Recombinant DNA Advisory Committee - 01/14/93 
In the interim, Dr. Royston said that he was able to transduce and grow this particular 
patient's glioblastoma tumor cells in culture. This procedure is no mean feat. The 
question was, can we help this patient? This patient is an individual with a Stage IV 
glioblastoma who has failed all conventional therapies. Therefore, the only therapy 
available to this patient is experimental therapy. It is right that she receive this 
treatment. This individual is a thriving woman who has a life expectancy of 1 to 2 
months, who otherwise functions normally. 
Dr. Royston stated that he is a physician and a medical researcher. He receives NIH 
grants; therefore, he does not intend on evading the RAC. However, if it comes to 
choosing between a physician and a researcher, he is a physician first. He wants to give 
his patient the best available therapy. 
Dr. Royston explained that based on the scientific literature, there is a strong rationale 
for providing this patient with genetically modified, transduced glioblastoma cells. All of 
the additional data that the FDA requested was submitted. However, he is not prepared 
to present this additional data today. Dr. Royston said that he would present the 
scientific rationale for this study and all of the data submitted to the FDA at the March 
1-2, 1993, RAC meeting. Dr. Royston commended Dr. Healy for granting approval of 
this compassionate use request. 
Dr. Krogstad asked for clarification regarding the submission of additional material. 
Was additional material submitted to the FDA that was not submitted to the RAC? Dr. 
Royston acknowledged that this statement is correct. The following additional safety 
data was submitted to the FDA: (1) replication competent retrovirus data, (2) sterility 
data, (3) data on the transduced cell line, (4) data demonstrating the lack of 
contaminating helper virus, and (5) vector identity data. 
Dr. Krogstad inquired how interleukin-2 (IL-2) production in transduced cells compared 
with the experimental data that the RAC reviewed. Dr. Royston stated that the RAC 
did not have access to the experimental data. Dr. Royston said that the transduced 
glioblastoma cells secrete 30 units of IL-2 per 2 x 10 6 cells per 24 hours. 
Dr. Krogstad said that there is merit to having the RAC review the data on which this 
single case exemption was granted, even retrospectively, to satisfy the concerns that have 
arisen among the committee members. Dr. Royston explained that the RACs concerns 
have to be weighed against the needs of individuals. Dr. Krogstad said that he 
understands these considerations, because he has been a physician for over 20 years. Dr. 
Krogstad said that the patient's interests must be paramount; however, there is no excuse 
for overlooking the need to review these other issues. 
Dr. Royston said that he would provide the RAC with any data that it believes is 
Recombinant DNA Research, Volume 17 
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