Human Gene Therapy Subcommittee - 7/30/90 
place on a demonstration of expected efficacy involving human 
gene therapy protocols. He said the whole issue of the 
questioning by the subcommittee on the ADA protocol centered 
around issues of efficacy of the treatment. He asked that this 
issue be clarified. Dr. Mulligan said that it was important, in 
order to look at efficacy, to initially ask at what dose it may 
be detected. This is the type of proposal which falls between 
being a diagnostic study and being an attempt to elicit an 
efficacious result. 
Dr. Parkman said that for a Phase I study to be instituted there 
must be adequate preclinical information to prove that the basic 
concept is valid. There must be some reason to believe an 
efficacious outcome will occur as a result of the protocol in 
order to justify undertaking a study of safety and toxicity in a 
Phase I trial. Therefore efficacy is not really of tertiary 
concern, but must be viewed as a primary concern, even in a Phase 
I trial. 
Dr. Childress agreed with Dr. Parkman and said that if there were 
no theoretical or experimental basis for thinking a therapy could 
work, there would be no interest in questions of safety about 
that therapy . 
Dr. Zallen asked the following four questions relative to 
biopsies of tumor or tissue: 
1. How many biopsies will be taken from each patient? 
2. Will the patient submit or sign a separate consent form 
for each biopsy? 
3 . How could the patient distinguish between a biopsy 
performed for therapeutic reasons and one performed for 
research reasons? 
4. Could a patient decline to have a biopsy taken? 
Dr. Rosenberg said the consent form would allow multiple biopsies 
to be taken on any patient, and the number would vary from 
patient to patient. However, in most cases only local anesthesia 
would be required, the investigator would explain the purpose of 
each biopsy, and the patient would have the option of accepting 
or rejecting the procedure. A patient's declination of a biopsy 
would not affect his/her status in any way. Dr. Rosenberg said 
the protocol called for a maximum of five biopsies, one 
pre-treatment and four post-treatment. 
Recombinant DNA Research, Volume 14 
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