Human Gene Therapy Subcommittee - 11/30/90 
Dr. Erickson said that the same questions as to percentage of cells and methods of 
detection still remain. Neuroblastoma is probably more likely to be clonal, but he 
did not know if clonality could be confirmed. 
Dr. Erickson said that the final difference between this protocol and the earlier 
protocol is that there are two very different groups of patients that will be studied. 
One group are in Stage D of their first remission and will receive high dose 
chemotherapy and autologous bone marrow transplant. The second group of 
patients are refractory to chemotherapy and have relapse. The second group is 
expected to have a much lower survival rate than the first group. 
There is concern over using retroviral marker genes in a group of patients with a 
very high survival rate. Further, many of the other concerns that were expressed 
about the previous protocol are also concerns in this case. 
Dr. Mahoney said that once again a major issue was transduction efficiency in 
neuroblastoma tumor lines. The data presented in the protocol suggests a 
transduction efficiency of between 10 and 30%. Could such rates be obtainable in 
patients? The protocol paralleled the AML protocol closely and had the same 
probability of useful information being generated from it without significant risk to 
the patients. Neuroblastoma of this type is a pediatric disease; therefore, it is 
appropriate for this protocol to begin in children. 
Ms. Meyers said that the informed consent document stated "The intent of the 
experiment is to test two drugs," but noted that it did not seem to be the intent of 
the experiment. Dr. Brenner replied that a word was missing, the statement should 
read "The therapeutic intent of the experiment..." Ms. Meyers added that she had 
the same concern about publicity and privacy in this protocol that she had in the 
previous protocol. Dr. Brenner said this concern would be addressed in the 
informed consent document. 
Mr. Capron said that there was some sensitivity in the ethics community to the use 
of the phrase "therapeutic research," and he a^ked the investigators if this marker 
study will produce therapeutic results. The phrase could be misleading to the 
patient. Ms. Meyers said it should really state that there are two goals to the 
research. Mr. Capron agreed and said it should read "It has two goals. One, we will 
give you therapy, and the other we will study this marker." 
Dr. Parkman said that the strength of the previous proposal was that the 
investigators had demonstrated the ability to insert their retrovirus into the cells of 
interest in primary culture, and that these cells were able to be grown up and 
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Recombinant DNA Research, Volume 14 
