Human Gene Therapy Subcommittee - July 29-30, 1991 
Dr. Neiman said that the public concern with the application of gene therapy is not 
confined to human genome alteration. An equally important issue is the creation of 
new biological entities such as new pathogens. There is concern about the creation of 
vectors using retroviruses. The HGTS has to evaluate the implications of vectors and 
gene delivery systems being employed to improve public health. There could be 
significant potential to improve public health through the introduction of genetic 
material into humans. Initially, there was as much concern about the potential for 
introducing a live new virus as there was about the implications for the recipients. Dr. 
Neiman noted that the RAC has had working groups, including appropriate experts, to 
deal with new issues as they arise. The HGTS could use working groups with 
appropriate experts to deal with the issue of adventitial introduction of genetic material 
into the germ line. 
Dr. Erickson said Dr. Cook-Deegan's letter states the reasons for this review have to 
do with the sociology of the review process and public confidence. Dr. Erickson added 
that when an experiment has been performed a number of times with the same results, 
it becomes recognized as safe. When significant results have been supplied from the 
HGTS-approved marking experiments, the subcommittee could decide not to review 
future protocols of this type. The HGTS serves a useful function in reviewing the new 
classes of protocols. 
Ms. Areen said the HGTS should still review marking protocols. There is a real 
scientific benefit to the exchange between the investigators and the scientific members 
of the HGTS. The question to disband the HGTS is an issue for the RAC to decide. 
The RAC has to determine if the HGTS and RAC are performing duplicate functions. 
If not, they may need to think about ways of expediting HGTS approval. 
Mr. Brewer said it is very important to develop a framework for the review of germ 
line therapy protocols. Also, it would be useful to form a working group to standardize 
the review process. He asked to what degree gene therapy experiments could continue 
without the purview of this subcommittee if the research were wholly funded by private 
companies. 
Dr. Wivel said the NIH Guidelines must be observed if institutions or investigators are 
receiving NIH funds for recombinant DNA research, including gene therapy. 
Conversely, there is no requirement for a private company without NIH funding to 
comply with the RAC review process. Investigators would still require FDA approval. 
Dr. H. Miller said FDA does not have a position on this issue. He stated that he had 
been involved with the RAC for a long time and has been involved in FDA with the 
review process. Clinical research of all types is evaluated with relevant expertise in 
other forums. There is a great deal of clinical research that is performed without the 
benefit of HGTS or RAC review. Much research is reviewed only by Institutional 
Recombinant DNA Research, Volume 14 
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