Recombinant DNA Advisory Committee - 2/10-11/92 
the terms "engrafted" and "harvested marrow" to make an understandable document for 
the patient. Another area of concern was whether the transduced cells would survive the 
freezing process. Dr. Cometta submitted data on an overnight freezing process. However, 
in most instances, the transduced cells would be frozen longer. There is a concern about 
the low percentage of transduced cells which could affect the results, particularly if only a 
few malignant cells contribute to the relapse. There is also the issue of reimbursement to 
patients for unanticipated injury. Indiana University officials indicated that they would not 
rewrite the patient compensation clauses but did require written documentation of 
insurance specifically for BMT. While this assures that the cost of the transplant will be 
covered, it does not address coverage of unexpected injury. 
Review— Dr. Carmen 
Dr. Carmen stated that the protocol is markedly uncontroversial. Evidence shows that 
there is no risk to human subjects. The HGTS had insisted that Dr. Cornetta agree to 
three stipulations: (1) to add to the informed consent document the phrase that no new 
information might be elicited from the research; (2) to provide proof that the transduced 
cells would survive freezing prior to reinfusion; and (3) to develop statistics demonstrating 
the probability that the experiment would be successful with such likelihood being a 
function of the percentage of transduced leukemic cells transplanted and the number of 
those cells that lead to relapse and to provide a stopping point after a certain percentage 
of patient recipients have been monitored. Dr. Carmen stated that Dr. Cornetta has 
fulfilled these three stipulations of the HGTS. 
The HGTS recommended that Dr. Cornetta renegotiate with the Institutional Review 
Board (IRB) concerning the consent form language which states that the patient would 
receive no compensation if injured in the research and would be responsible for payment 
of emergency medical treatment required as a result of the treatment. Dr. Cornetta 
responded that discussions were held with the IRB, but no change resulted. Dr. Carmen 
stated that compensation should not be an appropriate criterion for assessing Dr. 
Cometta's protocol. 
Other comments 
Dr. Doi asked Dr. Cornetta if the percentage of transduced cells was sufficient and if the 
sensitivity of the PCR was 1,000-fold more than necessary to detect the gene in the 
leukemic cells. If Dr. Cornetta's assay is this sensitive, then the protocol is quite good. 
Mr. Barton asked if there was excess overlap among the gene marking protocols being 
used to study leukemia, if these different researchers would exchange data, and if such 
data exchange would affect stopping points. 
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