Human Gene Therapy Subcommittee - 11/30/90 
Study may not take much time, and it would go a long way toward assuring the 
HGTS that the protocol was scientifically feasible. 
Mr. Capron asked whether the concern was over a recombinant risk or a viral risk to 
the patient. Dr. Parkman said that the risk is one of insertional mutagenesis in the 
hematopoietic cells if the patient is cured of his/her primary disease. Dr. Brenner 
noted that the patients in second remission has a very limited life expectancy. Dr. 
Parkman countered that those in first remission did have a significant life 
expectancy, and that insertional mutagenesis was a concern in those patients. 
Ms. Meyers said that the issue is not the patient's risk in participating in the 
protocol, but whether or not all the basic research has been conducted to assess both 
the risk and the scientific knowledge to be gained. For years, the emphasis in this 
field had been on going slow to assure that all the basic science is conducted before 
treating human subjects. Humans should not be experimented on even if they have 
a poor prognosis for survival. 
Dr. Mclvor said it is important to note that the HGTS approved the first gene 
transfer experiment into bone marrow earlier in the day, and the risk-benefit 
requirements are pretty stringent at this point in time. It is imperative to see some 
indication that it is possible for a protocol to yield expected results. In the future, 
gene transfer experiments may be found to be risk-free. Right now the risk 
assessment is only an estimate. 
Dr. Parkman said that there was a hierarchy in looking at this type of situation. 
First, the investigators must have the demonstrated competence in performing the 
experiment. Secondly, the experimental design must indicate technical competence 
and lead to a legitimate scientific answer. Only after these two questions are 
answered can one begin to consider risk-benefit issues. 
Dr. R. Murray said that he had concerns over the principles governing research with 
human subjects. If an experiment had essentially no evidence of outcome, human 
dignity implies that it should not be performed. 
There being no further discussion. Dr. Walters restated the motion "to defer this 
protocol to a future meeting of the HGTS and asked the investigators to perform 
additional transduction experiments in vitro." Dr. Walters called for a vote on the 
motion. The motion passed unanimously by a vote of 10 in favor, 0 opposed, and no 
abstentions. 
FUTURE MEETING DATES 
Dr. Walters reminded the HGTS that the next meeting would be on April 5, 1991, 
and a list of meeting dates through July 31, 1992, is included in their materials. 
Recombinant DNA Research, Volume 14 
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