Human Gene Therapy Subcommittee - 4/5/91 
proof be supplied that the model used to show efficacy was the best model for whatever 
human application is being contemplated. 
Ms. Meyers cautioned the subcommittee to ensure that the public does not have the 
perception that human beings are being experimented upon in cases where animal 
studies could be used. It is important that, if a question can be answered by animal 
studies, that animals used, and that human beings continue to be held in the highest 
regard. It is important that animal studies be required, so that ideas are not taken 
directly from the laboratory and applied to humans without first having some preclinical 
data. 
Dr. Blaese felt the language currently in the Points to Consider was sufficient. Making 
the language stronger or setting certain goals may lead to an enormous increase in the 
expense of performing human gene therapy experiments without leading to further 
progress in either the clinical application or the ability to predict what will actually work 
in the human situation. At present, each protocol is reviewed in detail, and the 
subcommittee can ask for specific preclinical data if it is not satisfied with what is 
supplied in the proposals. He expressed his doubts that codifying a more rigid approach 
would have any benefit. 
Dr. Parkman said the problem was that protocols had been coming to the subcommittee 
without the necessary data. This amendment would reinforce the expectation of the 
subcommittee that such data be included in the protocols when they are first presented 
for review. 
Dr. Anderson said there is no need for making the rules more strict when the system had 
proven to work well. If the experiments which were approved had not worked, then it 
might be time to reassess whether enough preclinical data had been presented. 
Dr. Mclvor said that the current data were encouraging, but by no means conclusive. 
This amendment would state more effectively what the subcommittee would need to 
have insofar as preclinical data to facilitate its work. The major focus of all of the day's 
discussions would be on this very issue-consideration of anticipated efficacy based on the 
inclusion, or absence, of preclinical data. 
Dr. Leventhal felt the amendment was reasonable and that a major weakness of all the 
protocols that had been considered in the last sk months had been the demonstration 
that a gene could actually be transferred to a target cell. The most important aspect of 
this whole issue is how one defines "efficacy," and it is important that the subcommittee 
be clear as to what it considers efficacy. 
Mr. Capron noted that the Points to Consider were not regulations, but statements of the 
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