Human Gene Therapy

By Knorr, Debra | National Forum, Summer 1999 | Go to article overview

Human Gene Therapy


Knorr, Debra, National Forum


Ensuring Progress In the Next Millennium

The year 2000 will mark the tenth anniversary of the first human gene therapy experiment. This first year of the new millennium is also the twenty-fifth anniversary of the National Academy of Sciences conference known as "Asilomar." In the early 1970s, the importance of recombinant DNA technologies to the development of a host of new therapeutics to treat many of humanity's most devastating diseases was widely recognized. At the same time, public anxiety increased about the possible generation of hazardous biological agents and unknown public health risks that might arise as a result of this new technology.

Recognizing the depth and breadth of public concern, the scientific community self-imposed a moratorium on recombinant DNA research and convened a conference at the Asilomar Conference Center in California to publicly discuss the scientific, ethical, legal and social issues related to recombinant DNA research. An unprecedented gathering of scientific experts convened at Asilomar with a single goal - to develop an oversight plan that would permit the research to move forward, while at the same time would reassure the public. Emerging from Asilomar was a set of guidelines establishing practices and procedures for the safe use of recombinant DNA.

A UNIQUE OVERSIGHT PROCESS

The system of oversight that was stimulated by Asilomar and adopted by the National Institutes of Health (NIH) has far exceeded its original expectations. Since 1975, the NIH has provided effective and efficient oversight of recombinant DNA research as an extension of its purview over individuals and institutions that receive NIH funding and support for research. Founded on the principle that public participation in policy development, access to information, and accountability are paramount to research progress, the non-regulatory oversight provided by the NIH offers several combined advantages that cannot be achieved by any single regulatory mechanism.

NIH oversight over recombinant DNA research is based on compliance with the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines). The NIH Guidelines specify practices and procedures for the safe conduct of recombinant DNA research and delineate the roles and responsibilities of individual research investigators, NIHfunded institutions, Institutional Biosafety Committees, the Recombinant DNA Advisory Committee (RAC), the Office of Recombinant DNA Activities (ORDA), and the NIH Director for ensuring compliance with the principles set forth in the NIH Guidelines. The NIH Guidelines also provide guidance for optimal preclinical and clinical research design and standards for informed consent. Failure to comply with the requirements set forth in the NIH Guidelines can result in the limitation, suspension, or withdrawal of NIH support. NIH support includes direct funding of recombinant DNA research, other fiscal support, contractual agreements, and infrastructure support such as equipment, supplies, and facilities.

This unique mechanism of research oversight has been endorsed by the U.S. scientific community, the biotechnology industry, the general public, and numerous international regulatory bodies as a highly efficient and effective means of oversight that facilitates research progress, while at the same time maintaining public confidence that research is being conducted in a safe and responsible manner.

PUBLIC ACCESS TO CLINICAL TRIAL INFORMATION

Human gene transfer research holds extraordinary therapeutic promise. But because of the prominence of the genetic revolution and the rapid discovery of new genes, much of the public believes that gene therapy is already a viable therapeutic option for treating many serious and lifethreatening diseases. Yet in fact, while hundreds of clinical investigations involving gene transfer have been proposed in the United States, there is still no statistically significant data demonstrating long-term therapeutic benefit. …

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