Recombinant DNA Advisory Committee - 03/1-2/93 
Dr. Miller said that most protocols have been deferred based on the lack of safety data 
about the proposed vector and packaging cell line. Dr. Seigler has demonstrated safety; 
however, he is lacking data regarding the immune response. Dr. Miller said that if the 
RAC members insist that the investigators submit the required data, they should be 
allowed the opportunity to submit the data in response to a contingent approval. Dr. 
Motulsky agreed with Dr. Miller stating that the RAC should not stand on ceremony and 
precedence. 
Dr. Haselkom asked the RAC members to specify the exact information that should be 
submitted. Dr. Miller said that the CTL response data should be determined to be 
statistically significant. Dr. Geiduschek said that Dr. Motulsky is making the assumption 
that the RAC would be completely satisfied with the submission of additional data. Dr. 
Leventhal said that it is in the investigators' best interest to resubmit their data for full 
RAC review. 
Committee Motion 
A motion was made by Dr. Leventhal and seconded by Ms. Meyers to defer approval of 
the protocol. Dr. Miller asked the investigators if they had an anticipated time frame in 
which the vector would be available for transduction. Since the master and working 
banks have not been established and tested, additional review at the June RAC meeting 
should not significantly delay the process. Dr. Jolly responded that an additional 3 
months could introduce a slight delay. Dr. Leventhal reminded the investigators that the 
Food and Drug Administration (FDA) and RAC approvals are parallel processes; 
therefore, a delay in RAC approval will not affect their ability to obtain FDA approval. 
Dr. Miller asked the investigators if they have submitted their Investigational New Drug 
(IND) application to the FDA yet. Dr. Seigler responded that they had not yet 
submitted their application to the FDA. 
The motion to defer approval of the protocol was approved by a vote of 16 in favor, 1 
opposed, and 1 abstention. Approval of the protocol was deferred until data is 
submitted for full RAC review of the following: (1) generation of CTL from human 
PBL from tumor-bearing patients in response to transduced autologous tumor, (2) 
quantitative correlation between Class II antigen expression and the amount of y-IFN, 
and (3) quantitative correlation between the number of CTLs generated per unit of y- 
IFN. 
VIII. ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING A HUMAN 
GENE THERAPY PROTOCOL ENTITLED: PHASE I STUDY OF NON- 
REPLICATING AUTOLOGOUS TUMOR CELL INJECTIONS USING CELLS 
PREPARED WITH OR WITHOUT GRANULOCYTE-MACROPHAGE COLONY 
STIMULATING FACTOR GENE TRANSDUCTION IN PATIENTS WITH 
[94] 
Recombinant DNA Research, Volume 17 
