Recombinant DNA Advisory Committee - 03/1-2/93 
6. What system was used to determine pre-clinical efficacy? 
7. What is the endpoint of the protocol? 
8. a. What assay was used to detect replication competent helper virus? 
b. Was replication competent helper virus detected? Yes No 
c. What is the level of sensitivity of the assay? (Attach documentation) 
9. a. What was the date of IRB approval? ________ (Attach copy of 
approval) 
b. What was the date of IBC approval? (Attach copy of 
approval) 
c. What was the date of IND submission to the FDA? 
10. a. If the FDA permits the protocol to proceed, is clinical grade material 
available? Yes No 
b. If clinical grade material is not available, on what date will it be available? 
XVII. DISCUSSION REGARDING THE HUMAN GENE TRANSFER PROTOCOL 
MATERIALS SUBMITTED FOR APPROVAL ON A COMPASSIONATE USE 
BASIS/Drs. Royston and Sobol 
Background 
In a letter dated December 7, 1992, Dr. Ivor Royston of the San Diego Regional Cancer 
Center, San Diego, California, requested compassionate plea approval for a human gene 
transfer protocol. The protocol was entitled: Phase I Study of Cytokine Therapy of 
Cancer ; Active Immunotherapy of Glioblastoma with Tumor Cells or Fibroblasts Genetically 
Modified to Secrete IL-2. On December 28, 1992, the NIH Director and the FDA 
granted approval of Dr. Royston's request on a compassionate use basis. 
On January 14, 1993, the NIH Director called a special meeting of the RAC to discuss 
expedited review procedures. During this meeting, Dr. Royston stated that he would 
provide the RAC with additional data that was supplied to the FDA Dr. Royston said 
that the FDA-submitted material included the following: (1) additional safety data on 
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