On September 17, 2007, a committee set up by the National Institutes of Health (NIH) concluded it was too early to tell whether an experimental gene therapy treatment for rheumatoid arthritis contributed to the death of a 36-year-old woman in July 2007. The committee and the woman’s husband also questioned the process used for recruiting patients, including the woman, for the study.

“The biggest question I have is would my wife still be alive today if she hadn’t participated in this study?” Robb Mohr, from Taylorville, Illinois, said. “I have it in my heart that she’d still be here.”

Jolee Mohr died on July 24, 2007 at the University of Chicago Medical Center, three weeks after being injected with trillions of genetically engineered virus in her right knee. She was receiving a test of an experimental treatment for rheumatoid arthritis.

An autopsy suggested the main cause of Jolee’s death was a fungal infection, histoplasmosis, which destroyed her organs. She suffered from internal bleeding that created a pool of blood in her abdomen so large it displaced her kidneys and other organs.

While the virus did not directly kill her, the committee will determine whether the gene therapy drug suppressed her immune system, allowing the infection to spread quickly.

The therapy was meant to cause knee joint cells to product an anti-inflammatory protein identical to the one in Enbrel, a common arthritis drug that can leave patients more susceptible to infections. Jolee was also taking consistent injections of Humira, a similar drug also linked to infections.

Studies found the genetically engineered virus had spread to Jolee’s liver and spleen, raising the possibility that the immune-suppressing protein was produced outside the joint.

Tests on Jolee’s blood and other tissues will continue, but some panel members said the role of the gene therapy in her death may never be definitively determined.

Some members of the panel questioned whether Jolee had enough time to consider enrolling in the trial, and the fact that she was recruited by her arthritis doctor may have led her to believe she would benefit from the trial, while the purpose of it was just to gather information.

President of the American Society of Gene Therapy, Dr. Arthur W. Nienhuis, told the committee that researchers should pay more attention to informed consent.

“No regulations were violated in this case,” Dr. Nienhuis said. “But perhaps we should hold ourselves to a higher standard.”

Source: Andrew Pollack, “Death in gene therapy treatment is still unexplained,” New York Times, September 18, 2007.