7 
other tissues. Dr. Miller wrote the section might request information on "the 
specificity of transformation or other effects on the target ceils." 
Drs. Anderson and Gottesman said they preferred to leave the phrase "specifically 
germ line cells" in the points to consider document. The subworking group agreed. 
Car. Walters called the sukworking group's attention to Car. Miller's comment con- 
cerning Section I-B-2-c-(l)-(e) of the points to consider document. Dr. Miller 
had suggested the first sentence of this section be revised to read: "Describe 
animal experiments completed or in progress employing protocols similar to 
that proposed." 
Dr. Anderson said Dr . Miller had not grasped the importance of primate studies; 
these very important studies should be emphasized in the document because 
endogenous primate retroviruses differ from those in mice or other animals. 
He felt very strongly that this language should be in the document. 
Dr. Waiters said rar. Sheldon Horowitz of the University Hospital and Clinic 
of the CJhiversity of Wi scons in-Madison, Madison, Wisconsin, had c ament ed that 
prior primate testing should not be required in urgent cases. Dr. Horowitz 
said he personally vas treating a young girl with ADA deficiency and severe 
combined immune deficiency vho should receive gene therapy as soon as possible. 
No other therapy is possible for this patient. He felt he had about 12 months 
in which to treat this child. The urgency of this case would not leave sufficient 
time to perform primate testing. 
Ms. Areen said the information requiranents in the points to consider document 
are flexible; the document simply states the type of information the working 
group wishes to review. Decisions would be made on a case-by-case basis. 
Dr. Anderson said the document was not requiring a priori that primate testing 
be performed prior to human gene therapy. The language of this section had 
been carefully drafted to simply inquire whether primate testing has been done 
without requiring it for approval. 
Dr. Gottesman agreed primate testing is not an a priori requirenent; she suggested 
the document emphasize the flexibility of the points to consider. Or. Walters 
suggested the points to consider document should explicitly state that proposals 
would be reviewed on a case-by-case basis. Cr. Childress thought the working group 
might also indicate the rationale for developing a "points to consider" document. 
Dr. Walters said Dr. Miller's next comment was that the criteria for selection of 
subjects should be included in Section I-B-3 rather than in Section I-C of the 
document. 
Dr. Walters said the selection of subjects issue has two components. These 
are: (1) who receives a scarce resource if several people are competing for 
access; and (2) how subjects are chosen as part of the research design in a 
large randomized clinical trial. Cr. Walters said Section I-C addresses the 
first of these issues rather than the second. The subworking group agreed the 
position of this section in the working group document was correct. 
[ 7 ] 
