Recombinant DNA Advisory Committee - 12/3-4/92 
Ms. Meyers asked for clarification regarding the investigator's obligation to obtain RAC 
review. Dr. Murray explained that research conducted at an NIH sponsored institution is 
subject to the NIH Guidelines. Therefore, if an investigator does not receive NIH 
funding, the RAC has no purview over the proposed experiment. For the record, Dr. 
Murray reminded the RAC that they have reviewed a number of protocols that were 
submitted voluntarily by non-NIH funded institutions. Dr. Murray said that she is not 
aware if the requesting institution receives NIH funding. 
Dr. Ivor Royston, Scientific Director of the San Diego Regional Cancer Center, 
responded to the RAC members' comments. The San Diego Cancer Center is an NIH 
grantee institution; therefore, this research is subject to the NIH Guidelines and RAC 
review. He noted that he had submitted a human gene therapy protocol to the HGTS 
for review in November 1991. The original protocol involving cytokine gene therapy 
with fibroblasts was deferred by the HGTS. He said that he will return with a formal 
request in 1993. 
Dr. Royston explained that this compassionate plea request is for an individual patient 
who is the wife of the chairman of the board of the San Diego Cancer Center. 
Therefore, the patient is aware of the research that is being conducted and has asked the 
investigators if gene therapy could provide any therapeutic benefit. The patient has a 
Stage IV glioblastoma. This brain tumor is incurable. The patient's cells have been 
established in culture and have been transduced with the IL-2 gene. 
Dr. Royston stated that prior to the patient's request for gene therapy, brain tumor 
research was never contemplated. The patient's cells have been transduced with a vector 
that has not been approved by the RAC and a vector from Dr. Bemd Gansbacher that 
has received RAC approval. Dr. Royston stated that the FDA has been very responsive 
to his request, but the NIH does not have a similar mechanism for providing guidance 
regarding the treatment of individual patients who could die prior to the next regularly 
scheduled RAC meeting. Dr. Royston said that the recourse for proceeding without the 
NIH Director's approval could be the withdrawal of all NIH grants from the San Diego 
Cancer Center. He stated that he did not want to see this happen. If FDA approval is 
not received, then he would not seek RAC approval. However, if the FDA approves the 
single patient IND exemption, he would request that someone from the RAC or NIH 
authorize the treatment of this individual. 
Ms. Meyers stated that there are approximately 20,000 to 30,000 glioblastoma patients in 
the U.S. What do you say to these individuals? Dr. Royston stated that he is not 
optimistic that this therapy will work for this patient. He is merely a scientist who has 
been requested to provide this therapy. This patient has presented her case strongly to 
numerous doctors throughout this country and to Federal government officials. Dr. 
Royston said that he encouraged the patient to enroll in Dr. Edward Oldfield's NIH 
Recombinant DNA Research, Volume 16 
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