July 30, 1990, Human Gene Therapy Subcommittee 
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. 
Dr. Walters said he wished to continue with the next agenda item before taking a 
luncheon recess and called on Dr. Mclvor to begin the discussion. 
Recombinant DNA Research, Volume 14 
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