Human Gene Therapy Subcommittee - July 29-30, 1991 
and TK- ratios. 
Dr. Parkman said the ratio of cells used in humans and mice probably cannot be 
extrapolated. The mouse tumor is transplantable with almost all the cells cycling. 
Human tumors may have a very different sensitivity to treatment because a large 
proportion of cells are noncycling. Further, mouse data may not generate any more 
useful information. However, there is a degree of efficacy indicated in the preclinical 
model. Whether that will be the translatable to human disease is unknown. The major 
question has to do with the delivery system. 
Dr. Parkman moved to provisionally approve the protocol with the stipulation that the 
PA-1 ovarian cancer cell line be tested for potential pathogens. This testing should be 
in accordance with FDA guidelines. Further, it was requested that there be more 
preclinical studies on the MFG vector to verify the absence of replication-competent 
retroviruses. Mr. Brewer seconded the motion. Dr. Leventhal asked to call the 
question. 
There being no opposition. Dr. Walters called the question. Dr. Walters put the 
motion to provisionally approve the protocol to a vote. The motion passed by a vote of 
7 in favor, 1 opposed, and 2 abstentions. 
Dr. Leventhal requested that the investigators expand their analysis of initial tumor 
volume in patients. She asked that the metabolic and renal status of the patients be 
examined more closely during the first few days. 
V. ROLE OF THE HUMAN GENE THERAPY SUBCOMMITTEE 
Dr. Walters noted that Mr. Capron's and Dr. Cook-Deegan's comments are in the 
written materials. 
Mr. Capron noted that most of the protocols reviewed in the last year has been "gene 
marking" rather than "gene therapy" protocols. The HGTS should not be reviewing 
gene transfer protocols. The HGTS was formed to review gene therapy protocols and 
to learn about vectors and the processes of review. The HGTS was created to be a 
balanced subcommittee with expertise to review these protocols and to provide 
recommendations to the RAC. The HGTS appears to operate as a separate, 
independent committee when it reviews protocols, although it reports to the RAC. The 
RAC reviews the subcommittee's discussions and recommendations and has the 
responsibility to report to the NIH Director. Given the potential load of gene transfer 
experiments, it may not be useful for the HGTS to continue to review all the gene 
transfer experiments which are not therapeutic or that employ recombinant DNA 
techniques to incorporate a change into the genome of a treated individual. 
Recombinant DNA Research, Volume 14 
[903] 
