C. HESDORFFER 4/93 
The goal of initial experiments will be to look for any side 
effects, to quantitate the number of bone marrow stem cells 
transduced with the MDR gene both before and after MDR gene 
transfer, and to evaluate MDR expression and whether selection of 
MDR-transduced cells occurs after subsequent exposure to the 
taxol . 
The patients used will have no preferential ethnic background, 
and it is anticipated that in the first studies only adults will 
be used. In ABMT at CPMC to date, the demography of ABMT 
patients reflects that of the patients seen at CPMC in general. 
The bone marrows harvested since June 1990 at CPMC are comprised 
of 67% female, 10% black, and 10% hispanic. The bone marrow 
transplants done since July 1990 are comprised of 80% women, 20% 
black, and 12% hispanic. The preponderance of women is due to 
transplanting patients with breast and ovarian carcinomas which 
comprise 40% of the transplants done at CPMC. The New York City 
population in general is -10% hispanic and 25-30% black. The 
health status of these patients will depend on their underlying 
oncology condition and it is anticipated that transduction of the 
bone marrow of these patients will have no detrimental effect on 
their health status. 
B. Sources of Research Material : 
As indicated in #1 above, patients' bone marrow cells 
will be analyzed for the presence and expression of the MDR gene 
at various times subsequent to the initial reinfusion of marrow. 
The additional number of bone marrow aspirations, if any, 
required of these patients will be low, perhaps 1-3. Primarily, 
peripheral blood samples will be used to monitor the survival of 
transduced cells and MDR gene expression; blood will be obtained 
from these patients periodically and examined by PCR for the 
presence and expression of the MDR gene and and by FACS analysis 
for MDR protein and for intact retrovirus. The peripheral blood 
sampling may or may not require venipunctures in addition to 
those ordinarily being performed in the course of monitoring 
these patients. 
C . Recruitment of Subjects and the Consent Procedures to 
be Followed: 
As described above, initially subjects undergoing ABMT 
in association with intensive chemotherapy for advanced cancer 
not involving the bone marrow will be used. The investigational 
aspects of the gene transfer into bone marrow cells will be 
completely explained to the patients as a procedure that; 
1) Initially offers them no significant advantage; 
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