Human Gene Therapy Subcommittee - 7/30/90 
3. That a stop criterion of two therapy-related deaths be 
inserted; 
4. That intraperitoneal (i.p.) infusions not be utilized 
without further approval by this committee; 
5. That proceeding to Phase 2B of this protocol would 
require approval by the IBC and IRB; 
6. That full data from the Milan experiments and any 
related data be provided for review of a working group 
of this subcommittee prior to a meeting of the RAC and 
that a formal review of those data be brought to the 
RAC when this protocol comes up for approval; 
7. That a final version of the inclusion/exclusion 
criteria reflect the parameters that were addressed in 
the subcommittee discussion, including age, length of 
time on PEG- ADA; and, 
8. That there be a specific protocol for the follow-up 
evaluation of the immunological and clinical status. 
Dr. Parkman noted that the revised protocol still included 
reference to the use of an i.p. route of administration. 
However, he felt this was a minor administrative oversight in 
revising the protocol. 
Dr. Parkman noted the investigators had supplied a summary sheet 
of preclinical trials showing that the insertion of the human ADA 
gene into the peripheral blood leukocytes of cells from ADA- 
deficient patients appeared to normalize their function in vivo. 
He said this was important because one of the issues in the 
"Points to Consider" is the matter of an appropriate animal 
model. He said he had several questions about these trials: 
1. The data appear to consist primarily of information 
that was presented at the UCLA meeting last winter. 
What new data does Dr. Bordignon have on experiments 
that have been done since February, 1990? Has the NIH 
group done similar experiments since June 1? 
2. Are the experiments described a single experiment with 
one patient, or are they multiple experiments with 
multiple patients? If it is a single experiment with 
one patient, have the investigators been able to 
reproduce the findings in a second set of experiments? 
If it is with multiple patients, what was the person- 
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