I. Call to Order and Opening Remarks 
Dr. Walters called the meeting to order and noted that a system for rotation 
of the membership of the subccmmittee had been established. He said that 
the members whose terms would be completed in June 1987, Drs. Gorovitz, 
Grobstein, Motulsky, and Rich, would be invited to assist the subccmmittee 
as ad hoc consultants. 
II. Submission of Preclinical Data 
Drs. Anderson, Blaese, Nienhuis of the National Institutes of Health (NIH), 
and Dr. O'Reilly of Memorial Sloan Kettering Cancer Center, submitted a 
document entitled "Human Gene Therapy Preclinical Data Document" to the 
subccmmittee. [This document is not attached to these minutes because of 
its length but is available from the Office of Recanbinant CNA Activities.] 
Dr. Anderson stated that the document was submitted in response to an 
invitation that investigators supply preclinical data for information 
purposes as part of an education process for the subcommittee and submitters. 
It is not being submitted for formal review and approval at this time. He 
said he felt that many of the issues are not controversial, but several 
issues have no absolute answers. He said that each of the questions raised 
in the "Points to Consider in the Design and Submission of Human Saratic-Cell 
Gene Therapy Protocols" document had been addressed in a general way. He 
said that three critical questions, with no absolute answers, haw emerged: 
1. Will the postulated in vivo growth advantage of adenc6ine 
deaminase (ADA) plus cells in ADA-def icient patients be 
sufficient for clinical improvement? 
2. Will the vector-delivered ADA gene be expressed at a 
sufficiently high level in stem cells and their progeny 
so that the postulated _in vivo growth advantage will be 
maintained throughout the life cycle of the T-cell? 
3. Must the retroviral vector particle preparation be totally 
free of helper virus in order to make the risk/benefit ratio 
satisfactory? 
With regard to the last question, Dr. Anderson said that a totally helper- 
virus-free system is not currently available and may not be possible. 
In general, Dr. Anderson said that the submission had been prepared to 
provide data to the subccmmittee and the public, to clarify the issues, 
and to determine what additional information the subcommittee wants. 
Dr. Miller said that the document appears to be in a form acceptable to the 
Food and Drug Adminstration (FDA); he said new Investigational New Drug 
regulations placed more enphasis on safety than on efficacy in early 
clinical trials. 
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