Gene Therapy Research :: Science Genetics Health Essays

Gene Therapy Research The National Institute of Health and the Food and Drug Administration are the government bodies responsible for the regulation of gene therapy experimentation. The NIH oversees gene therapy clinical trials through the NIH Guidelines for Research Involving Recombinant DNA Molecules and the Recombinant DNA Advisory Committee (RAC). The Food and Drug Administration (FDA) also oversees the trials through such regulatory measures as scientific review, testing, and inspection.10 Following the death of Jesse Gelsinger, the Advisory Committee to the Working Group on NIH Oversight of Clinical Gene Therapy Research was established to examine the purpose and role of the NIH in gene therapy regulation. It made several recommendations, many of which were adopted by the NIH as amendments to its research guidelines.12 In March of 2000, two initiatives by the FDA and the NIH were announced by the Department of Health and Human Services to increase protection for individuals involved in gene therapy experiments. The first initiative, the Gene Therapy Clinical Trial Monitoring Plan, requires that the sponsors of gene therapy studies regularly submit their monitoring plans to the FDA for review. The FDA will also inspect clinical trials to insure that the plans are being followed, and any problems are managed. In addition, scientists experienced in gene therapy will be gathered to discuss monitoring practices at conferences of the inspectors. The second initiative, the Gene Transfer Safety Symposia, includes a series of symposia that take place four times a year to discuss gene therapy data, inviting some of the most experienced authorities in their respective fields. The NIH and FDA are also providing support for other organizations interested in holding safety conferences on the topic of g ene therapy.12 Proposed Review Process3 The guidelines of the NIH for gene therapy research have also changed since 2000. In one amendment to the guidelines governing gene transfer research, the NIH altered the way research subjects are enrolled for gene therapy experiments. Under the amendment, clinical trial protocols must be submitted to the NIH Office of Biotechnological Activities (OBA) for RAC review, and the RAC must finish its review before the local Institutional Biosafety Committee (IBC) gives its approval to the trial. Subjects for research can only be accepted once the IBC and the International Review Board, IRB, approve the trial.