Patient in Gene Therapy Study Dies
Friday, July 27, 2007
A patient in a gene therapy experiment died on Tuesday in what may have been a reaction to a novel treatment for arthritis, federal health officials said late yesterday.
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 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 potentially life-threatening condition. The agency immediately shut down the study pending more information. Four days later, the patient died.
The company said 127 people were enrolled in the trial at 20 U.S. research centers. Treatment involves injections of viruses engineered to produce special proteins that can suppress the inflammatory reaction that underlies arthritis.
The company had gained FDA permission last year to increase the viral dose in the study. The company's chief scientific officer, Barrie Carter, said he could not release any details about the patient.
The adeno-associated virus, or AAV, used in the experiment is different from that used in the Gelsinger study at the University of Pennsylvania. 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 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 previously reported that the treatment seemed safe and "may lead to improvements" in joint swelling and tenderness. The company announced the death late yesterday on its Web site under the headline: "Targeted Genetics Provides Update on Inflammatory Arthritis Phase I/II Trial."