University of California, San Francisco 
Informed Consent 
Consent To Be A Research Subject 
Gene Therapy of Primary and Metastatic Malignant Tumors of the Liver Using ACN53 Via 
Hepatic Artery Infusion: A Phase I Study - UCSF 45294 
PURPOSE AND BACKGROUND 
Dr. Alan Venook, Dr. Robert Warren, Dr. Thierry Jahan, and Dr. Ernest Ring at the 
University of California, San Francisco (UCSF) Medical Center, are interested in finding 
out whether patients with malignant tumors or cancers in the Ever, including Ever cancer 
and colon and rectum cancer which has spread to the Ever, can be safely treated with a new 
and investigational drug called ACN53. This drug has been designed to provide gene 
therapy with the p53 gene. Gene therapy refers to a very new form of therapy in which 
genes are introduced into ceEs to correct or modify their properties. In the case of ACN53, 
the gene to be introduced is caEed p53, and the method of introduction or deEvery is with a 
modified virus. The modified virus has been constructed from adenovirus, a virus which 
sometimes causes the common cold. Both of these components, the p53 gene and the 
modified adenovirus, comprise the invesrigational drug ACN53. ACN53 is being tested to 
see if it can safely and efficiently introduce a normal copy of the p53 gene into cancers 
containing an altered copy or copies of p53. The p53 gene is being used for a number of 
reasons. The p53 gene is caEed a tumor suppressor gene because it is believed to prevent 
normal ceEs from developing into cancer ceEs. When the p53 gene becomes altered (such 
as by mutation), this helps cancers to form. The p53 gene is frequently altered in Ever 
cancer and in colon and rectum cancer. In scientific experiments using ceEs in the test tube 
and in animals, introducing a normal copy of the p53 gene into cancer ceEs which have 
altered p53 can cause them to grow more slowly, stop growing, or die. 
I am being asked to participate in this study because I have developed cancer in my Ever 
which is not curable with existing therapies. 
The purposes of this study are: 
1 . To assess the safety of gene therapy with ACN53 in patients with Ever 
tumors when a single dose is given into the Ever by way of the hepadc 
artery. (In other words, how safe is ACN53 when given to the Ever and 
what are its side effects?) 
2 . To assess the efficiency of gene therapy with ACN53 against Ever tumors. 
To do this, samples of the tumor wiE be obtained for analysis after the 
treatment. (In other words, how weE does this gene therapy concept work 
in tumor cells?). 
3 . To assess the effect that the dose of ACN53 used has on safety and 
efficiency of gene therapy. Part of this objective wiE involve measuring the 
amount of ACN53 in the body at various points of time. (In other words, 
what is the highest dose of ACN53 that can be safely given?). 
Recombinant DNA Research, Volume 20 
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