Gene Therapy Trials Under Scrutiny Following Death of Illinois WomanAug 13, 2007
A woman with mild rheumatoid arthritis died of multiple organ failure after receiving an experimental drug as part of a gene therapy trial. Her death has prompted a Food and Drug Administration (FDA) investigation of the trial, and has increased scrutiny of 28 other such studies.
Jolee Mohr was just 36 when she died on July 24. Her husband, Robb Mohr said that she agreed to participate in a gene therapy trial run by Targeted Genetics Corp. because she thought it might ease pain caused by rheumatoid arthritis. But the Targeted Genetics trial was being conducted to test the safety of a new drug, not its effectiveness.
Prior to starting the trial, Mohr had signed a 15-page consent document supplied by Targeted Genetics. One clause several pages into the document stated that the trial was not expected to improve the health of any participants. A warning that the trial could pose serious risks was also buried deep within the document. But Mohr signed the paper in the office of her rheumatologist, who was paid by Targeted Genetics to recruit participants. Standard research protocols require that potential trial subjects take consent documents home to read prior to signing them. It is questionable whether Mohr read the entire consent form in the presence of her doctor.
Robb Mohr’s attorney says that it is doubtful Jolee knew of the trial's potential risks, and she sincerely believed the new drug might help her arthritis. “She wasn’t going to risk her life for science or medicine or the profits of some company,” the attorney told the Associated Press. While the attorney said that it is unclear who is to blame for Mohr’s death, her family is certain her death is connected to the trial. Robb Mohr insists that his wife was healthy before receiving a second injection of Targeted Genetics’ experimental medication.
Mohr received that injection on July 2 in her rheumatologist’s office. At the time, Mohr was running a slight temperature of 99.6. By the next day, she was vomiting and had a temperature of 101. On Saturday, Mohr went to the emergency room with a fever of 104.1. Tests indicated that she could be suffering from an infection and liver damage, but 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.
Targeted Genetics’ trial involved a virus that the company hoped would block a substance that causes joint inflammation in some forms of arthritis. Rules require a company to report serious complications that have even the smallest chance of being related to experimental treatment to the FDA within seven days of learning of them. But it was doctors at the Chicago hospital where Mohr died who first contacted the FDA on July 20.
The FDA shut down the Targeted Genetics trial after the agency learned of Mohr’s death. More than 100 patients who were involved in the same trial as Mohr are being evaluated, and 28 studies using the same virus are being reviewed. And in September, the National Institutes of Health’s advisory committee on gene therapy will meet to discuss the implications of Mohr’s death.