July 30, 1990, Hunan Gene Therapy Subconmittee 
Dr. Leventhal said she wanted clarification as to whether NIH would pay outside 
physicians to treat patients closer to their homes, or whether the patients would have to 
return to NIH to be provided with free care. She suggested that, if the latter were being 
considered, perhaps discussion of care closer to home should be removed from the 
forms. Dr. Anderson said removal of the discussion may be the best alternative, so as 
not to mislead patients and families. 
Dr. Zallen asked that some statement be included that the treatments be "in the best 
interest of the patient," so that, as new therapies become available, patients will be 
advised of preferential treatments. Dr. Anderson said this is a common circumstance 
which occurs often in cancer therapy, and would continue to be the case for patients in 
this protocol. 
Dr. Walters said such a statement would not be a guarantee to the patients that the NIH 
would provide such treatment but merely a notice that "if something better comes along, 
we'll let you know." 
Dr. Walters noted consensus of the subcommittee that Dr. Anderson's suggestion of the 
Chairman providing final approval was acceptable for dealing with the consent and 
assent forms, and he suggested the subcommittee turn its attention to the other two 
issues still remaining, namely the i.p. route of administration and the Milan data. 
Dr. Mulligan asked whether the review of the Milan data was meant to be a review of 
scientific issues, or whether there were specific issues involved. Dr. Parkman replied that 
the "Points to Consider" document requires preclinical data supporting the mechanism of 
action of the proposed gene therapy. He said the one page summary, supplied 
previously, did not contain enough hard data to warrant a clearcut determination that 
these experiments fulfilled the criteria of being a preclinical model for this particular 
protocol. Dr. Mulligan asked how these issues could be resolved in light of the fact that 
the experiments had been done without this purpose in mind. Dr. Parkman said each 
member of the subcommittee would have to determine this on his own after reviewing 
the data. 
Dr. Miller said that the focus in FDA Phase I drug trials is threefold: the primary 
concern is safety to patients; secondly, data must be gathered on the pharmacokinetics 
and pharmacodynamics of the drug being tested; and thirdly, there must be a likelihood 
of efficacy in the patient population being tested. He urged that this protocol not be 
held to a different standard from routine drug trials. 
Dr. Parkman said that the investigators were putting this forward as a Phase I/Phase II 
trial. Because the patients are children, it is not deemed proper to do a toxicity study 
without looking for efficacy. Furthermore, he said the "Points to Consider" document 
does address the issue of efficacy. Dr. Miller said he understood this, but that looking 
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Recombinant DNA Research, Volume 14 
