Patient in AAV Gene Therapy Trial for Arthritis Dies

A patient who received an experimental gene therapy product in a clinical trial for the treatment of active inflammatory arthritis died on July 24, 2007. Targeted Genetics Corporation of Seattle notified the U.S. Food and Drug Administration on July 20th that the patient had experienced a serious adverse reaction. Upon receiving that notification, the FDA shut down the study while it began to gather more information. Four days later, the patient had died.

According to the FDA, the gene therapy product uses a particle, also referred to as a vector, to deliver genes to target cells. In this particular study, the vector (a recombinant adeno-associated virus or AAV derived vector) delivers the gene for tumor necrosis factor-receptor, intending to inhibit a key mediator of inflammation. The gene therapy product was administered directly into the affected joint. This was the second injection received by the patient who became ill and later died. The first dose, given several months earlier, had been uneventful.

There are 127 people enrolled in the clinical trial which was being conducted at 20 research centers in the United States. According to a statement issued by Targeted Genetics, "Since the trial began in October 2005, 127 subjects have received an initial dose of active drug or placebo, 74 subjects out of the total 127 have received a second dose of active drug, and of those 74 subjects, 55 have received two doses of active drug." The FDA is investigating whether the death of the patient was directly related to the experimental treatment. The timing of the serious adverse event and subsequent death make it suspect, but the information to this point is considered preliminary.

Update 8/6/2007 from washingtonpost.com - Death Points to Risks in Research

Related Resources:

• Judging Clinical Trials
• Clinical Trials: Why Volunteer?
• Clinical Trials for Arthritis
• Placebos: Is Every Placebo the Same?

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