The Food and Drug Administration (FDA) temporarily shut down a series of human gene therapy experiments at the University of Pennsylvania last month following the death of Jesse Gelsinger, an 18 year old man from Arizona who was being treated for a liver disorder.
The order to place the university's entire gene therapy programme on indefinite “clinical hold” came two days after inspectors issued a report noting 18 specific violations of government protocols.
The administration said that the hold would not be lifted until the university's Institute for Gene Therapy showed that it could follow procedures that protect volunteers from harm.
Among the evidence gathered by the administration was the institute's failure to consider Mr Gelsinger's eligibility for the treatment, in the light of reports of serious effects in other patients.
This violation was made public in a report issued during a December meeting of the recombinant DNA advisory committee, the National Institutes of Health's panel established to oversee gene therapy research.
But the final report included numerous other violations. The FDA said that the informed consent process “was not well documented” and thus it was difficult to determine who had conducted or attended the discussions on informed consent.
Meanwhile, Paul Gelsinger, Jesse's father, who supported the experiments even after last month's advisory committee report, has said that he has now changed his mind and hired a lawyer. So far no suit has been filed.
Jesse Gelsinger was undergoing treatment for ornithine transcarbamylase deficiency, a disorder that prevents the liver from effectively processing ammonia, a toxin produced in the breakdown of proteins. He died of multiple organ failure triggered by a severe immune reaction to an infusion of corrective genes.
Adding to the controversy was the fact that the inherited disorder is relatively mild and that the patient had previously kept the problem in check through diet modification and prescription drugs.
Mr Gelsinger's death is believed to be the first directly resulting from gene therapy. The episode—widely reported in the media—has sent shock waves through the gene therapy research establishment.
Figure.
The artist Alexa Wright collaborated with the dermatologist Professor Irene Leigh to create this image of a woman with vitiligo. The photograph is part of the Invisible Body exhibition at the Atrium Gallery, Whiteleys Shopping Centre, London, until 13 February.