Gene Therapy Trials in the Spotlight After Woman's DeathAug 6, 2007 | Parker Waichman LLP, LLP
The Food and Drug Administration (FDA) is investigating the death of an arthritis patient last month who was involved in a gene therapy trial. The death of Jolie Mohr from massive bleeding triggered the suspension of the trail, and has called into question the tactics used by drug companies to recruit subjects to clinical trials.
According to an article in the Washington Post, Mohr had been recruited by her doctor to take part in a gene therapy trial to test a new arthritis treatment. The goal of the gene therapy experiment conducted by Targeted Genetics Corp was to test the safety of an experimental arthritis treatment. That treatment had no chance of helping the 36-year-old Illinois woman. While no one can say as yet if the treatment was to blame for Mohr’s death, the Washington Post article highlights failures of an FDA oversight system, as well as breaches of clinical research standards, that drew Mohr into an experiment that was not what she thought it was.
Mohr’s husband told the Washington Post that his wife believed that the gene therapy trail, which involved the injection of a virus into her arthritic knee, would help her condition. He also claims that she was not made aware that the trail could present serious risks. The 15-page consent document Targeted Genetics gave its trial subjects to sign seems to back up this assertion. A two-sentence paragraph halfway through the voluminous document warned of “unknown side effects” and “in rare circumstances, death”. Later, one single sentence reads “We do not expect you to receive any direct medical benefit from participation in this study.” One expert in medical ethics interviewed by the Washington Post said that the consent form used extremely technical language, and was ambiguous about the true aims of the experiment.
Mohr signed the document at the office of her rheumatologist, Robert Trapp. Trapp was receiving financial compensation for every patient he signed up for the gene therapy trail. Mohr should never have signed the consent form in Trapp’s office. Standard clinical research rules mandate that such forms be taken home by patients so that they have time to read through them completely. Also, because Trapp was being paid by Targeted Genetics, someone else should have asked Mohr to consider signing up for the study.
Mohr received two injections during the gene therapy trial. The first in February had no effect. When Mohr arrived at Trapp’s office on Monday, July 2nd for the second injection, she had a slightly-elevated temperature of 99.6. Despite the fever, Mohr was given the shot. The next day, she was vomiting and had a temperature of 101. By Saturday, Mohr was in the emergency room with a fever of 104.1. Though tests indicated that Mohr could be suffering from an infection and liver damage, she was sent home. Mohr continued to get worse, and on Thursday she was admitted to the University of Chicago Hospital and placed on the liver transplant list. But by July 24th, she had died.
Rules require a company to report serious complications that could even be “possibly” related to experimental treatment to the FDA within seven days of learning of it. But Targeted Genetics made no such report. Rather, doctors at the Chicago hospital contacted the FDA. It was that report that caused the FDA to shut down the clinical trial on June 20th.
Companies like Targeted Genetics are focused on their stock price, and setbacks in clinical trials do not help a company’s bottom line. Targeted Genetics’ stock dropped 40-percent after Mohr’s death was announced. What role the company’s concern for profits played in Mohr’s case is not known, but her death highlights the need for stricter oversight of clinical trials.