Human Gene Therapy Subcommittee - July 29-30, 1991 
severely, it might become impossible to find patients in a reasonable amount of time. 
Also, there is a chance that one type of patient might react and another might not. 
There should be no further restrictions. 
Dr. Walters put the amendment to remove the restriction on the receptor classification 
of the patients to a vote. The amendment passed by a vote of 12 in favor, 1 opposed, 
and no abstentions. 
Mr. Capron moved to limit treatment to subjects who are capable of giving their own 
consent. The age limit will depend on what the Michigan Institutional Review Board 
(IRB) considers adequate. Certainly, there are children who have suffered with the 
disease and are capable at a younger age to give their consent. Mr. Brewer seconded 
the motion. 
Dr. Parkman said this is an important issue. However, it is important that children 
have equal access to innovative therapy, and there are reasons to believe that children 
may derive more benefit and provide clearer results. The treatment should not be 
limited. 
Mr. Capron said the treatment should start with adults. If it is shown to have any 
beneficial effects, parents could be asked to give consent for their children. 
Dr. Wilson said the chances of finding a receptor-negative patient who survives to the 
age of 18 is low. The other issue is that the treatment is much more feasible and more 
likely to be successful if it is performed in children. This is due to the increased 
success in the isolation of the hepatocytes and the efficiency of gene transfer in cells 
derived from patients. 
Dr. Mclvor moved to call the question, both on this amendment and on the entire 
issue. There being no objection to calling the question, Dr. Walters put the 
amendment to restrict the patients to those who can give their own consent. The 
amendment failed by a vote of 3 in favor, 9 opposed, and 1 abstention. 
Dr. Walters restated the original motion for provisional approval of the protocol. The 
motion required that: (1) the consent form changes be reviewed by a small working 
group of the HGTS, (2) additional data be submitted about the quality control of the 
vector system, the characteristics of the packaging line, and a protocol for quality 
control procedures following infusion of the transfected cells, and (3) the patient 
population may include both receptor-negative and receptor-deficient patients, either 
children or adults. 
There was a discussion on the reporting procedures in the case of provisional protocol 
approvals going forth from the HGTS to the RAC. Specifically, the discussion dealt 
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Recombinant DNA Research, Volume 14 
