Debate over safety of gene therapy trials focuses on issue of informed consent

Can a patient who agrees to participate in a safety study of a gene therapy protocol give truly informed consent and understand the risks involved when the consent forms are highly technical and the physician or institution seeking their consent has a stake in the study and its outcome?

The continuing debate over informed consent and the acknowledgement of risk and responsibility in gene therapy trials are the focus of a series of probing and provocative commentaries published in the January 2008 issue (Volume 19, Number 1) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The commentaries are available free online.

In the Editorial, James M. Wilson, MD, PhD, Editor-in-Chief and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, at the University of Pennsylvania School of Medicine, in Philadelphia, calls on the gene therapy, regulatory, and research communities to re-explore the issue of informed consent as it relates to the safety of viral vector-based gene transfer therapies and the appropriateness of having physicians and others with an interest in the trials and their outcomes recruit patients and obtain the necessary informed consent.

“I encourage the professional gene therapy societies and various related foundations to work together in the new year to undertake a critical review of the conduct of our clinical trials...it behooves us to get ahead of this issue and put in place more effective ways to assure that research subjects who courageously give of themselves can do so with a full and unbiased understanding of the risks and benefits of their participation,” writes Dr. Wilson.

This issue of the Journal also includes five stimulating commentaries that discuss the key controversies related to patient recruitment and trial management. They explore the question of whether patients are able to comprehend the lengthy and often highly technical study descriptions and consent forms to the extent needed to make an informed decision about enrolling in a trial. The authors also present suggestions and innovative strategies for improving the recruitment and informed consent process.

Dr. Arthur Caplan, Professor of Bioethics, Chair of the Department of Medical Ethics, and Director of the Center for Bioethics at the University of Pennsylvania, authored a commentary entitled, “If It Is Broken Shouldn't it Be Fixed? Informed Consent and Initial Clinical Trials of Gene Therapy.” Another, entitled, “Case of Leukaemia Associated with X-Linked Severe Combined Immunodeficiency Gene Therapy Trial in London,” was prepared by the Board of the European Society of Gene and Cell Therapy, Executive Committee of the Clinigene Network of Excellence, and Executive of the Consort Integrated Project. Dr. Jeffrey Kahn, Director and Professor, Center for Bioethics, at the University of Minnesota Medical School, presents his perspectives in, “Informed Consent in Human Gene Transfer Clinical Trials.” Also contributing a commentary is Dr. Tomas Jose Silber, Chair of the Institutional Review Board and Director of the Office of Ethics at Children's National Medical Center, in Washington, D.C. It is entitled, “Human Gene Therapy, Consent, and the Realities of Clinical Research: Is It Time for a Research Subject Advocate?” Ms. Suzanne Pattee, Vice President of Regulatory and Patient Affairs for the Cystic Fibrosis Foundation authored, “Protections for Participants in Gene Therapy Trials: A Patient's Perspective.”