6 
Dr. Gorovitz said in a democracy no individual should be so disparaging as to 
call attempts to inform the public "pandering." He said seme segments of the 
public are genuinely concerned about the potential applications of human gene 
therapy, and these concerns must be taken into account. He reminded the group 
that the courts are open to the full spectrum of the public, and the influence 
of public groups will be felt through the courts. 
Dr. Gorovitz said one way of protecting the public interest is to defend against 
the loss of worthy beneficial treatments because of actions by an uninformed 
segment of the public. He reminded the ccrmittee that one injunction can 
block research that has sailed through technical review. He said by educating 
the public the working group would minimize the extent to which ill-informed 
segments of the public exert influence. 
Dr. Walters suggested the working group proceed through the subgroup draft 
documents (Attachment II) point by point. 
Dr. Gottesman reiterated that the entire working group document should be 
prefaced by a preamble which would state the scope of the document, the 
review procedure to be followed, and the purpose of the review. The 
preamble might also address the issue of vh ether there are any considerations 
unique to human gene therapy; this might include questions of whether risks 
exist in the procedure or whether the recombinant vector might spread . Ethical 
implications, particularly as they apply to somatic cell therapy as opposed to 
germ line cell therapy, could also be mentioned. 
Dr. Gottesman thought the preamble to the working group document should offer 
general advice to the investigator; requests for specific data and information 
would appear in the body of the document. She thought specific examples of 
diseases currently thought to be amenable to gene therapy should net be listed 
in the preamble as she felt this might eventually restrict both the investigator 
and the working group. 
Dr. Gorcvitz suggested the working group document should be prefaced by the 
preamble suggested by Dr. Gottesman, should then call attention to all existing 
applicable regulations, and should finally specify what additional information 
would be required by the working group for RAC review of protocols involving 
human gene therapy. 
Dr. Capron suggested the preamble might indicate why such a structure was 
chosen. He said the preamble might also indicate IRB responsibilities, and 
then state that for reviews of human gene therapy protocols the RAC and its 
working group® would evaluate specific additional information. 
Dr. Mot ul sky suggested the introductory preamble to the document should state 
that the "points to consider" document has been constructed to aid RAC and 
working group review and perhaps to aid IRBs and investigators and is not 
intended to be rigid policy. Dr. Gottesman agreed; she said the RAC should 
accept this as a points to consider document which will evolve as the technology 
evolves. A points to consider document would be a working document and not 
part of the NIH Guidelines for Research Involving Recombinant ENA Molecules. 
[ 94 ] 
