Recombinant DNA Advisory Committee - 12/3-4/92 
discuss why he is required to comply with this policy since his own IRB has raised 
questions regarding the issue. 
Ms. Meyers said that patients will be compensated for participation in this protocol. 
There are moral and ethical concerns regarding such compensation. Specifically, patients 
who are disabled and without income may be coerced unintentionally into participating 
in the study. 
Dr. Post asked Dr. Ginsberg if the Ela minus virus in monkey cells is significantly 
different from the Ela minus virus in human cells. Dr. Ginsberg replied that in the 
consideration of safety, one should always consider results obtained in the animal model 
in which pathology is produced. Since pathology is not produced in the monkey, then it 
should not be used for comparison to the human situation. Dr. Doi asked for 
clarification regarding data on the MOI. 
Dr. Parkman noted that the investigators will monitor virus secretion from the patient's 
nose. If a patient were to swallow some of the vector and it replicated in the 
gastrointestinal tract, there could be secretion from the stools. Therefore, a patient 
could have negative cultures from the nose but still have positive excretion for the stools. 
Why me stool cultures not being performed? If patients are still secreting or excreting 
virus after 1 month, they would be allowed to leave the negative pressure containment 
facility for humane reasons. What are the risks to other individuals of releasing a 
patient that is known to be actively secreting virus? 
Presentation-Dr. Welsh 
Dr. Welsh stated that he would limit his presentation to the issues of safety and efficacy. 
The safety profile in monkeys, cotton rats, hamsters, and cultured cells are very 
encouraging; however, there are specific questions about this therapy that only can be 
answered in the human. Since minor safety concerns still exist, a conservative approach 
to this therapy is being proposed. Information derived from this early study will provide 
critical data that will be used for the design of future clinical efficacy trials. 
Dr. Welsh presented a brief outline of the protocol. Three patients will be entered into 
the study. In response to Dr. Hirano's concern about the limited number of patients 
enrolled in this study, Dr. Welsh stated that this number of individuals will provide 
sufficient information to proceed to trials in the lung. The dosages of vector that will be 
administered, between 2 x 10 6 and 5 x 10 7 PFU, have been chosen to provide safety and 
efficacy data. Exclusion criteria and precautions have been outlined in the protocol. 
Nasal epithelium has been chosen as a model system for the preliminary studies because 
the nasal and intrapulmonary epithelial cells have the same morphology, i.e., barrier 
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