FDA Halts Three Gene Therapy Trials After Patient Develops Cancer

In a "major setback" for the field of gene therapy, the FDA yesterday announced it suspended three gene therapy trials early last month after learning that one child who received the treatment has developed a leukemia-like disease, the New York Times reports. While it is unclear if the patient -- one of 14 in gene therapy trials in the United States and France for severe combined immunodeficiency disorder, known informally as "bubble boy disease" -- developed the blood disorder from the gene treatment, FDA officials said they thought it was likely. The case is the first time that a gene therapy patient has developed cancer, although scientists have long suspected that retroviruses, which the suspended trial used, could trigger the disease. Researchers said it is too early to know if the other children in the SCID trials are at risk. The field of gene therapy is "still reeling" from the 1999 death of Arizona teenager Jesse Gelsinger while participating in a gene therapy trial at the University of Pennsylvania, the Times reports (Gay Stolberg, New York Times, 10/4). The FDA next week will hold a meeting of "outside experts" to discuss the gene therapy trials, Dr. Philip Noguchi, who oversees gene therapy research at the FDA, said (New York Times, 10/4).