Recombinant DNA Advisory Committee - 12/2-3/93 
Dr. Parkman explained that he would not vote for disapproving this protocol. The RAC 
has approved other protocols with similar preclinical data. Since the investigators 
propose to use lethally irradiated cells, there is minimal risk associated with the 
experiment. The potential risk to patients is that other forms of therapy may be 
withheld from these patients. 
Dr. Haselkom stated that there are other forms of harm that could occur as a result of a 
patient's participation in this study such as psychological harm to the patient's family 
based on false expectations and discrediting the reputation of the medical research 
community because of misrepresentation of data (i.e., the Reuters new report). Dr. 
Sobol responded that he does not have control over the press. Dr. Leventhal quoted a 
statement from a scientific abstract that was submitted by the investigators to the 
International Conference on Brain Tumor Research and Therapy 1993: "these 
encouraging results suggest that evaluation of this form of IL-2 gene therapy in 
additional patients with glioblastoma is warranted." Dr. Smith stated that immunological 
data obtained from the single patient trial are uninterpretable; however, the animal data 
provided in support of the investigators' colon cancer protocol should be considered 
acceptable preclinical data for the glioblastoma study. Immunological experiments could 
be redesigned to assess the baseline that would render the present data interpretable. 
Additional immunophenotype data are necessary. 
Dr. DeLeon stated that the RAC should be objective and consistent in its review and 
approval of protocols. Since the investigators have provided in vitro data as well as in 
vivo data to support the colon carcinoma protocol, she recommends approval of this 
protocol. Dr. Ifrogstad said that this protocol could be strengthened to obtain 
interpretable data. Dr. Krogstad said that the RAC should consider approval of this 
study on the basis of the material submitted, and should not consider the confrontational 
circumstances in which the single patient protocol was approved. 
The motion to disapprove the protocol passed by a vote of 10 in favor, 5 opposed and 1 
abstention. The majority of the RAC members concluded that the preclinical data 
derived from a single patient protocol was inadequate to justify the proposal. 
XI. ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING A HUMAN 
GENE TRANSFER PROTOCOL ENTITLED: RETROVIRUS-MEDIATED TRANSFER 
OF THE cDNA FOR HUMAN GLUCOCEREBROSIDASE INTO PERIPHERAL 
BLOOD REPOPULATING CELLS OF PATIENTS WITH GAUCHERS DISEASE /DR. 
SCHUENING 
Review-Dr. Haselkom 
Dr. Walters called on Dr. Haselkom to present his primary review of the protocol 
submitted by Dr. Friedrich Schuening of the Fred Hutchinson Cancer Research Center, 
Seattle, Washington. Dr. Haselkom noted that the RAC has previously reviewed and 
approved other gene transfer protocols for the treatment of Gaucher's disease. 
Gaucher's disease is caused by a genetic defect in which the lack of glucocerebrosidase 
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