Gene therapy trial set to resume

The US Food and Drug Administration is allowing a controversial gene therapy trial to linkurl:resume,;

By | November 26, 2007

The US Food and Drug Administration is allowing a controversial gene therapy trial to linkurl:resume,; after the trial was linkurl:halted; when a 36-year-old participant died in July. The therapy, developed by Seattle based company Targeted Genetics, seeks to treat inflammatory arthritis, and is delivered via an adeno-associated viral (AAV) vector through an injection in the arthritic joint. At a meeting of the National Institutes of Health's Recombinant DNA Advisory Committee (RAC) in September, RAC investigators linkurl:announced; preliminary autopsy results of the participant, Jolee Mohr, and suggested that a massive linkurl:fungal infection,; and not the AAV treatment, caused her death. Investigators at the University of Chicago, where Mohr died, failed to find any of the AAV vector outside of Mohr's knee, where the virus was injected. According to a Targeted Genetics press release announcing the FDA decision, when Mohr was treated with the experimental AAV, she was taking other arthritis drugs that carry a risk for the fungal infection - called histoplasmosis - that killed her. The RAC will make the final results of its investigation public on December 3. The FDA lifted the hold on the clinical trial last week after reviewing data on the study's 127 participants and on Mohr's death. Before resuming the trial, however, Targeted Genetics must revise informed consent permissions for study participants to reflect the events surrounding Mohr's death and the results of the ensuing investigation. But some researchers are raising questions about the study's design, which allows patients to continue taking immune-suppressing drugs while receiving the experimental treatment, according to a linkurl:report; in __The Washington Post__. Kyle Hogarth, an intensive care unit doctor at the University of Chicago who treated Mohr and participated in the investigation of her death, told the __Post__: "I think they have a horrible design. It muddies the picture."

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