Recombinant DNA Advisory Committee - 06/7-8/93 
injured as a direct result of their participation in research. The Informed Consent 
document informs patients that further information about their rights is available from 
the Hospital Risk Management Office. 
Dr. Walters stated that compensation for research injuries is a generic issue that the 
RAC has discussed previously. On January 6, 1993, the RAC sent a letter addressing 
this issue to the NIH Director, Dr. Bernadine Healy. To date, no specific response to 
this letter has been received. There was a lengthy discussion on this issue, and Dr. 
Walters asked Dr. Parkman to draft a follow-up letter to the NIH Director that could be 
circulated for later discussion. 
Committee Motion 
A motion was made by Dr. Haselkom and seconded by Dr. Parkman to approve the 
protocol. The motion passed by a vote of 20 in favor, 0 opposed, and no abstentions. 
VI. ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING A HUMAN 
GENE THERAPY PROTOCOL ENTITLED: USE OF MODIFIED RETROVIRUSES TO 
INTRODUCE CHEMOTHERAPY RESISTANCE SEQUENCES INTO NORMAL 
HEMATOPOIETIC CELLS FOR CHEMOPROTECTION DURING THE THERAPY OF 
OVARIAN CANCER: A PILOT TRIAL/ DRS. DEISSEROTH, KAVANAGH, 
CHAMPLIN 
Review-Dr. Leventhal 
Dr. Walters called on Dr. Leventhal to present her primary review of the protocol 
resubmitted by Drs. Albert B. Deisseroth, John Kavanagh, and Richard Champlin of the 
University of Texas M.D. Anderson Cancer Center, Houston, Texas. This protocol was 
deferred at the March 1993 RAC meeting. Dr. Leventhal explained that the 
investigators plan to introduce the cDNA of the multi-drug resistance gene (MDR-1) 
into normal hematopoietic early progenitor CD34( + ) cells in patients with advanced 
ovarian cancer in an attempt to determine toxicity and the effect of modifying these 
normal stem cells. Responding to a question on the transduction procedure, the 
investigators have provided data demonstrating that human CD34( + ) cells can be 
isolated and transduced, and have demonstrated adequate expression of MDR-1. The 
desired outcome will be that each successive incremental dose of Taxol will result in an 
increased percentage of MDR-positive bone marrow cells. She was concerned about the 
actual benefit these patients will derive from introducing the MDR-1 gene into their 
bone marrow stem cells. Adverse side effects of Taxol, such as neurotoxicity, will not be 
prevented by MDR-1 transduction; therefore, hematologic protection is the only possible 
beneficial outcome of this study. Dr. Leventhal stated that she is not completely satisfied 
that the experimental design of this protocol will provide a definitive answer about 
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