Recombinant DNA Advisory Committee - 09/14-15/92 
most appropriate animal model. Models are not the same as wild-type human disease. 
Dr. Roth stated that only serum free supernatants will be used for treatment of patients. 
Therefore, an immune response to fetal calf serum is not a concern. 
Dr. D. Miller suggested that data should be provided demonstrating K-ras induced foci 
and this rate of transformation should be compared with the supernatant that will be 
used for the clinical protocol. If 10 7 CFUs corresponds to 10 milliliters of supernatant, 
then the investigators should demonstrate that there is no transforming virus in this 
volume or 100 milliliters. Dr. Post suggested that mixing experiments should be 
included. 
Committee Motion 
A motion was made by Dr. D. Miller and seconded by Dr. Krogstad that the protocol be 
approved contingent on the review and approval of the following information by Drs. D. 
Miller, Hirano, and Geiduschek: (1) submission of data demonstrating the transforming 
potential of 100 milliliters of retroviral supernatant analogous to the preparation that will 
be used for the clinical protocol, (2) submission of data obtained from in vitro mixing 
experiments, (3) submission of in vitro data demonstrating that the new vector 
preparations have activity, and (4) incorporation of minor changes in the informed 
consent document as noted by Drs. Carmen and Hirano. Dr. Murray called for a vote. 
The motion passed by a vote of 18 in favor, 0 opposed, and no abstentions. 
XI. ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING A HUMAN 
GENE TRANSFER PROTOCOL ENTITLED: A PHASE II TRIAL OF THE BAXTER 
NEUROBLASTOMA BONE MARROW PURGING SYSTEM USING GENE MARKING 
TO ASSESS EFFICACY /DRS. BRENNER AND MILLS 
Review-Dr. Haselkorn 
Dr. Murray called on Dr. Haselkorn to present his primary review of the protocol 
submitted by Dr. Malcolm K. Brenner of St. Jude Children's Research Hospital, 
Memphis, Tennessee, and Dr. Bonnie J. Mills of Baxter Healthcare Corporation, Santa 
Ana, California. In Mr. Barton and Dr. Brinckerhoff s absences, Dr. Haselkorn 
summarized their comments in addition to his own remarks. This Phase II protocol of 
the Baxter neuroblastoma bone marrow purging system, based on magnetic bead 
separation of cell populations using gene marking to assess efficacy. This protocol is 
very similar to the neuroblastoma protocol submitted previously by Dr. Brenner. The 
patient's bone marrow is harvested and separated into two fractions, purged and 
unpurged. The purged fraction will undergo separation through the magnetic bead 
column. Each fraction will then be marked with distinguishable retroviral vectors. At 
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