4 
Dr. Motulsky agreed with Mr. Capron and said unless informed consent considerations 
for human gene therapy differ fr on other therapies, the working group should 
not spend much time discussing these considerations. He asked if the introduction 
of HWA into a patient would raise different informed consent issues. Dr. Miller 
contended it would not. He offered an example of a therapy involving introduction I 
of DNA into human subjects: the polynucleotide poly IC is administered to 
patients to induce interferon production. 
Dr. Mahoney said although gene therapy does not present many issues vhich differ 
significantly frcm those confronted with other types of therapy, the expectation 
being created in the public mind differs frcm the way in which science progresses. 
The public expects this therapy to provide cures; however, a partial amelioration 
of the downward course of these illnesses is an acceptable therapeutic goal. 
Dr. Walters agreed and said human gene therapy may be analogous to the treatment 
of childhood leukemia where no single treatment affected a cure but used together 
these treatments have greatly improved the survival rate. 
Dr. Miller said human gene therapy will not present considerations different 
from those already seen and faced everyday with other therapies. He thought 
the subgroup draft document overemphasized informed consent issues and did not 
offer enough detail on the types of scientific information the investigator 
should provide for review. Dr. Miller said the working group should pro/ide 
a data checklist for investigators and not create guidelines or forums for 
public opinion. 
Dr. Grobstein said the working group has two o/erlapping responsibilities: (1) 
to review proposals from a technical aspect, and (2) to function in the formation 
of public policy. Although these two reponsibilities overlap, they should not 
compete and both aspects must be considered by the working group. Dr. Grobstein 
pointed out that public perceptions, vh ether correct or incorrect, are part of 
public policy and must be considered. Mr. Mitchell agreed with Dr. Grobstein. 
He said legitimate public interest is the degree to which the public is 
interested . 
Dr. Grobstein said a working group draft document should have two sections: the 
first section should set the context for review, and the second section should 
detail both technical and public policy considerations. 
Dr. Grobstein felt the working group document should inform the IRB and the 
Institutional Biosafety Committee (IBC) of \hat RAC and its walking groups would 
evaluate in the review process. 
Dr. Gottesman said the working group will eventually be asked to review proposals 
involving germ line gene therapy; this type of therapy is probably the public's 
major concern. For the more immediate proposals involving somatic cell therapy, 
the working group must translate scientific information for the public and 
explicitly explain why somatic cell gene therapy presents no special considerations 
beyond those encountered in other therapies. 
Dr. Gottesman suggested the preamble of a working group document should explain 
the review process. The proposal would first be reviewed by the local IRB to 
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