Recombinant DMA Advisory Committee - 5/30-31/91 
Mclvor said that Southern analysis is used for detecting genetic polymorphisms in bone 
marrow engraftment and he did not know whether PCR had been used in this respect. He 
asked Dr. Ledley to comment on this. 
In summary, Dr. Mclvor said the major reservation of the subcommittee was that there 
needed to be a feasibility assessment and that he felt this had been accomplished now by 
these investigators. 
Dr. Bourquin said he thought the investigators should be complimented on the thoroughness 
with which they addressed all the issues brought before them. He said that essentially he felt 
the comment could be lumped into two questions: 
1. Will it work? and, 
2. Is it necessary? 
He noted that both of these questions were discussed during the presentation by Dr. Ledley 
and his coworkers. The first question was addressed in terms of the genetic marker and the 
sensitivity with which valid results are expected. The second question was also addressed by 
a sentence from the materials provided by Dr. Ledley: 
"The use of the genetic marker appears to add minimal, if any, risk to the patient while 
greatly enhancing the probability of obtaining a meaningful result and gaining 
knowledge of value from the experiments." 
Dr. Bourquin said other issues included in the subcommittee's review included the ability to 
pay and how patients were selected. He noted the investigators had stated that only patient 
who were financially eligible for organ transplants could enroll in this program. However, 
they had now amended this slightly and provided the following statement: 
"But to avoid the opposite problem of denying treatment to those financially unable, 
the investigators are working very hard to obtain additional funding through Medicaid 
to support these patients as well." 
Dr. Bourquin said he felt that now, in light of this, that the program was open to anyone who 
meets other standard clinical criteria. He noted the investigators had changed the consent 
form and provided evidence that their informed consent program has demonstrated a 
significant increase in knowledge of the procedures following a review of the materials 
provided to the patients and that it was apparent that this program was providing a good 
information transfer. 
Mr. Barton said that a central point to be considered in this protocol was that it was an 
experiment in children and that he felt the committee needed to be very careful about the 
degree of risk which people were being asked to undertake for altruistic purposes. He also 
said that he felt that if this protocol were approved that the committee would, in essence, be 
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