A gene therapy study halted after an arthritis patient died will resume with one main change: participants won't get injections if they have signs of infections, the treatment's developer said Monday.
Jolee Mohr had a slight fever -- a sign of infection -- when she received the last of two injections at a Springfield, Ill., clinic on July 2. She died three weeks later after doctors discovered a massive fungal infection and internal bleeding.
Targeted Genetics has chosen a "conservative" approach in resuming the experiment, chief executive officer H. Stewart Parker said in a telephone interview Monday. Of 127 total participants, 35 still need a second shot and will be told of Mohr's death when the study resumes, the company said.
Investigation participants included the company, the U.S. Food and Drug Administration, and the University of Chicago Medical Center, where Mohr was transferred after she fell ill and where she died. Final results are to be discussed at a federal gene therapy committee meeting on Dec. 3 at the National Institutes of Health outside Washington.
Panelists at a previous NIH committee meeting in September had raised concerns about Mohr receiving the experimental treatment when she already had signs of illness.
Questions also had been raised about whether genetically engineered viruses injected into patients' joints as part of the experiment might spread throughout the body, causing illness.
But tests on Mohr found only trace amounts in tissues outside her joints, not enough to have contributed to her death, said Dr. Kyle Hogarth, a University of Chicago physician who treated Mohr after she fell ill.
Seattle-based Targeted Genetics believes arthritis medicine Mohr already was taking, which has been linked to an increased risk for fungal infections, likely played a role.
Hogarth said "it's great" that the study is resuming because the experimental treatment could be useful. But he also said troubling questions remain, including why participants will be allowed to continue taking arthritis medicine that could complicate efforts to prove that the gene treatment is safe.
Hogarth also noted that Mohr's own doctor recruited her for the study and also performed the experimental treatment, which he said raises ethical concerns. That's because patients tend to trust that their doctors are working in their behalf, and so Mohr could have mistakenly assumed he was recommending an already proven treatment.
Alan Milstein, an attorney for Mohr's husband, Robb, said the family still believes her death was avoidable.
The study involved injecting patients with trillions of genetically engineered viruses carrying a gene intended to help the body produce an inflammation-fighting protein.
The FDA halted the study after Mohr's death but last week told the company it could continue.
"We believe this is a vindication for the product and for the gene therapy field in general," Parker said.
While the study's first phase was to examine safety, there were early indications that the experimental treatment helped relieve pain for those with inflammatory arthritis, Parker said. Inflammatory arthritis includes rheumatoid arthritis and affects more than 2 million Americans.
Illustration: Microsoft clipart.
Seattle Times (11/09/07)
Targeted Genetics Corporation (11/10/07)
Washington Post (11/26/07)
San Francisco Chronicle (11/26/07)
United Press International (11/26/07)
Associated Press (11/26/07)
Denver Post (11/26/07)
Fox News (11/26/07)
CBS News (11/26/07)
Westfall Weekly News/CA (11/27/07)