8.2 Laboratory Measures of Response to Therapy 
Weekly samples of peripheral blood samples will be analyzed for the presence of tumor specific 
cytotoxic T-cells as described in Section 4.7 to obtain an assessment of biologic response and for 
correlation with clinical response. 
8.3 Safety Considerations 
The sample size requirement for this study is driven in part by the need to monitor for toxicity and 
unexpected effects. We will treat 12 patients with LD SCLC with 11-2 transfected tumor cells. This 
will provide an 80% probability of finding at least one episode of any adverse event that occurs with 
a true frequency of at least 13%, and a 90% chance if the true frequency is 17%. 
8.4 Accrual of Patients 
Using 11-2 transfected tumor cells, we will treat 12 patients with LD SCLC within two years. In 
order to provide 12 patients who are eligible and evaluable for gene therapy approximately 40 patients 
over two years will have to be entered on study because of the following considerations. 
The proportion of patients with limited disease SCLC: 
• who satisfy eligibility criteria, especially good performance status and absence of adverse 
prognostic factors is approximately 80%. 
• whose tumor cells can be established as cell lines in culture is approximately 70% (from Dr. 
Savaraj’s experience). 
• whose cell lines can be stably transfected with the gene for 11-2 production is approximately 90% 
(from Dr. Podack’s experience). 
• who will achieve a complete or good partial remission with chemotherapy is approximately 80%. 
• who will relapse and require therapy or for a variety of medical and non-medical reasons will 
become ineligible prior to gene therapy is approximately 25%. 
Assuming that these conservative estimates are all independent variables, then the proportion of 
entering patients who will be eligible for gene therapy is 0.8 x 0.7 x 0.9 x 0.8 x 0.75 = 0.30 Since 
only approximately 3/10 entering patients will undergo subsequent gene therapy, we will need to accrue 
40 patients for initial evaluation so that 12 will be available to receive gene therapy. At the University 
of Miami Medical Center, we care for approximately 60 patients annually who are newly diagnosed 
with SCLC. Approximately 1/3 (or 20) of these patients are LD stage. We believe our current 
population will provide an adequate number of patients to meet our accrual goals. 
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