Cause of gene therapy death may be known

More than a year after a Penn gene therapy trial claimed the life of 18-year-old Jesse Gelsinger, researchers have discovered what they believe to be the culprit. James M. Wilson, the head of Penn's Institute for Human Gene Therapy, presented his findings at a closed-door medical conference earlier this month, The Philadelphia Inquirer reported Friday. The cause, Wilson revealed at the meeting in Utah two weeks ago, is the protein coating of a cold virus used in the experiment, which was halted after Gelsinger's death in 1999. A team led by Wilson traced the cause of death to the coating of a virus used to deliver genes to treat Gelsinger's liver disorder, the Inquirer reported. A protein on the surface of the virus initiated an immune reaction that ultimately led to massive organ failure. Gelsinger, who suffered from a mild inherited liver disorder, died four days later. Some scientists at the conference applauded Wilson's efforts to help explain the tragedy. "He did a nice piece of detective work and made an extremely important observation," Jeffery Chamberlain, a gene-therapy researcher at the University of Washington, told the Inquirer. But Alan Milstein, attorney for the Gelsinger family, told the Inquirer that Wilson's findings only prove that the experiment should never have been performed. "What Dr. Wilson seems to have confirmed is this clinical trial should never have been approved and started," he said. In November, Penn settled a lawsuit filed by the Gelsinger family for an undisclosed sum. The Food and Drug Administration recently initiated proceedings to disqualify Wilson from ever again performing human drug trials in the United States. Wilson remains the head of the IHGT. Last May, Penn announced that the IHGT would no longer conduct human gene therapy trials. Wilson is accused of not properly informing the FDA about the deaths of two monkeys that died after being given a drug similar to the one given to Gelsinger. FDA officials contend that had they known about the deaths, the trial would have been halted prior to the start of any human testing. But Penn researcher Stephen Eck pointed out that while the gene treatment given to the monkeys was somewhat similar to Gelsinger's, it was nevertheless different. "That was much ado about nothing," Eck said. "That virus made a totally different protein. I don't think it is relevant." Today -- while controversial in light of Gelsinger's death -- the use of deactivated viruses to deliver therapeutic genes is still being pursued as a possible treatment for many disorders. "People overlook the fact that people got a virus similar to Gelsinger, but did not die," Eck said. Penn is currently in the process of restarting two halted gene therapy trials under sponsorship outside the IHGT. "Out of the nine trials sponsored by the IHGT, all are inactive right now," Arthur Asbury, dean of Penn's Medical School, said. "Two are under different sponsorship and are using different [viruses]."

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