Human Gene Therapy Subcommittee - 4/5/91 
type of information the subcommittee believes it needs to have for reviewing any given 
protocol. The document was written for the purpose of allowing investigators to come 
before the subcommittee with the knowledge of what information is expected of them. 
Rather than adding for the sake of clarity a sentence which is redundant, the original 
sentence should be modified and honed to properly express the subcommittee needs 
when considering the submitted protocols. 
Dr. Childress said perhaps Dr. Mclvor was really aiming his proposal at the 
subcommittee itself, rather than the investigators. It is important for the subcommittee 
to determine just what it considers sufficient preclinical data to support approving a 
protocol. 
Dr. Leventhal said that perhaps a second sentence should be added to Dr. Mclvor's 
proposed wording so that it is clear to the investigators that the subcommittee also 
requires some proof that the model system utilized is the best model available. 
Dr. Mclvor suggested the following wording for Dr. Leventhal's addition: 
"Describe the experimental basis, as derived from tests in cultured cells and 
animals, for the proposed system of delivery and explain why the models 
chosen is/are the most appropriate." 
Dr. Mclvor accepted this amendment to his motion, and Dr. Parkman agreed to the 
amendment as the person seconding the motion. 
There was continued discussion over the wording and syntax of the proposed 
amendment. Dr. Epstein moved that discussion of the motion be tabled until a point 
later in the day, after a small group had met to formulate satisfactory language for the 
amendment. Dr. Erickson seconded the motion to table. 
Dr. Walters called for a vote on the motion to table discussion on the motion. The 
motion to table passed by a vote of 9 in favor, 0 opposed, and 2 abstentions. 
Dr. Walters then called on Dr. Erickson to begin the discussion of the next agenda item. 
IV. PROPOSED ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING 
HUMAN GENE TRANSFER PROTOCOLS ENTITLED: (1) A PHASE //// TRIAL OF 
HIGH DOSE CARBOPLATIN AND ETOPOSIDE WITH AUTOLOGOUS BONE 
MARROW SUPPORT FOR TREATMENT OF STAGE D NEUROBLASTOMA IN FIRST 
REMISSION: USE OF MARKER GENES TO INVESTIGATE THE BIOLOGY OF 
MARROW RECONSTITUTION AND THE MECHANISM OF RELAPSE-, AND (2) A 
PHASE II TRIAL OF HIGH-DOSE CARBOPLATIN AND ETOPOSIDE WITH 
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Recombinant DNA Research, Volume 14 
