July 30, 1990, Human Gene Therapy Subcommittee 
whether they are naturally IL-2 responsive and what happens to them 
without growth factors; 
3. Whether intracellular expression of the enzyme is more beneficial than 
direct infusion; and, 
4. Whether it is possible to introduce enough modified cells to result in the 
necessary detoxification. 
Dr. Mulligan said he felt it was important to have answers to these questions before the 
preclinical data could be reviewed in relation to the protocol. 
Ms. Meyers said she was surprised to see that Dr. Zallen's critique of the consent form 
was so like her own, despite her not having communicated with Dr. Zallen. Ms. Meyers 
said they both came up with virtually the same questions, mainly dealing with who will 
cover the cost of follow-up and continuing care once the children have left the NIH and 
gone home. Should the NIH make some guarantee to cover some medical expenses in 
light of the fact that the children will be uninsurable? Most general practitioners may be 
unable, or unwilling, to deal with such things as severe infections because of their lack of 
knowledge of gene therapy. 
Dr. Zallen said she noted that the investigators had promised to provide this care to the 
family indefinitely. She asked that if there were to be any restrictions on long-term care 
that the families know in advance what those restrictions may be. 
Dr. Walters asked if the subcommittee wished to hear from Dr. Bordignon. Dr. 
Parkman said he felt it would clarify issues and make it easier for the members of the 
subcommittee to make a decision on the protocol. 
Dr. R. Murray asked what the status was of the requests for time to make presentations 
which were previously submitted in writing and whether it was felt that such 
presentations would help in understanding the technical aspects of the research that has 
been proposed. 
Dr. Parkman said he had discussions with both the investigators and Dr. Wivel. 
Presentation of data in a public forum is a difficult issue for the investigators, as many 
peer reviewed journals have difficulty publishing work which has been presented in 
public. However, he said he thought the primary responsibility of the subcommittee was 
to set the precedent in this and try to gain as clear an understanding of this proposal as 
possible. He reiterated that Dr. Bordignon was not an integral part of the team seeking 
approval of the protocol and therefore it should be recognized that he was in a 
somewhat precarious position relative to making his data public. 
Recombinant DNA Research, Volume 14 
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