Aug 12 2007
The death of a woman taking part in a clinical trial for an arthritis therapy has set alarm bells ringing in the United States.
The 36 year old Illinois woman died on July 24th from internal bleeding and organ failure after receiving the second injection of an experimental gene therapy treatment to treat her rheumatoid arthritis.
Jolee Mohr, who has a 5-year-old daughter, took part in the study because she believed it would help the rheumatoid arthritis which affected her whole body.
Her death has triggered a U.S. government investigation and resulted in the suspension of the gene therapy study being conducted by the Seattle company Targeted Genetics.
The study involved a hundred patients and other such studies around the country are also now under review.
The therapy uses AAV to deliver a gene that in turn blocks tumor necrosis factor, a substance that fuels the joint inflammation behind crippling forms of arthritis.
Mohr received the experimental injections at an Illinois clinic and died 22 days after receiving her second injection.
According to a hospital spokesman by the time she was admitted, Mohr had liver and kidney failure, was on a ventilator and appeared to be reacting to a severe infection.
The company says it is too early to speculate how Jolee Mohr died but adds that patient safety is of paramount importance and all of their trials are designed and conducted with that in mind.
Targeted Genetics says they are confident in the safety of the product and are eager to complete the clinical trials, which they say will determine the efficacy and safety profile of this or any other drug candidate.
The FDA says it is not aware of any side effects of studies using AAV.
Some expert believe that until such early gene therapies are better understood, they should only be tested on people who are suffering from severe, life-threatening conditions.
Most agree that in regard to gene therapy research, an increase in federal funding would allow the science to develop in a more regulated environment with researchers released from commercial pressures.