Gene therapy patient dies

WASHINGTON - A patient in a gene therapy experiment died on Tuesday in what appears to have been a reaction to a novel treatment for arthritis, federal health officials said late Thursday.

The precise cause of death remains unexplained. But the event immediately revived memories of a similar tragedy in 1999, when teenager Jesse Gelsinger succumbed in a gene therapy test in which researchers at the University of Pennsylvania were eventually shown to have violated safety rules.

That disaster was a major setback for the field, which for more than 15 years has sought to treat diseases by giving people new genes. The only documented successes - in a handful of children - were undermined when the treatment was found to have caused cancer in some.

Food and Drug Administration officials said they were notified by Seattle-based Targeted Genetics last Friday that a patient had experienced a "serious adverse event," regulatory code for a life-threatening reaction. The agency immediately shut down the study pending more information. Four days later the patient died.

The company said 127 people have been enrolled in the trial at 20 U.S. hospitals. Treatment involves injections of viruses engineered to produce special proteins that can quench the inflammatory reaction that underlies arthritis.

The company had recently gained FDA permission to increase the viral dose in the study.

The so-called adeno-associated virus, or AAV, used in the experiment is different than that used in the Gelsinger study in Philadelphia. In recent years, AAV has gained popularity as a safer alternative to other gene-delivery viruses, some of which can cause cancer or dangerous immune system reactions.

To date, about 600 people have been enrolled in 29 gene therapy tests with AAV, according to FDA records. The number of serious reactions in those studies was not immediately available.

Targeted Genetics has previously reported that the treatment seemed safe and "may lead to improvements" in joint swelling and tenderness." The company announced the death on its Website under the headline: "Targeted Genetics Provides Update on Inflammatory Arthritis Phase I/II Trial."