The death of Jesse Gelsinger in a gene therapy trial for OTC deficiency at the University of Pennsylvania has raised important questions about informed consent on the frontiers of clinical research. First and foremost, what are the hopes being raised about the promise for gene therapy and is this compromising enrollment in these trials? Second, of what should informed consent for these breakthrough trials consist (e.g. evidence of toxicity in animal tests or in administration of related adenoviral compounds)? Third, who should be overseeing these cutting edge trials and is it possible to maintain safety while not overburdening science? And finally, how should the confounding interests of the investigator-businessman be managed?
By the fall several groups will have made recommendations for better administration of gene transfer research, including a NIH Working Group on Oversight of Clinical Gene Transfer Research.* The FDA, OPRR and the RAC have initiated investigation and discussion of adverse event reporting. Congressional hearings have been held with some potential for legislative action. NBAC may also take up informed consent in gene therapy research or human subjects protections broadly. A review of what went wrong in the Gelsinger case and what the proposed reforms promise to correct is an important discussion for the forum at this juncture in gene therapy research.
Learning Objectives: Understand the special challenges of consent for gene therapy research and compliance with federal regulation
Keywords: Clinical Trails, Genetics
Presenting author's disclosure statement:
Organization/institution whose products or services will be discussed: None
I have a significant financial interest/arrangement or affiliation with any organization/institution whose products or services are being discussed in this session.
Relationship: I am a consultant to the National Institutes of Health for the purposes of the Gene Therapy Working Group and the Director's Council of Public Representatives. I receive travel expenses and a $200/day stipend when I am serving as consultant