Recombinant DNA Advisory Committee - 12/3-4/92 
Dr. Walters said that he was in complete agreement with the statements made by Drs. 
D. Miller and Leventhal. When a committee is charged with data monitoring, it is 
extremely important that it not draw premature conclusions. These human gene therapy 
studies have been performed on a very limited number of patients. 
Dr. Leventhal stated that the important issue is whether there are implementation 
problems with the protocol. If an approved vector is not working under any 
circumstance, then the investigator should not be allowed to treat another 10 patients. 
These are the types of issues that the RAC should be considering. 
Dr. Leventhal asked if she had the authorization of the committee members to pursue 
specific questions with investigators. Specifically, could she contact Dr. Rosenberg about 
the tumor necrosis factor/tumor infiltrating lymphocyte trial. More data needs to be 
provided regarding the fate of the transduced cells after they were readministered to the 
patients. The request for additional information would be limited to data regarding the 
efficacy of the gene transfer procedure. The RAC members agreed that Dr. Leventhal 
should pursue necessary requests for additional information. 
Dr. Leventhal proposed that investigators will be requested to submit a copy of the 
patient death reports that are sent to the FDA. The RAC cannot request this 
information from the FDA, but it can request a copy from the investigator. She 
proposed to develop a data reporting format that the investigator will be required to 
complete for each protocol. The Points to Consider requires that investigators should file 
a report every 6 months; however, she suggested that once a year will probably be often 
enough if patient enrollment is small. Perhaps investigators should be required to file a 
report once a year or after every 10 patients, whichever comes first. Any published 
results of trials that are received by the Office of Recombinant DNA Activities (ORDA) 
should be circulated to the entire RAC. 
X AMENDMENT TO THE POINTS TO CONSIDER REGARDING SAFETY OF 
DELIVERY /EXPRESSION SYSTEMS AND REPORT OF MURINE REPLICATION- 
COMPETENT RETROVIRUS (RCR) ASSAYS/DR. ANDERSON 
Review-Dr. D. Miller 
Dr. Murray called on Dr. D. Miller to give his primary review of the report submitted by 
Dr. Anderson regarding RCR data. Dr. D. Miller stated that the report is a very 
comprehensive summary. However, the RAC should recognize the fact that a procedure 
must be developed for the standard testing of RCR that will apply universally to 
retrovirus vectors. It is clear that these standards cannot be extrapolated from the 
history of the production of a particular vector by one company. The development of 
standards for the testing of retrovirus vector preparations should be balanced between 
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