July 30, 1990, Human Gene Therapy Subcommittee 
Dr. Parkman reiterated his concerns about multiple protocols being put forward under 
the rubric of a single proposal and said he felt it would be easier to review them as 
separate proposals. Dr. Brenner said that despite there being three different protocols, 
the only issue for the subcommittee was the gene marking portion of the experiments, 
which was identical for all three. Dr. Parkman said that was true; however, the reason 
for performing the gene manipulation in each case is different and must be viewed 
separately in terms of risks and benefits. 
Dr. Anderson said the only problem with submitting them as three separate protocols is 
that there would then be a necessity to submit three responses to the "Points to 
Consider," three sets of CV's, and three sets of documentation. The result would be 
even more difficult in finding information contained within the material. 
Drs. Mulligan, Leventhal and Parkman discussed various assay techniques that could be 
employed in trying to determine acceptable preclinical data in each of the three diseases 
under consideration. Dr. Mulligan summed this up as requiring assays: 
1. To demonstrate infection of the range of hematopoietic cells 
of interest, so that the progenitor cells giving rise to the 
cancers could be ascertained; 
2. To answer questions of the necessity of growth factors, the 
difference of infection between purged and unpurged cells, 
and the difference between marrow from previously treated 
patients versus patients who are non-ablated. 
Dr. Mulligan moved that further review of the protocol be deferred until a proper 
collection of preclinical data could be made. Mr. Brewer seconded the motion. 
Dr. Miller suggested a mechanism be set up whereby an investigator could meet with 
either a subcommittee of the HGTS or perhaps individual expert members, or be able to 
come in and seek guidance in protocol preparation at various stages before formal 
submission of protocols. 
There being no further discussion of the motion. Dr. Walters put it to a vote. The 
motion passed by a vote of 13 in favor, none opposed and no abstentions. 
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Recombinant DNA Research, Volume 14 
