Human Gene Therapy Subcommittee - July 29-30, 1991 
A protocol could be unconditionally approved, conditionally approved, or deferred 
pending further data before proceeding to the RAC for consideration. Mr. Capron 
said the HGTS is in a position to either fully endorse a protocol or send it to the RAC 
with stipulations. He agreed that there may be a third situation of requiring the 
protocol to return to the HGTS before proceeding to the RAC. 
Dr. Leventhal proposed that the working group discussions should also focus on 
creating a review mechanism designed to provide the subcommittee with information 
that has been learned from the progress of approved protocols. Did the treatment go 
as well as expected? Is there something the HGTS and RAC should have asked during 
the review? Has the experiment been done, and was the experiment successful? This 
analysis should be impartial and not merely performed by the investigator responsible 
for the study. 
Dr. Mclvor agreed that the review process of protocol results should involve a report 
from the investigators and a formal evaluation by the HGTS. Before protocols come 
to the HGTS, they should contain an analysis of the packaging cells for replication 
competent helper virus. They should also demonstrate gene transfer and expression in 
cultured human cells that will be targeted by the therapy in the patients. If the 
investigators have not provided these data in their preclinical work-ups, the protocols 
should not receive HGTS approval. 
Dr. Erickson said that as the RAC divests itself of other duties, all of these proposed 
new HGTS duties ought to be done by a committee that is part of RAC. It is still 
valid to try to merge the HGTS and the RAC. 
Mr. Brewer said the subcommittee should be careful not to overload Dr. Wivel and his 
small staff. The systematization of these concerns can be communicated in a checklist 
form to investigators, as with the Points to Consider. 
Dr. Wivel restated the three points of Mr. Capron's motion: (1) ORDA staff should 
screen the protocols with regard to the Points to Consider, so that the documents which 
come to the HGTS are complete and thereby expedite the process; (2) a working group 
should be established to classify vectors and/or other parts of protocols which may be 
considered more routine in nature; (3) a working group should be established to 
discuss the objectives of this committee over a wide range of issues. 
Dr. Walters put the motion to a vote. The motion passed by a vote of 11 in favor, 0 
opposed, and no abstentions. 
VI. FINAL LOCAL IRB AND IBC APPROVAL BEFORE REVIEW OF PROTOCOLS 
Dr. Walters stated that this is an important policy decision regarding what IRB and 
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