Human Gene Therapy Subcommittee - 11/21-22/91 
pay research-related costs. Researchers and their institutions should be obligated to 
cover these experimental costs through alternative funding mechanisms. Dr. Parkman 
added that the majority of institutions do not agree to cover research-related injuries as 
part of their official policy. Mr. Capron agreed that the majority of institutions include 
an open-ended statement in the informed consent document that the issue will be 
discussed or that cost will only be covered if the injury is a result of neglect. 
Dr. Walters reminded the HGTS to consider the larger context of the protocol. The 
subcommittee has reviewed similar protocols, and the same standard of review should 
apply to this protocol as to previously reviewed protocols. 
Ms. Meyers asked about the differences, other than the patient population, between this 
protocol submitted by Dr. Cometta and Dr. Brenner's protocol. Dr. Mclvor explained 
that Dr. Cometta's protocol involves AML and acute lymphocytic leukemia (ALL) in 
adult patients with varying stages of disease. Dr. Brenner proposes studies only in 
children with AML who are in remission. Dr. Mclvor questioned if the interpretation of 
Dr. Cometta's protocol would be complicated by these variables. 
Presentation--Dr. Cometta 
Dr. Cometta said that this protocol is a collaboration between investigators at Indiana 
University, GTI, and the Molecular Hematology Branch of the National Heart, Lung, 
and Blood Institute of NIH. He presented an overview of the bone marrow 
transplantation protocol. Bone marrow will be harvested from leukemia patients, 
cryopreserved, and stored for reinfusion following high dose chemotherapy. Controversy 
exists regarding the contribution of transplanted marrow to disease relapse. Sixty-five 
percent of all second or later remission patients will relapse with their leukemias. This 
protocol is designed to determine if relapse occurs as a result of remaining leukemia 
cells not purged by the chemotherapy regimen, or from the transfer of malignant cells in 
the transplanted marrow. Relapse may also result from a combination of both of these 
factors. Determining the source of relapse will be a key to success of bone marrow 
transplantation for the treatment of leukemia. 
Dr. Cometta addressed the issue of tumor burden in these patients. All eligible patients 
will be in remission. Following their first relapse, patients will be treated with two 
courses of intensive chemotherapy that will result in a four log decrease in cell number 
(approximately 10 6 cells). Even in those rare cases where a patient's tumor burden can 
be reduced to 100 total tumor cells, the patient will still relapse with his/her disease. 
Statistical analyses indicate that if tumor cells remaining in the transplanted marrow are 
the source of relapse, there is a greater than 95% chance of detecting leukemia cells in a 
total of 10 patients. 
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