Human Gene Therapy Subcommittee - 11/30/90 
Myelogenous Leukemia (AML), it is impossible to discriminate AMLs from normal 
precursor cells on the basis of surface antigens. This approach may be useful in 
analysis of ALL; however, it could be problematic since normal pre-B cells in the 
bone marrow may exhibit the same surface markers as ALL cells. 
Dr. R. Murray noted that the reviewers had expressed that there was a high 
probability of obtaining a negative result. Therefore, the investigator may not be 
able to answer experimental questions. Dr. Epstein said there was a possibility that 
a negative result might be uninterpretable in terms of the experiment. Dr. R. 
Murray said that in light of human subject considerations, it may be difficult to 
approve this protocol. Dr. Epstein reminded Dr. R. Murray that each protocol must 
be judged on its own merits, and that the difficulty in assessing this protocol stems 
from the lack of preclinical data available. 
Dr. Childress focused his attention on issues of informed consent. It is important to 
draw a distinction between the informed consent being solicited in the form which 
the patient will sign and consent in the social and institutional context of the word. 
It was obvious that the patients were being asked to take some minimal risk, but it is 
unfair to ask them to bear the cost of the research and any medical treatment that 
may be required for treating injuries which may result from the research. He noted 
that he was unclear as to the differences between this consent form and the one that 
every patient completes for a bone marrow transplantation. This problem is not 
confined to human gene therapy research; no higher standard should be placed on 
any particular research project or investigator than would be operative outside the 
study of human gene therapy. 
Mr. Capron asked Dr. Cornetta if these issues were discussed at a local level in 
regard to the consent form for the protocol. 
Dr. Parkman said that in his institution the standard consent form, including that for 
bone marrow transplantation, has a two page addendum which states that the 
hospital has malpractice insurance to cover negligence by its physicians and staff, and 
that the institution bears no responsibility for injuries suffered that are associated 
with untoward effects or injury resulting from any procedure other than negligence. 
Mr. Capron noted that institutional policies on this issue are heterogeneous, and the 
language in this protocol is fulfilling a Federal requirement to inform patients as to 
whether or not the institution will cover consequential injury. Institutions have 
varied in this respect, allowing for redress in an unjust situation without binding 
themselves in advance to paying for such costs. 
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Recombinant DNA Research, Volume 14 
