Fixing gene therapy trials

When my editor forwarded me a press release yesterday promoting a linkurl:series of articles;http://www.liebertonline.com/toc/hum/0/0 in January's issue of Human Gene Therapy on informed consent, he mentioned that the authors of those pieces were the key players in the death of an 18-year-old in a 1999 gene therapy trial that had called informed consent into question. The issue's editorial was written by James Wilson, the journal's editor-in-chief, and one of the articles was written by Univers

Written byAlla Katsnelson

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When my editor forwarded me a press release yesterday promoting a linkurl:series of articles;http://www.liebertonline.com/toc/hum/0/0 in January's issue of Human Gene Therapy on informed consent, he mentioned that the authors of those pieces were the key players in the death of an 18-year-old in a 1999 gene therapy trial that had called informed consent into question. The issue's editorial was written by James Wilson, the journal's editor-in-chief, and one of the articles was written by University of Pennsylvania bioethicist Arthur Caplan. Both had been defendants in the lawsuit brought by the family of Jesse Gelsinger, the teenager who died in 1999 while participating in a gene therapy trial at UPenn for the metabolic liver disease, ornithine transcarbamylase deficiency. The articles addressed the need to fix informed consent procedures for linkurl:gene therapy;http://www.the-scientist.com/article/display/23064/ trials in the wake of recent serious adverse events, including the death of 36-year-old linkurl:Jolee Mohr;http://www.the-scientist.com/news/display/53453/ last summer after receiving an linkurl:experimental therapy;http://www.the-scientist.com/blog/display/53949/ for rheumatoid arthritis. That made us think to call linkurl:Alan Milstein,;http://www.sskrplaw.com/attorneys/milstein/ who represented the Gelsinger family in linkurl:the case,;http://www.sskrplaw.com/links/healthcare2.html which settled out of court in 2000. Milstein also represents the Mohr family. "That's the pot calling the kettle black," Milstein uttered when I told him that that Wilson, the journal's editor-in-chief, had written the issue's editorial. linkurl:Wilson;http://www.the-scientist.com/article/display/20347/ was the lead investigator of the trial in which Gelsinger died, and was accused of several linkurl:ethical breaches;http://www.the-scientist.com/article/display/11900/ in the study. Caplan provided ethical oversight on the study. "The consent form in that trial is in many ways the best example of how these consent forms in gene therapy trials are misrepresenting the nature of the risk," Milstein told me. Apart from the obvious - the form didn't include information on prior adverse events, or results of studies in which monkeys had died from the treatment - it"compared gene therapy to a taxi cab delivering a healthy gene," he said, a description that "sounds fairly innocuous, as opposed to describing it as it really is" - a risky procedure. Milstein contends that informed consent procedures have changed little in the years between Gelsinger's death and Mohr's. "Just the name gene therapy is a misrepresentation," he said. "It's gene transfer, that may or may not provide any therapy." He noted that consent forms walk a difficult line - they have to be simple enough for subjects to understand, but still accurately represent the risks. One way to address that problem, he said, is by using patient advocates who can translate the sophisticated language into terms subjects can understand. linkurl:Wilson;http://www.liebertonline.com/doi/pdfplus/10.1089/hum.2008.0104 , linkurl:Caplan;http://www.liebertonline.com/doi/pdfplus/10.1089/hum.2007.1010 and other authors in the issue bring up many of these same points. Although the aftermath of the Gelsinger study "set into motion major changes in the oversight of clinical research in the United States," Wilson writes, "it appears we have not gone far enough, based on the concerns that have surfaced" in the study in which Mohr died. "I encourage the professional gene therapy societies and various related foundations to work together in the new year to undertake a critical review" to make sure subjects know what they're getting into.