In wake of gene therapy death, FDA to exercise stricter oversight

The Associated Press WASHINGTON, D.C. -- The Food and Drug Administration will begin forcing scientists to monitor patient safety better during gene therapy experiments and conduct surprise inspections to make sure researchers follow the rules. Federal regulations already require that researchers establish patient-safety monitoring systems before putting experimental treatments to the test. But the FDA acknowledged yesterday it has not enforced the rules for gene therapists. Indeed, in the Penn experiment that killed 18-year-old Jesse Gelsinger, ''there were deficiencies in the monitoring,'' FDA Gene Therapy Chief Kathryn Zoon said. University researchers did not have a formal patient monitoring system, something the FDA said it discovered only when it inspected the hospital's gene therapy lab after Gelsinger died. Under the FDA's new initiative, all gene therapy researchers must appoint someone not directly involved with their experiments to monitor patient safety. The monitors could comprise contract research organizations or other scientists at a hospital or university. The monitors are not truly independent -- the gene therapists hire them. But the FDA pledged to ensure gene therapy monitors are working and know the law requires them to report patient-safety problems to the government -- and to conduct random, surprise inspections to make sure. Experiments that do not comply can be halted. ''We believe these are important steps? to ensure patient safety,'' Zoon said. The initiative sparked a mixed reaction. ''They were supposed to be doing that already,'' said bioethicist George Annas, a Boston University professor of health law. ''The FDA has let this get out of hand. They don't inspire a lot of confidence.'' He questioned whether FDA inspectors had the time or expertise to oversee how more than 350 gene therapy experiments now under way are conducted. But another critic welcomed the change. ''The patient-safety monitoring is the most important element that's been missing in all of this,'' said Abbey Meyers, president of the National Organization for Rare Disorders and a former National Institutes of Health gene therapy adviser. Today, gene therapists can conduct experiments for years without anyone checking how their patients fared, Meyers said. So FDA inspections are vital, but the agency lacks the money and people, she said. ''The FDA's budget has been raped, absolutely raped, over the last 10 years,'' Meyers said. ''Congress has got to give them the money to do this job right.'' The FDA employs between 150 and 200 clinical trial inspectors, but they never have been asked to judge patient-safety monitoring in early-stage experiments or to focus on gene therapy instead of the many other types of human experiments now under way. Gelsinger's death is the only one so far blamed directly on gene therapy, among experiments on about 5,000 patients in 10 years. This new field has not cured anyone yet. And the NIH has discovered hundreds of adverse reactions patients suffered that researchers delayed reporting to government experts who oversee the experiments' safety.

Please note All comments are eligible for publication in The Daily Pennsylvanian.