Cover Sheet for Accelerated Review of a Human Gene Transfer Experiment 
A. Background 
Title: A Phase I Study of Vaccination With Autologous, Irradiated 
Melanoma Cells Engineered to Secrete Human Granulocyte- 
Macrophage Colony Stimulating Factor 
Principal Investigator: Glenn Dranoff, M. D. 
Participating Institutions: 
Dana-Farber Cancer Institute, Boston, MA 
Massachusetts General Hospital, Boston, MA 
Somatix Therapy Corporation, Alameda, CA 
B. Vector, Target Cell, and Transduction Procedures 
1. The vector is MFG-S. The gene is the human GM-CSF gene. 
2. The MFG-S vector containing the human GM-CSF gene has been 
NIH RAC reviewed and approved in protocol 9303-040 in March, 
1993 (Phase I Study of Non-Replicating Autologous Tumor Cell 
Therapy with and without GM-CSF Gene Transduction in Patients 
with Metastatic Renal Cell Carcinoma, Jonathan W. Simons, 
Principal Investigator). 
3. The vector and irradiated, transduced melanoma vaccine cells are 
supplied by Somatix Therapy Corporation under FDA approved 
IND BB5229 for Phase I study. 
4. The target cells we have transduced are autologous human 
melanoma cells in primary culture. 
5. Copy number ranges from 0.1 to 1.0 at Somatix Therapy Corporation 
in primary melanoma cells using a single transduction of a 
production lot of MFG-S-GM-CSF. 
6. The level of gene expression in the study is 40-1000 ng/10^ cells/24 
hours. 
7. Gene expression is measured by ELISA (copy number determined by 
Southern analysis). 
Recombinant DNA Research, Volume 20 
