APPENDIX C 
ABSTRACT OF PROTOCOL 
This protocol is a study of patients with advanced neuroblastoma who have 
failed conventional therapy. In an attempt to increase the patient's immune response 
to the tumor, the interleukin-2 (IL-2) gene will be introduced into a tumor cell line 
established from the patient. These gene-modified autologous tumor cells will then 
be injected subcutaneously into the patient. To determine the toxicity associated with 
returning transduced tumor cells to the patient a Phase I dose escalation study is 
proposed. The patient's immune system will be studied both at the local site(s) of 
tumor injection as well as in the peripheral blood. These studies will look for evidence 
that the presence of IL-2 secreting tumor cells has changed the patient's immune 
responses, in particular their response to their tumor cells. The patient will also be 
assessed clinically to see if this intervention has affected the patient's disease course. 
Animal models have shown both the injection of gene modified tumor cells and 
the derived stimulated lymphocytes to have important antitumor effects. In vitro 
experiments using patient lymphocytes and patient transduced tumor cells has shown 
increased antitumor activity of the lymphocytes related to the gene transfer into tumor 
cells. The patients will be evaluated for antitumor effects engendered by the injection 
of the gene modified tumor cells. The injection of gene modified tumor cells may 
serve to "immunize" the patient to their tumor and may be amenable to use in a wide 
variety of tumor types, especially those that are poorly immunogenic. This protocol 
may increase the effectiveness of anticancer treatments available to patients with 
neuroblastoma or other forms of cancers. 
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