Recombinant DNA Advisory Committee - 12/2-3/93 
examination of the data. Ms. Grossman stated her preference to defer approval of the 
protocol until the next meeting. Dr. Chase stated that the scientific review of these data 
could be adequately addressed by a subcommittee. Dr. Secundy disagreed with Dr. 
Chase's comments noting that public members offer valuable contributions to such 
reviews. 
Dr. Doi suggested that the RAC should specify their recommendations to the 
investigators. Dr. Miller said that the mice involved in the ongoing toxicity studies 
should be observed for a period of 4 months based on the fact that this length of time is 
required to evaluate engraftment of transduced stem cells in mice. Dr. Smith said that 
in addition to the animal data, the RAC should have access to Dr. Dunbar's data 
demonstrating the effect of growth factors on the ex vivo transduction of stem cells. Dr. 
Liu asked if he would be required to submit the additional data 8 weeks prior to the 
next RAC meeting and whether resubmission of the entire protocol would be mandatory. 
Dr. Secundy suggested that the Informed Consent document should be amended to 
include bone marrow examination as a condition for continuation in the protocol. Ms. 
Grossman, Drs. Parkman, Leventhal, and Zallen all stated that bone marrow 
examination with each reinfusion cycle should be an inclusion criterion in order to obtain 
scientifically meaningful data. Dr. Liu agreed to include this requirement in the protocol 
and in the Informed Consent document. 
Dr. Walters called for a vote on the motion to conditionally approve the protocol 
contingent on the review of data derived from ongoing safety experiments by a 
subcommittee of the RAC. The motion was defeated by a vote of 5 in favor, 10 
opposed, and 1 abstention. Dr. Parkman suggested that a new motion should waive the 
8 week deadline for submission and allow the investigators to resubmit the protocol up 
to 4 weeks prior to the next RAC meeting. 
Committee Motion #3 
A motion was made by Dr. Miller and seconded by Dr. Secundy to defer approval of the 
protocol until the investigators return to the full RAC with the following: (1) murine 
data demonstrating in vivo expression of the FACC gene and safety data accumulated 
over a period of * 4 months demonstrating that the FACC-transduced cells do not result 
in any untoward effects; (2) data as cited in Dr. Cynthia Dunbar's semi-annual data 
report (RAC protocol # 9206-025) regarding the possibility that "stem cell factor could 
favor the growth of leukemic versus normal progenitors during ex vivo culture periods;" 
and (3) revisions to both the protocol and Informed Consent document to modify the 
eligibility criteria regarding the necessity for bone marrow examination following each 
reinfusion. 
The consensus of the RAC was that the investigators are not required to submit this 
additional data until 4 weeks prior to the RAC meeting at which time the information 
will be reviewed. Submission of previously reviewed information is not required. The 
motion to defer approval of the protocol pending full RAC review of additional data 
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Recombinant DNA Research, Volume 18 
