Recombinant DNA Advisory Committee - 12/2-3/93 
the antibiotic of choice for the treatment of pneumonia. 
Ms. Meyers recommended that a letter be sent to Dr. Sorcher's IRB requesting that the 
statement about compensation for research-related injury be deleted from the Informed 
Consent document. 
Committee Motion 
A motion was made by Dr. Post and seconded by Dr. Secundy to approve the protocol 
contingent on the submission of data derived from the ongoing toxicity studies as 
outlined in Dr. Sorscher's response to Dr. Post dated December 2, 1993. The motion to 
approve the protocol passed by a vote of 15 in favor, 0 opposed, and 1 abstention. 
The RAC recommended that a letter be sent to the University of Alabama's IRB 
requesting deletion of the following paragraph from the Special Risks and Discomfort 
Related to Being Part of a Study of Gene Administration section of the Informed Consent 
document: 
"(b) Throughout the study I will continue to be monitored for complications which 
are normally associated with CF. Should any of these complications develop, I 
will be given appropriate therapy. If the complications do not appear to be 
related to the gene transfer protocol, the usual means of payment (for example, 
insurance) should be arranged. The University of Alabama, Birmingham, and the 
Children's Hospital of Alabama have made no provision for monetary 
compensation in the event of physical injury resulting from research and in the 
event of such injury, medical treatment is provided, but is not provided free of 
charge." 
XX. ADDITION TO APPENDIX D OF THE NJH GUIDELINES REGARDING A HUMAN 
GENE TRANSFER PROTOCOL ENTITLED: ADENOVIRUS-MEDIATED GENE 
TRANSFER OF CFTR TO THE NASAL EPITHELIUM AND MAXILLARY SINUS OF 
PATIENTS WITH CYSTIC FIBROSIS /DR. WELSH 
Review-Dr. Post 
Dr. Walters called on Dr. Post to present his primary review on the protocol submitted 
by Dr. Michael J. Welsh of Howard Hughes Medical Institute, Iowa City, Iowa. Dr. Post 
stated that this protocol is an extension of Dr. Welsh's first RAC approved protocol in 
which an adenovirus-CFTR vector was administered to the nasal epithelium of CF 
patients. This current proposal involves the multiple administration of increasing doses 
of a new vector to the nasal epithelium as well as the maxillary sinus. Safety of the 
modified dosing schedule will be demonstrated in the nasal epithelium prior to maxillary 
sinus administration. 
Dr. Post noted that the results obtained from Dr. Welsh's previous trial are promising. 
Gene transfer for CF is an excellent example of different investigators using separate 
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