Gene Therapy for Meningeal Carcinomatosis 
(301) 590 2626 (available 24 hours a day, recorder after working hours). 
The sponsor, Genetic Therapy, Inc. takes the responsibility of informing and preparing 
reports for the Recombinant Advisory Committee (RAC) through the NIH Office of Recombinant 
DNA Activities (ORDA) and to the FDA. 
9.0 Evaluation of Respo nse/Endpoints 
9.1 Study Endpoints : 
9.1 1 Safety Endpoints: Development of grade 4 toxicity not responsive to therapy in 3 
patients in phases A-C and 4 consecutive patients in phase D are the endpoints for each specific 
(and subsequent) phases. 
9.12 Efficacy Endpoints: Clinical, radiographic, and cytologic evidence for eradication of the 
leptomeningeal disease within 3 months of follow up. (See Appendix VIII for details) 
9.2 Response Criteria : 
Patients will be evaluated repeatedly throughout the study. Their neurological exam will be 
recorded twice daily during the initial 48 hours after both the injection of producer cells and 
infusion of GCV. Additionally, their neurological exam will be documented when they are 
discharged. Responses will be based on the following grading system: 
Level I Complete Response: No evidence of disease by cytology/tumor 
markers, MRI, and clinical exam. 
Level II Partial Response: A decrease in malignant cells remaining in the 
CSF by 50% and/or an improvement in neurological exam, and/or 
change in the MRI evidence of leptomeningeal involvement or 
tumor markers. 
Level m Stable disease and parameters of LMC (cytology/tumor markers, 
MRI, clinical exam). 
Level IV 
Progression of Disease 
9.3 Required Documentation : 
Gadolinium-enhanced MRI scans of the brain and spinal cord will be performed at 2, 6, 
and 12 weeks after therapy. Ventricular and spinal CSF samples will be obtained at 2, 6, and 12 
weeks after treatment. These samples will be assessed for cytology and tumor markers. 
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