June 1, 1990, Human Gene Therapy Subcommittee Minutes 
Dr. Parkman said he was still opposed to the IP route of administration. Despite the 
glycosylation argument posed by Dr. Blaese, Dr. Parkman would still like to see 
comparative studies of IV versus IP administration in animals to substantiate this 
hypothesis before using it in the protocol. Further he said the subcommittee would have 
to decide if the Milan data could be extrapolated to the Bethesda data in terms of it 
being preclinical data appropriate to recommending approval of the trial. 
Dr. Anderson said that the Milan group had been collaborating with them for five-sk 
years, that they exchange data constantly, and they exchange techniques and vectors. 
The two systems are very similar and only through testing in humans can better and safer 
techniques be developed. 
Dr. Leventhal said it should be clearly stated that if any malignancies are found, the 
investigators will attempt to determine if their vector is in any of them. Dr. Anderson 
agreed. Dr. Leventhal also said the parents had to be made aware of what to look for to 
spot early symptoms of malignancy so that they can be able, especially at long distances, 
to get tissue to NIH for testing. Dr. Anderson agreed. 
Dr. Neiman discussed the issue of patient selection and oncogenesis. Dr. Walters asked 
what types of malignancies were caused by mutagenesis. Dr. Neiman said that it is 
generally a malignancy of the cell involved. In this case, they would be primarily T cell 
lymphomas. However, it would not be out of the question to see epithelial cell cancers, 
breast cancers, and possibly some leukemias. 
Dr. Mahoney asked whether the investigators would return to the IRB or the 
subcommittee before making a move to go to Part 2B of the protocol. Dr. Blaese said 
he was happy in going to the IRB with this as he feels this kind of decision is more 
realistically handled at the local level. Dr. Anderson said the NIH Institutional Biosafety 
Committee (IBC) had given unconditional approval of Part 1, but the investigators have 
to go back before that committee for Parts 2A and 2B. He said it would be helpful to 
have as few restrictions as possible above the local level. 
Dr. Walters asked whether the investigators wanted the subcommittee to approve of 
enrolling patients into Part 2B as well as Part 1 and Part 2A. Dr. Blaese said he would 
prefer to have the protocol considered as it stood. 
Dr. Mclvor said the Milan data was only a summary. He asked to review the actual data 
to check the conclusions before making any decisions. Dr. Parkman asked if Dr. 
Bordignon would share the data. Because of time constraints Dr. Bordignon was not 
able to send a complete manuscript with all the necessary tables and, therefore, supplied 
the summary. 
Dr. Walters called upon members of the public for comment. Dr. Palella from the 
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