Conflict at the RAC: researchers protest delay in approving therapy protocols; panel members shoot back with criticisms of the science

Article Abstract:

At the March 30, 1990 meeting of the National Institutes of Health's (NIH) Recombinant DNA Advisory Committee (RAC), which met to discuss gene therapy in humans, a debate occurred on the role of the RAC in scientific research. It is felt by some that the RAC and the human gene therapy subcommittee are hindering important medical research. There are delays in approving treatments using the gene for tumor necrosis factor, which can kill tumor cells in patients dying of cancer. The proposal will not even be considered until October 1990. It was thought that there was some conflict of interest, as some of the members of the RAC are also conducting gene therapy research with cancer patients. Steven A. Rosenberg requested an increase in the number of patients in his study from 10 to 40. These patients with melanoma (a fatal cancer of melanocytes, the pigment-containing cells of the skin) are given lymphocytes to infiltrate the tumor; the lymphocytes are labeled with a gene to allow them to be tracked through the body, to see if they reach the tumor. The data on six patients who were successfully treated with these cells was presented. Although the RAC voted to expand the study, there was some discussion on the data and how data should be reviewed, which caused much tension. The next request up for approval was from R. Michael Blase and W. French Anderson who wanted to treat a rare and fatal disease in children. These children do not have functional immune systems because they are missing the enzyme adenosine deaminase, and treatment with the gene coding for this enzyme was proposed. This request was denied, even though the proposal had been previously approved by the NIH. It is questioned if the delays by the RAC are simply bureaucratic, or if they are based on good scientific judgement. Many members of the RAC are not satisfied with the format for scientific review. Some solutions were reached on new procedures including the written presentation of the data to the committee before the meeting, the existence of primary and secondary reviewers, and written reviews. The new review system will be implemented before the next meeting on June 1, 1990. (Consumer Summary produced by Reliance Medical Information, Inc.)

Therapeutics, Political aspects, Science and technology policy, Genetic engineering, United States. National Institutes of Health, United States. Recombinant DNA Advisory Committee

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Gene therapy begins

Article Abstract:

Gene therapy has begun. Four hours after final approval was granted by the Food and Drug Administration, a little girl received an infusion of her own white blood cells, which had been removed for genetic 'doctoring'. The patient has a deficiency of an enzyme (adenosine deaminase, ADA) because she lacks the gene responsible for making it. Her treated white blood cells contain the gene she needs. The principal investigators for the experiments are R. Michael Blaese, Kenneth W. Culver (both of the National Cancer Institute), and W. French Anderson (of the National Heart, Lung, and Blood Institute). Initial results were positive, and the patient left the hospital soon after the treatment. It is hoped that the child's immune system will be functional, with the addition of the ADA gene. Evaluation of the treatment's success will take several months. (Consumer Summary produced by Reliance Medical Information, Inc.)

Care and treatment, Case studies, Adenosine deaminase deficiency, ADA deficiency, Anderson, W. French, Blaese, R. Michael

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