Non-Technical Abstract 
A ONE-PAGE DESCRIPTION OF THE PROPOSED EXPERIMENT IN 
NON-TECHNICAL LANGUAGE : 
In the treatment of patients with metastatic cancer, i.e., cancer 
that has spread beyond the primary site involved, the 
administration of several anti-cancer drugs is usually required. 
High doses of these drugs is so damaging to normal human bone 
marrow cells that removal of some bone marrow from the patient 
prior to being given the drug is routinely performed; this marrow 
is then put back into the patient after the effects of the 
chemotherapy are gone. 
The goal of these studies is to transfer a gene into the 
patient's normal bone marrow cells when they are removed (bone 
marrow transplantation) as ordinarily occurs in treatment to make 
these cells resistant to the effects of these drugs. The result 
of this procedure will be the creation of normal bone marrow 
cells more resistant to some types of routinely used anticancer 
drugs. Bone marrow expressing this new added gene should be 
resistant to the killing effect of these drugs. The patient can, 
therefore, produce more normal levels of red and white blood 
cells and platelets during chemotherapy. This may confer a 
significant advantage to patients with cancer that has spread, 
especially when given subsequent rounds of chemotherapy. 
Theoretically, the more chemotherapy the patient receives after 
getting the drug-resistant gene the more resistant bone marrow 
cells will remain in the patient since bone marrow cells that do 
not express the added gene will be preferentially killed. This 
treatment, if successful, could allow patients with advanced 
cancer to receive high doses of potentially effective drugs, or 
allow patients to be treated with drugs who could not otherwise 
be treated because of the side effect to their bone marrow. 
The potential harm of the use of this "gene therapy" is minimal. 
Animal studies and other laboratory tests have shown this type of 
gene therapy to be safe. Thus, this gene therapy treatment may 
benefit patients with cancer that has spread but not involving 
the bone marrow. We are trying this therapy first in patients 
with advanced breast cancer in the hope that we can decrease the 
damage done by drug treatment to the bone marrow, and allow more 
and higher doses of drugs to be given to these patients. At 
future times, this treatment, if successful, could lead to higher 
doses of chemotherapy to be given earlier, and a greater chance 
of remission and possible cure. 
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Recombinant DNA Research, Volume 18 
