Human Gene Therapy Subcommittee - 7/30/90 
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 i 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 
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