APPENDIX 5 
NON-TECHNICAL ABSTRACT 
Over the past several years, autologous stem cell 
transplantation has been used to treat many types of malignancies, 
including multiple myeloma, chronic myelogenous leukemia, and 
breast cancer. In this procedure, the patient is treated with 
standard chemotherapy until the tumor is in the best remission 
possible. Bone marrow and/or peripheral blood cells are collected 
and stored. The patient can then receive very intensive drug and 
radiation treatments aimed at destroying any remaining tumor. The 
patient's bone marrow function is also destroyed, but the 
previously collected bone marrow and/or peripheral blood cells can 
be infused back into the patient to "rescue" him or her and 
reconstitute bone marrow function. This procedure has been very 
promising, but a number of questions remain about the best way to 
perform it. It is not known if the transplanted cells simply 
provide a "bridge" of bone marrow function until stem cells 
remaining in the patient recover from the high-dose therapy. 
Treatment approaches to these three diseases are in development 
that involve the introduction of new genes into bone marrow or 
peripheral blood* cells to help the patient overcome the tumor, but 
for these treatments to work, the transplanted cells must survive 
in the patient for long periods. It is also unclear if tumor cells 
contaminating the harvested bone marrow and blood are responsible 
for relapse after the transplantation procedure. 
The aim of this protocol is to obtain information about 
autologous transplantation and about the feasibility of 
transferring genes to bone marrow and peripheral blood cells that 
could help other patients with these diseases in the future. We 
will use specially designed vectors to carry a marker gene into 30% 
of the harvested bone marrow and/or peripheral blood cells in 
patients undergoing autologous transplantation for multiple 
myeloma, chronic myelogenous leukemia, and breast cancer. The 
remaining 70% will be stored and frozen without gene marking, and 
will be enough cells to allow recovery after transplantation even 
if the gene marked cells are not given back. If marking is 
successful, it will allow us to trace these cells after 
transplantation, and learn more about the contribution of these 
cells to recovery and tumor relapse. It will be very important for 
future gene therapy treatments to learn if "stem cells", or cells 
in the marrow and peripheral blood that have the ability to produce 
daughter cells of all blood lineages for prolonged periods of time 
survive autologous transplantation and can be marked by this gene 
transfer technique. Gene marking is the only method currently 
available to distinguish cells originating from the harvested 
marrow or peripheral blood cells from cells remaining in the 
patient and surviving the high-dose therapy. 
Recombinant DNA Research, Volume 16 
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