Informed Consent 
2 ) CONSENT FORM 
The purpose of this consent form is to provide you with 
the information you need to consider in deciding whether to 
participate in this research study. 
STUDY TITLE: ADDITION OF HUMAN MDR GENES TO BONE MARROW CELLS TO 
PROVIDE RESISTANCE TO CHEMOTHERAPY ADMINISTERED TO PATIENTS WITH 
ADVANCED CANCER. 
IRB STUDY NUMBER: 
You are invited to participate in a research study evaluating the 
effect of transferring a specific human gene to your bone marrow 
cells. This gene, known as the Multiple Drug Resistance (MDR) Gene, 
induces cells to become resistant to certain chemotherapy agents 
such as taxol. The reason for asking you to participate in this 
study is that you have advanced cancer which has been treated with 
surgery, radiation and chemotherapy. Your doctor believes that this 
therapy is not sufficient to control your disease so he has 
referred you to Dr. Charles Hesdorffer to undergo a course of high 
dose chemotherapy followed by autologous bone marrow 
transplantation (ABMT) as a means of again controlling your 
disease. The introduction and expression of the MDR gene in your 
bone marrow cells may prevent drugs you recieve later on from 
injuring your marrow. 
Once you undergo a bone marrow harvest for a standard course of 
high dose chemotherapy you may elect to participate in this study; 
no additional tests or treatment would be required for you at that 
time to do so. If you agree to participate, approximately a third 
of the marrow harvested will have the MDR gene inserted using a 
technique known as retroviral gene transfer. This technique 
involves taking a mouse retrovirus, stripping this organism of its 
capacity to reproduce, and attaching to it the human MDR gene. The 
retrovirus serves as a carrier only. This process has been 
evaluated in animals as well as in humans and has been shown to be 
safe. On completion of the high dose chemotherapy you will have 
both "normal" marrow as well as the "gene inserted" marrow 
reinfused. This will avoid the risk of the marrow transplantation 
being inadequate for your treatment. 
Your participation in this study involves the following risks: 
1. Although extremely small, it is conceivable that the defective 
retroviruses we use to transfer new genes to your bone marrow cells 
may result in harm. In the worst possible case, which has never 
been reported in humans, this might result in the development of a 
new cancer. We have done extensive studies in animals that show 
that the defective retroviruses we use in these studies are 
extremely unlikely to cause this problem. In addition, it is 
possible that this treatment may affect cancer cells in your bone 
marrow thus making them more resistant to certain drugs. However, 
we will be isolating specific marrow progenitor cells in your bone 
marrow (CD34 positive cells) prior to introducing the MDR gene in 
Recombinant DNA Research, Volume 18 
[119] 
