Recombinant DNA Advisory Committee - 3/3-4/94 
GTAC has approved 6 human gene transfer trials to date; two of these trials are 
currently in progress, one for the treatment of cystic fibrosis and one for the treatment of 
adenosine deaminase deficiency (Netherlands collaboration). Studies that have not yet 
been initiated involve: (1) cytokine-mediated therapy in malignant melanoma, (2) a 
neuroblastoma marking study (United States collaboration), and (3) a lymphoma vaccine 
trial. These trials are currently awaiting approval from the Medicines Control Agency 
(the United Kingdom's equivalent of the Food and Drug Administration). GTAC is 
currently in the progress of reviewing an additional 4 studies involving cancer, cystic 
fibrosis, and HIV infection. 
GTAC differs from the RAC in that its meetings are not open to the public. However, 
Principal Investigators are encouraged to publish their protocols in appropriate journals, 
and GTAC is considering the publication of an annual technical report regarding the 
activities of the committee. GTAC does not operate under statutory regulations; 
however, the Department of Health guidelines require that GTAC review all human 
gene transfer proposals. All biomedical research in the United Kingdom must be 
reviewed and approved by local research ethics committees. 
Dr. Parkman asked how often the GTAC meets and how its review process differs from 
the RAC review. Mr. Taylor responded that the Secretariat and the Chairman of GTAC 
screen all human gene transfer studies prior to GTAC review. The committee review is 
very similar to the RAC review in that one or two primary reviewers are assigned for 
each proposal. GTAC currently meets 5 times a year; however, this schedule may be 
modified to accommodate 6 meetings a year. Essentially, a given proposal will be 
cleared within a 3-month period. Dr. Motulsky inquired whether the gene transfer 
studies proposed in the United Kingdom differ from those approved by the RAC. Mr. 
Taylor responded that 3 of the first 6 trials approved by the GTAC utilized non-viable 
DNA/liposome or plasmid DNA delivery systems. Ms. Grossman asked whether there is 
any data reporting system to monitor research progress. Mr. Taylor said that the gene 
therapy trials are still in the very early stage in the United Kingdom and no such system 
has been in operation yet. Dr. Post asked whether there is any plan to establish a 
registry to track patients who have received gene therapy. Mr. Taylor responded that the 
plan to establish a registry is under consideration. Dr. Doi asked about coverage of the 
medical costs in the United Kingdom regarding gene transfer studies. Mr. Taylor 
explained that the National Health Service covers all medical costs. Dr. Smith asked 
whether there is any reimbursement to the National Health Service for trials sponsored 
by a company. Mr. Taylor responded that there is no system to recoup the costs for 
company-sponsored research. All the current studies are funded by the government and 
non-profit charities. Dr. Motulsky asked if Mr. Taylor knew of any additional oversight 
committees for human gene therapy besides the United States and the United Kingdom, 
such as other European countries or Japan. Mr. Taylor responded that Japan has 
recently established a dual mechanism for reviewing gene therapy protocols. In the 
Netherlands, Denmark, and Germany, protocols are reviewed through existing national 
advisory committees on genetic modifications. 
Dr. Marcel made an unofficial report on gene therapy review in France. There is a 
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