IHGT seeks permission to restart 2 trials

More than a year after the Food and Drug Administration halted all clinical trials at Penn, the Institute for Human Gene Therapy is looking to get back into the arena of human testing. Last year, a January FDA announcement put a moratorium on eight clinical tests. The University now plans to drop six of the trials, but is seeking permission to resume two of the tests. The FDA banned clinical trials at Penn following the September 1999 death of Jesse Gelsinger, an Arizona teenager who participated in an IHGT study of a rare liver disorder. According to Provost Robert Barchi, many of the halted trials were not worth pursuing because they are now outdated when compared to current gene therapy research. "For many of those clinical trials, the scientific rationale for doing them has passed," Barchi said. Penn likely will seek to continue gene therapy experimentation on certain types of brain tumors and on a deadly lung cancer known as mesothelioma. But the IHGT will not pursue its halted trials concerning cystic fibrosis, muscular dystrophy, breast and skin cancer and a rare liver disease. Barchi noted that if Penn does attempt to continue the previously banned research, it will be under the leadership of an organization other than the IHGT. "Of the eight clinical trials that were put on hold, perhaps two of them may eventually seek approval in a revised form, under a different organization," Barchi explained. George Martin, a senior vice president with the biotechnology company FibroGen, did not disagree with Barchi's labeling of some gene therapy trials as outdated. But he did suggest that politics and medical risk may have prompted Penn to drop the liver disease study in which Gelsinger was involved. As for the decision to drop the other trials, Martin said that the design of the studies may not have been up to par. "For the breast and skin cancer studies, they probably didn't have a good protocol," said Martin, who is also an emeritus scientist with the National Institutes of Health. But Martin added that it was fairly logical to continue with studies related to brain tumors and mesothelioma. "Part of this is just a practical decision of what would be more accessible," he said. Martin explained that diseases such as skin cancer and cystic fibrosis afflict large areas of the body, and consequently are much more difficult to target. The advantage to studying the mesothelioma disease, Martin explained, is that this kind of diseased cell is easier to access with gene therapy than are other types of cells. With brain tumors, "the outlook [for a patient] is so bad," Martin said. "They go in and operate, but don't get it all." "It may be that the gene therapy is effective on what is left behind," he said. In November, the FDA sent IHGT director James M. Wilson a letter, starting the process that could ultimately bar him from conducting clinical trials anywhere in the United States. Last week, the FDA refused to comment on whether the organization had received a response from the University or Wilson. Wilson, through his lawyer, said he would respond to the FDA in an upcoming meeting with the agency, The Philadelphia Inquirer reported Sunday. If Penn resumes IHGT clinical trials, Wilson will not be conducting them, according to Barchi. "Dr. Wilson has been proscribed from conducting clinical trials," Barchi said. Barchi also noted that IHGT trials are just a small part of the genetics research occurring at Penn. "With or without the IHGT gene therapy initiatives, the University of Pennsylvania still fully intends to remain at the cutting edge of biomedical research, and at the cutting edge of gene therapy," Barchi said.