Recombinant DNA Advisory Committee - 06/1-2/92 
Dr. Leventhal stated that since this study is not designed for a therapeutic effect, 15 
patients is excessive for a Phase I study. 
Dr. Parkman asked Dr. Smith if he was formally withdrawing the autologous protocol for 
consideration by the RAC? Dr. Smith stated that he is formally withdrawing the 
autologous protocol. 
Dr. Parkman stated that the syngeneic protocol is designed to stimulate PBLs from a 
normal sibling with OKT3 and IL-2 and to transduce them with retroviral vector 
supernatants. Since the investigators have only performed this transduction experiment 
one time, no data are available regarding the efficiency of transduction. The 
investigators have a responsibility to present efficacy data in an analogous system prior to 
submission of a protocol to the RAC for formal review. 
Committee Motion 
Dr. Post moved to defer approval of the syngeneic protocol. Dr. Secundy seconded the 
motion. 
Dr. Leventhal stated that when the investigators resubmit this protocol, the investigators 
must: (1) include extensive animal toxicity data, (2) clearly define acceptable levels of 
transduction and provide justification regarding the number of patients to be treated, and 
define protection. 
Dr. Smith asked the RAC to provide guidance as to what would be an acceptable range 
of in vitro experiments. Dr. Geiduschek cautioned that it is not the responsibility of the 
RAC to establish acceptable numbers for experiments. It is the responsibility of the 
investigators to provide adequate data to justify the approval of their protocol. 
Dr. Smith asked the RAC if the simian immunodeficiency virus animal model would be 
required. Dr. Parkman responded that the investigator should choose the most 
appropriate in vivo model. In vivo models are certainly better than in vitro models, and 
the SCID mouse model would provide useful information. 
Dr. D. Miller stated that there may be monkeys available that already have SIV at a 
particular stage. Perhaps if the investigators contacted primate centers, these monkeys 
may be available to them. The autologous experiment could be performed in this 
instance. 
Dr. Geiduschek asked if the over production of TAR sites resulted in the synthesis of tat 
protein? These experiments should be performed. 
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Recombinant DNA Research, Volume 15 
