PATIENT INFORMATION SUMMARY 
Project Title: Gene Therapy for the Treatment of Malignant Brain Tumors With In Vivo 
Tumor Transduction With the Herpes Simplex Thymidine Kinase 
Gene/Ganciclovir System 
Investigator: John C. VanGilder, M.D. 
Doctor has explained to you that this is a study to evaluate the effectiveness of 
genetic therapy for treatment of your malignant brain tumor. 
There is no effective treatment for malignant brain tumors which recur following surgery, radiation 
therapy, and/or chemotherapy. Therefore, a new experimental approach has been developed for the 
treatment of recurrent brain tumors that takes advantage of knowledge gained in the field of genetics 
and gene therapy. 
A virus that causes mouth sores and other types of infection is called herpes simplex. It can be treated 
with a drug called Cytovene. Herpes Simplex is killed by Cytovene because the virus contains a specific 
gene called a herpes/thymidine kinase gene (TK gene). The TK gene has been isolated in the laboratory 
and in experiments has been injected into brain tumors in rats. This causes the brain tumor cells 
containing the TK gene to be killed when the rats are treated with Cytovene. In some of the rats treated 
this way, the brain tumors disappeared. 
You are invited to participate in this study to gain information about whether or not this new approach is 
effective in treating human brain tumors. Although the findings in animals are encouraging, you will be 
one of the first humans to receive this investigational treatment for brain tumors. While we hope to gain 
information on the usefulness of this approach in humans, we cannot know whether your tumor will get 
smaller as a result of this treatment. It is possible that this treatment could make you worse. 
What is gene therapy for brain tumors? 
We will attempt to change the genetic material in your brain tumor cells by inserting the TK gene into the 
tumor cells. The way we will transport the TK gene into the tumor cells is to use a "vehicle" to carry 
the TK gene into the cells. An example is that if you want to move a passenger you need to put him in 
a car. The vehicle (or car) in this case is a different virus - a retrovirus that is found in mice - that has 
been inactivated so that it cannot cause disease. We call this vehicle a "vector". We insert the TK gene 
(the passenger) into the vector and the vector carries the gene into your brain tumor cells. The 
vector/gene combination is produced by "vector/producer" cells (little factories that produce the cars 
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