Recombinant DNA Advisory Committee - 10/7-8/91 
Dr. Leventhal said that the risks of the procedure are much lower for the patient who is 
symptomatic with angina pectoris than the risk of death, liver transplant, or coronary 
artery bypass. It is a deprivation, not a protection, to disallow a patient that needs 
treatment. 
Dr. Mclvor asked for Dr. Wilson's viewpoint on patient availability. Dr. Wilson said he 
thought it would be possible to find three patients who were over 18. He stressed that 
they would not be the patients most likely to benefit from this procedure. It is much 
easier to efficiently complement hepatocytes from a younger patient. Patients with the 
most aggressive fulminant disease are the younger patient population who are receptor- 
negative. 
Ms. Buc said recognizing that there are risks, the question is not really who is most likely 
to benefit but from whom it is appropriate to elicit consent. Unless the younger children 
are different in kind, not just in degree from the older children, then the benefit alone 
should not be the only factor in deciding to start with the younger patients. Dr. Wilson 
said he could enroll adults, but he reiterated that the chance of demonstrating efficacy in 
that group is not as high as it is with the younger group. 
Dr. Mclvor said that this protocol varies considerably from some of the previous 
protocols in that it could provide a therapeutic effect. He said he would like to see the 
investigator given a chance to find the circumstances under which efficacy will most 
likely be demonstrated, therefore, the experiment is most likely to work. He said he was 
in favor of not restricting this protocol to adults. 
Dr. Kelley suggested a compromise that would encourage the investigators to seek older 
patients if possible. This is an important experiment, and it would be a shame to see it 
delayed for a length of time awaiting that particular requirement. 
Dr. Schaechter said the biology of this situation, as well as the practicalities, demand that 
the work be done with children. 
Dr. Miller said he would be surprised if the FDA accepted a limitation to adults on the 
basis of these consent arguments. Dr. Carmen agreed. 
Dr. B. Murray added that because of the risk of other experimental therapies, such as 
liver transplantation or heart-lung transplantation, she would be opposed to the age limit 
restriction. 
Mr. Capron asked if the receptor-deficient patients are included in the protocol for 
comparative reasons. Will there be any clinical benefit to those patients? Dr. Wilson 
replied that the expectation is that they would get benefit. 
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Recombinant DNA Research, Volume 15 
