Human Gene Therapy Subcommittee - 7/30/90 
Dr. Parkman raised the question of financing of therapy not 
incurred on site at the NIH. Dr. Anderson said this issue was 
being discussed at all levels at the NIH and that it impacts on a 
number of protocols. He said the Clinical Center Director's 
Office and the Bioethics Program Office were trying to determine 
appropriate wording and that it would be included in the consent 
and assent forms. 
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 
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