Human Gene Therapy Subcommittee - 7/30/90 
Whereupon, there being no further discussion on the motion. Dr. 
Walters called for a vote. The motion passed by a vote of 12 in 
favor, none against, and 1 abstention. 
Dr. Walters said the final issue was the relevance of the Milan 
data as being preclinical data for the proposed protocol. Dr. 
McKay asked Dr. Anderson to address the issue of why the 
subcommittee should accept these data as providing the necessary 
information required by the "Points to Consider." 
Dr. Anderson said the purpose of Dr. Bordignon's presentation was 
to provide additional evidence that the mechanism suggested as 
the scientific basis for the protocol was correct and could work, 
i.e., that genetically corrected T cells could be more effective 
in the patient than simply the reduction of serum deoxyadenosine 
by PEG-ADA alone. He said he thought the data convincingly show 
this conclusion to be true. Further, he added that this was not 
just in vitro data, or non-human primate data, but a specific 
case of a patient's cells being corrected in a manner that 
supports the basic scientific hypothesis of the protocol. He 
said he felt it satisfied all the criteria of the "Points to 
Consider. " 
Dr. R. Murray moved that: 
"The protocol be accepted with the proviso that 
intraperitoneal therapy not be used at this time." 
Dr. Childress seconded the motion. 
Dr. Walters asked if, by saying "not be used at this time," Dr. 
Murray was referring to the motion of Dr. Leventhal made earlier. 
He said this was the case. 
Dr. Parkman said there were two issues he wished to see 
discussed: (1) number of patients to be treated, and (2) updates 
on an annual basis to the RAC. Dr. Anderson said there would be 
semi-annual reports to both the HGTS and the RAC on all patients 
being treated. 
Dr. Epstein said he favored the motion and said that in light of 
the history of the proposal and the scrutiny with which it had 
been reviewed, that the only way to finally test the hypothesis 
was to go ahead with the protocol. 
There being no further discussion on the motion. Dr. Walters put 
the motion to a vote. The motion passed by a vote of 12 in 
favor, 1 against, and no abstentions. 
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Recombinant DNA Research, Volume 14 
