17 
as part of the proposal. She said investigators might not realize the implication 
of patents will be discussed if the working group document does not mention 
public interest in this aspect of the application of human gene therapy. 
Dr. Grobstein asked what the working group would do if the research! had been 
supported by public funds but the results of the research will be patented. 
Cfcrs. Mahoney and Goro/itz said the response to this question would not influence 
the technical evaluation of the proposal and probably would not bear on whether 
approval would be given or not but should be public information used in arriving 
at public policy decisions. 
Dr. Mahoney pointed out that human gene therapy sits at the peak of millions of 
dollars of public funds dispensed for research; he felt the public has the 
right bo knew the cute erne of this financial support. 
Dr. Miller said if human gene therapy is to become widely applicable, it must be 
developed by industry. Dr. Goro/itz said human gene therapy will not be applied 
on the same scale as vaccines. 
Dr. Gorovitz emphasized that the question is simply a request fbr information. 
He said the working group has a two part agenda, and what may not be relevant 
technically may be relevant to public policy considerations. He said at some 
point RAC or the working group might make a public policy recommendation on the 
basis of reviewed protocols . 
Dr. Grobstein, referring to Section II-C, Impact on Germ Line , asked how the 
investigator would determine whether the gene therapy methods cited in the 
protocols would affect the sperm or egg cells of patients. Dr. Anderson said 
the investigator might generate this information from animal model systems. 
Dr. Grobstein said animal system results may not be applicable to humans. 
Dr. Miller suggested sperm counts or testicular biopsies might be performed 
in sexually mature patients. Dr. Anderson said candidates for human gene 
therapy are usually sterile as part of the natural history of the disease. 
Dr. Grobstein said Section II-D, Triplications , should mention issues of patient 
welfare and public health. 
Dr. Walters asked the working group whether they thought the document should 
contain specific language indicating the document is open to revision. 
Drs . Grobstein, Gottesman, McCarthy, and Mahoney said the document would be 
open to revision, but the document need not explicitly include such language. 
Mr. Capron said necessary modifications may be suggested by the review of 
the first protocol. 
Dr. Walters asked the working group what process should be followed in sending 
this document to the RAC for its approval. The working group agreed that 
following the October 12, 1984, meeting of the working group. Dr. Walters and 
a group of volunteers would meet to revise the working group document. That 
draft would subsequently be mailed to working group members for their comments. 
The working group would then meet on November 16, 1984, to continue to polish 
the document. When the working group feels the document is ready, the document 
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