Human Gene Therapy Subcommittee - 4/5/91 
Mr. Capron asked if the update reports could be written instead of oral presentations. 
He thought that possibly Dr. Wivel could review the written reports and convey his 
findings in writing to the subcommittee. If subcommittee members review the written 
report and identify a reason to ask questions of the investigator, then it could be 
requested that the investigator present an oral report. 
Dr. Leventhal thought it would be difficult to increase the efficiency of the subcommittee 
when there are new protocols that employ a particular vector whose long-term efficiency 
and safety have not been documented in the approved protocols. It is time to look at 
what is the most efficient and most efficacious way of obtaining this needed feedback. 
Dr. Walters said that with respect to the second gene transfer protocol, that too few 
patients had been treated and for too short a time for a report to be ready. Dr. 
Leventhal wondered if six months to a year is a reasonable interval. Perhaps it should 
be after the first group of patients has been treated. Dr. Walters agreed and added it 
would also be useful for the subcommittee to keep informed of the outcome of the FDA 
review and approval process. 
VIII. PROPOSED ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING 
A HUMAN GENE TRANSFER PROTOCOL ENTITLED: AUTOLOGOUS 
TRANSPLANTATION FOR CHRONIC MYELOGENOUS LEUKEMIA: RETROVIRAL 
MARKING TO DISCRIMINATE BETWEEN RELAPSES ARISING FROM RESIDUAL 
SYSTEMIC DISEASE VERSUS RESIDUAL CONTAMINATION OF AUTOLOGOUS 
MARROW: 
Dr. Leventhal noted that this protocol is very similar to the protocol that Dr. Brenner 
presented. Patients with chronic myelogenous leukemia (CML) who have resistant 
disease, defined either as failing to achieve remission with interferon, or having achieved 
remission and then had a blast crisis, are eligible for this study. During a period of 
remission, their bone marrow will be stored for a later autologous reinfusion. An aliquot 
of that marrow will be marked with the LNL6 vector, and, after the patient has been 
conditioned with cytotoxin, BP- 16, and total body radiation, the marrow will be reinfused. 
The patients will be followed and the marker studies performed to determine which cell 
populations are reconstituting, and in particular, which cell populations are giving rise to 
blast crisis if the patients go back into blast crisis, as a significant fraction of them will. 
Dr. Leventhal felt the issues were fairly similar to what had been discussed in the review 
of Dr. Brenner's protocol. She had asked Dr. Deisseroth whether his CML cells could 
be transduced with the reporter gene. He replied that he has transduced cells that 
would grow in selective medium although somewhat less efficiently than non-selected 
cells. 
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