Human Gene Therapy Subcommittee - 7/30/90 
determine this on his own after reviewing the data. 
Dr. Miller said that the focus in FDA Phase I drug trials is 
threefold: the primary concern is safety to patients; secondly, 
data must be gathered on the pharmacokinetics and 
pharmacodynamics of the drug being tested; and thirdly, there 
must be a likelihood of efficacy in the patient population being 
tested. He urged that this protocol not be held to a different 
standard from routine drug trials. 
Dr. Parkman said that the investigators were putting this forward 
as a Phase I /Phase II trial. Because the patients are children, 
it is not deemed proper to do a toxicity study without looking 
for efficacy. Furthermore, he said the "Points to Consider" 
document does address the issue of efficacy. Dr. Miller said he 
understood this, but that looking for efficacy was an attempt to 
get the maximum information possible from the study. 
Dr. Leventhal said she felt the study was inevitably a 
Phase I /Phase II study since no normal controls were being used 
in the study. However, she agreed with Dr. Miller that the major 
concern is that the patients are not subjected to a procedure 
that has no rationale. It is important to establish that the 
administered cells are working and that they are safe to use in 
the patients. She said she felt the Phase II component is 
implicit in the study by the patient eligibility criteria, but 
that the design is not strictly that of a Phase II study. 
Dr. Walters called on Dr. Claudio Bordignon to present the data 
from his experiments in Milan. Dr. Anderson voiced his 
appreciation that Dr. Bordignon had come from Italy to provide 
assistance in this review. 
Dr. Bordignon said that he was not a close collaborator in this 
study and that the experiments were not done with a view toward 
answering some of the questions raised during the review of the 
gene therapy protocol. He expressed concern over his ability to 
publish these data but said he had confidence in the fairness of 
the system. He noted that in his country, "a process like this 
would be unthinkable." He said he could simply go ahead and do 
gene therapy at any time without having the data reviewed. He 
noted that at this stage he did not feel he was ready to do a 
human gene therapy experiment. He also apologized for the 
limited information contained in the initial FAX, but noted that 
it was compiled quickly because he was traveling to Japan at the 
time. 
Dr. Bordignon prefaced his remarks about the experiments by 
Recombinant DNA Research, Volume 14 
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