listed are the omission of informing patients of the deaths of two monkeys who were receiving the experimental treatment, improperly completed consent forms, and patients being placed in groups other than the ones they were initially approved for by the FDA. Two patients in the trial also had side-effects strong enough that the trial should have been immediately halted and reviewed.
The director of Pennsylvania University's Institute for Human Gene Therapy, James Wilson, must respond to each of the points raised by the FDA. The university president, Judith Rodin, has appointed a committee to review the supervision of clinical gene trials.
Following the FDA report, there have been calls for more transparency in gene therapy trials, especially where the safety of patients is involved. The UK Gene Therapy Advisory Committee has shown plans to summarise on its website any serious side-effects that occur in UK gene therapy trials, and has initiated the long-term confidential monitoring of patients. Norman Nevin, chairman of the government's Gene Therapy Advisory Committee has stated that there are no plans to review gene therapy trials in the UK in the light of the FDA report.