Recombinant DNA Advisory Committee - 2/4/91 
And with that, Dr. McGarrity called on Dr. Walters. Dr. Walters said that Dr. Epstein 
had moved to approve this protocol and that Dr. Mclvor had seconded the motion and 
that five stipulations had been placed on the approval, two dealing with the consent form 
and assuring that the desirability of long-term follow-up was mentioned, as well as issues 
surrounding publicity and privacy issues. The investigators were also asked to prepare an 
assent form for children who were old enough to understand what was going on in the 
study. The final two points, raised by Dr. Mclvor, concerned the efficiency of the 
retrovirally-mediated gene transfer into human bone marrow cells and analysis of the 
origin and character of the regenerated tumor. Dr. Walters said the final vote to 
approve the protocol with the 5 stipulations was 10 in favor, 0 opposed, and 1 abstention. 
Dr. Walters reviewed the revised informed consent document and that the two points on 
long-term follow-up were addressed. The assent form was also included in the revised 
protocol. He believed the assent form needed to include the 14-year follow-up to more 
closely mirror the informed consent document, but otherwise it appeared the 
investigators had complied with the 5 stipulations, subject to Dr. Mclvor's critique on the 
issue of gene transfer into bone marrow. 
Dr. Geiduschek asked about the issue of what costs of this proposal would be borne by 
the patients. 
Dr. McGarrity called on Dr. Brenner to make a brief presentation. Dr. Brenner thought 
that Dr. Mclvor had summarized the aims of the study reasonably well. Dr. Brenner was 
concerned over whether the marked gene will enter the stem cells is not a question that 
can be answered definitively at the present time. There was no evidence that this would 
occur. As to the issue of whether the conditions of transduction used in the in vitro 
preclinical studies would match exactly the conditions in the patients, he said that they 
would. At present, they had not scaled this up to cell numbers representing full marrow 
conditions. If approval for the protocol was granted that they would perform a 
preliminary experiment on stored marrow to make sure that scale-up is possible. In all 
other respects, the methodology was exactly the same and there was no use of growth 
factors contemplated for the experiment. 
Dr. Brenner noted that in regards to the issue of the terms "extensively" and "strongly" in 
the informed consent document, the original wording had been "extensively." However, 
due to a rule that the informed consent document be worded at a fifth grade reading 
level, the word was changed via a computer program to concur with a fifth grade 
vocabulary. 
Dr. Brenner said that at St. Jude's Hospital all patients are cared for regardless of ability 
to pay and that all costs incurred, regardless of the manner in which they are incurred, 
are fully covered. Dr. Brenner also concurred with Dr. Walter's comment regarding 
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