Human Gene Therapy Subcommittee - 11/30/90 
additional preclinical data needs to be presented indicating that more than 1% of 
cells could be infected. 
Dr. B. Murray noted that so far none of the discussion had centered on issues of 
recombinant DNA and human gene therapy, only BMT. The local review 
committees should review the other science, and the HGTS should concern itself 
only with those issues of recombinant DNA and its relationship to human gene 
therapy. 
Dr. H. Miller offered a decision tree paradigm for looking at this type of experiment. 
The first question would be to look at issues of safety. In this case, the incremental 
risk to a patient who is suffering from leukemia and is scheduled to undergo BMT is 
infinitesimal. The next question would be to determine if some useful information 
would be derived from the experiment. Although the protocol was not outstanding, 
there was a finite likelihood that useful information would be derived. He urged the 
HGTS to make constructive suggestions for its improvement. 
Dr. Epstein suggested that during lunch Dr. Parkman and a small group of reviewers 
should devise a motion concerning the protocol outlining suggestions. 
Dr. Anderson agreed that Dr. Cometta needed more preliminary data to support the 
protocol. By the next RAC meeting, there could be sufficient data in 3-4 patients to 
show a 10-20% transduction rate. A small group of reviewers could examine the 
new data before the next RAC meeting. If such data supports the protocol, it could 
be voted on by the RAC at its February meeting, rather than delaying the 
experiment for seven months by returning to the HGTS and going to the RAC at its 
May meeting. 
Dr. Parkman said he was not personally comfortable with a review conducted by a 
small group and rendering a decision for the entire HGTS. The level of data 
presented thus far is so low that gathering enough information would take at least 2- 
3 months, which would not allow time for review before the February meeting of the 
RAC. Further, most HGTS members would want to have access to discussion of the 
data before approving this protocol. 
Dr. Walters said that perhaps the best strategy would be to have a group of 
reviewers meet to refine the description of the kinds of additional data needed. 
Perhaps defining the reviewers needs would place the entire HGTS in a better 
position to decide on whether to defer or approve the protocol pending submission 
of the additional data. Dr. Walters then asked Drs. Gellert, Epstein, Zallen, 
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