Recombinant DNA Advisory Committee - 03/1-2/93 
Dr. Knowles said that he prefers to use the term "gene therapy" because the objective 
protocol is to correct the cellular function of the cells in the nose. However, the 
terminology can be changed if required by the RAC. Dr. Miller said that the term "gene 
transfer" should be used instead of "gene therapy", because "gene therapy" implies that 
the corrected gene will restore function in the entire body. 
Dr. Knowles explained that the issue of patient follow-up is very controversial at his 
institution. The patients will be a young adult population. There is a high probability 
that they will move several times within their lifetime. Is it necessary that they return to 
the institution after a year? Ms. Meyers stated that it is incumbent on investigators to 
follow these patients for life. Ms. Meyers reminded the investigators that the patients 
may not necessarily have to return to the institution. The long-term follow-up clause 
implies that the investigator will maintain a registry and contact the patient periodically 
with regard to their clinical status. Dr. Parkman reminded the RAC that the Points to 
Consider clearly indicates that patients will be followed for life. 
Ms. Meyers commented on the previous discussion regarding the approval of duplicate or 
similar protocols. In the past 3 years, approximately 90% of the protocols reviewed by 
the RAC have been for the treatment of cancer. There have been very few protocols 
designed for the treatment of genetic diseases. The investigators should be applauded 
for pursuing multiple trials. 
Dr. Post asked if there was data about the particular cell types that are transduced. Dr. 
Wilson responded that the majority of cells that are transduced are epithelial cells; 
however, there may be low level transduction of alveolar macrophages. 
Committee Motion 
A motion was made by Dr. Post and seconded by Dr. Miller to approve the protocol 
contingent on the following: (1) The correct sequence of the CFTR cDNA must be 
determined prior to administration of the vector to patients and provide a should provide 
a report at the next RAC meeting regarding resolution of the CFTR cDNA sequence. 
(2) Replace the phrase "gene therapy" with "gene transfer" throughout the document. 
The motion was approved by a vote of 16 in favor, 0 opposed, and 1 abstention. 
XIV. PROPOSED ADDITION TO APPENDIX D OF THE NIH GUIDELINES REGARDING 
A HUMAN GENE THERAPY PROTOCOL ENTITLED: PHASE I STUDY OF 
TRANSFECTED CANCER CELLS EXPRESSING THE IL-2 GENE PRODUCT IN 
LIMITED STAGE SMALL-CELL LUNG CANCER /DR. CASSILETH 
Review-Dr. Miller 
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Recombinant DNA Research, Volume 17 
