Human Gene Therapy Subcommittee - 11/30/90 
2. That the investigators would develop an assent form for children over 7 
years of age; 
3. That more detailed information will be supplied as to how the 
transduction will be undertaken that includes: numbers of cells, amount 
of virus, conditions under which transduction will be undertaken, growth 
factors, etc., 
4. That more details of the molecular analyses be included in the protocol 
with identification of AML blast colonies; and 
5. That the promise of confidentiality and protection from the press will be 
included in the informed consent document. 
There being no further discussion on the motion. Dr. Walters called for a vote. The 
motion passed unanimously by a vote of 10 in favor, 0 opposed, and 1 abstention. 
IX. PROPOSED ADDITION TO APPENDIX D OF THE NIH GUIDELINES 
REGARDING HUMAN GENE TRANSFER PROTOCOL ENTITLED: A PHASE II 
TRIAL OF HIGH-DOSE CARBOPLATIN AND ETOPOSIDE WITH 
AUTOLOGOUS MARROW SUPPORT FOR TREATMENT OF 
RELAPSE/REFRACTORY NEUROBLASTOMA WITHOUT APPARENT BONE 
MARROW INVOLVEMENT: USE OF MARKER GENES TO INVESTIGATE THE 
BIOLOGY OF MARROW RECONSTITUTION AND THE MECHANISM OF 
RELAPSE 
Dr. Walters called on Dr. Erickson to begin the discussion on the next protocol. Dr. 
Erickson said that this protocol was very similar to the previous protocol of Dr. 
Brenner's, and that the major difference was that these were patients with 
neuroblastoma being treated with BMT. Since neuroblastoma cells can persist in the 
marrow, the main question is much the same as in the previous protocol--to what 
extent are these cells contributing to relapse? The studies of transduction of bone 
marrow cells and the data provided were the same as the previous protocol. 
Dr. Erickson noted that one reason for approving the previous protocol was that it 
was possible to identify tumor cells by PCR. Further, these cells can be cultured and 
identified as being different from normal bone marrow cells. This protocol has the 
same rationale. Much of the supporting data given in this particular proposal was 
conducted on AML cells, and data should be presented today by the investigators to 
substantiate that the same can be proven using neuroblastoma cell lines. 
Recombinant DNA Research, Volume 14 
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