Informed Conseni 
Medical Record: Consent to Participate in a Research Study 
Childrens Hospital Los Angeles 
Study Number: 
Principal Investigator: Corev Raffel, M.D.. Ph.D. 
CONSENT TO PARTICIPATE IN A RESEARCH STUDY: GENE THERAPY FOR THE 
TREATMENT OF RECURRENT PEDIATRIC MALIGNANT ASTROCYTOMAS WITH IN VIVO 
TUMOR TRANSDUCTION WITH THE HERPES SIMPLEX THYMIDINE KINASE 
GENE/ GANCICLOVIR SYSTEM 
FEDERAL REGULATIONS REQUIRE WRITTEN INFORMED CONSENT FROM PARTICIPANTS 
IN A RESEARCH STUDY. THE NATURE AND RISKS OF PARTICIPATION MUST BE 
EXPLAINED. INDIVIDUALS MUST DECIDE TO PARTICIPATE OR NOT TO PARTICIPATE 
IN A FREE AND INFORMED MANNER. YOU ARE ASKED TO READ THE FOLLOWING 
MATERIAL TO ENSURE THAT YOU ARE INFORMED OF THE NATURE OF THE RESEARCH 
STUDY. THE REQUIREMENTS FOR PARTICIPATION IN THE STUDY ARE DESCRIBED. 
SIGNING THIS FORM WILL INDICATE THAT YOU HAVE BEEN INFORMED AND THAT YOU 
GIVE YOUR CONSENT. 
BACKGROUND AND PURPOSE 
Your child has a diagnosis of recurrent malignant astrocytoma and, 
therefore, is eligible to participate in this study program. 
There is no effective treatment for malignant brain tumors in 
children that recur following surgery, radiation therapy and/or 
chemotherapy. Therefore, we have developed a new experimental approach 
for the treatment of recurrent brain tumors that takes advantage of 
knowledge gained in the fields of genetics and gene therapy. A virus 
that causes mouth sores and other types of infections is called Herpes 
simplex. It can be treated with a drug called Cytovene. The Herpes 
simplex virus is killed by Cytovene because the virus contains a 
specific gene called the 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. Because the TK gene is delivered to the brain tumor using 
a method that will only effect tumor cells and not normal brain cells, 
only tumor cells should be killed by Cytovene. The purpose of this 
study is to gain information about whether or not this new approach is 
effective in treating pediatric brain tumors. Although the findings in 
animals are encouraging, your child 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 the tumor in your child will get smaller as a result 
of this treatment. It is possible that this treatment could make your 
child worse. 
What is gene therapy for brain tumors? 
We will attempt to change the genetic material of your child's 
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