Report from the ASGT Ad Hoc Committee on Retroviral-mediated Gene Transfer to Hematopoietic Stem Cells
April, 2003

In February, 2003, ASGT established an ad hoc committee of scientists to collect and analyze the relevant data that exists from among many individual studies performed world-wide, including experimental research studies in animals and the relevant clinical trials. Understanding the mechanisms that underlie the development of leukemia may lead to improved methods of gene therapy for this inherited immune deficiency disease that can minimize the risks but preserve the benefits that have been seen in this trial. As with all clinical research, patient safety is of paramount importance and all possible efforts must be made to minimize risks.

The results of the deliberations of the ad hoc committee have been posted publicly on the society's web-site (www.asgt.org) and are available to regulatory and advisory agencies to assist in their assessments of the risks and benefits of this new form of therapy. ASGT continues to work closely with the FDA and Office of Biotechnology Activities/National Institutes of Health to examine the scientific, medical and ethical issues involved with gene therapy studies.

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