Recombinant DNA Advisory Committee - 06/7-8/93 
"III-A-4. Deliberate transfer of recombinant DNA or DNA or RNA derived from 
recombinant DNA into one or more human subjects [21]..." 
The term "subject" and "subjects" will be changed throughout the Points to 
Consider and the NIH Guidelines to "one or more human subjects." 
Committee Motion 
A motion was made by Dr. Motulsky and seconded by Ms. Meyers to accept the 
proposed amendment. The motion passed by a vote of 20 in favor, 0 opposed, and no 
abstentions. 
XVIII. DISCUSSION REGARDING INITIATION OF NIH-APPROVED HUMAN GENE 
TRANSFER PROTOCOLS AT SATELLITE INSTITUTIONS 
The RAC initiated discussion regarding the possible format that would be employed for 
the review and approval of human gene transfer protocols conducted at satellite 
institutions. The RAC noted several issues that would have to be examined in further 
detail, i.e., uniform Informed Consent documents, quality control, and the cell 
transduction process, such as in vivo versus ex vivo, if transduction is performed by the 
sponsor or at a satellite site, etc. The RAC recommended that this issue should be 
examined in further detail and discussed as a future agenda item. 
XIX. ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING A HUMAN 
GENE THERAPY PROTOCOL ENTITLED: GENE THERAPY FOR THE 
TREATMENT OF RECURRENT PEDIATRIC MALIGNANT ASTROCYTOMAS WITH 
IN VIVO TUMOR TRANSDUCTION WITH THE HERPES SIMPLEX THYMIDINE 
KINASE GENE/ DRS. RAFFEL AND CULVER 
Review-Dr. Hirano 
Dr. Walters called on Dr. Hirano to present her primary review of the protocol 
submitted by Dr. Corey Raffel of the Childrens Hospital of Los Angeles, Los Angeles, 
California, and by Dr. Kenneth Culver of Iowa Methodist Medical Center, Des Moines, 
Iowa. This protocol is very similar to two protocols by Drs. Oldfield and Culver 
previously approved by the RAC. The approach is to use the murine vector producer 
cell line (PA317) to deliver the Herpes simplex thymidine kinase (HS-tk) by means of a 
retrovirus vector. Tumor cells that express the HS-tk gene will be killed by 
administration of the antiviral drug, ganciclovir. The major difference between this 
protocol and the other two previously approved protocols is that the proposed study will 
involve children, 2-18 years of age, with malignant brain tumors. Dr. Hirano asked the 
Pis to clarify several questions: (1) What are the statistics on treatment responses? 
Recombinant DNA Research, Volume 17 
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