Human Gene Therapy Subcommittee - 11/30/90 
Ms. Meyers said she had concerns about the insurance coverage and the costs of the 
research. In cancer research, insurance carriers refuse payment because of the 
impossibility of determining what has happened as a result of the cancer or as a 
result of the experimental therapy. She also expressed concern with the lack of 
preclinical data and that this protocol is similar to another protocol which is also 
under consideration by the RAC. 
Dr. Walters summarized the comments of the reviewers into four areas of concern: 
1. the lack of sufficient data on transduction efficiency of bone marrow 
cells as presented in the research design, 
2. does a negative result have any meaning and the low 
likelihood of obtaining a positive result? 
3. the informed consent transaction between the patient and the 
investigator, and 
4. the financial aspects of the study if something goes wrong or the 
patient's health insurance will not pay for part of the treatment. 
Dr. Parkman said that in acute lymphoblastic leukemia (ALL), most genetic marker 
studies show that the origin of the disease is monoclonal; however, a major question 
is whether a single daughter cell produces the relapse or if it requires multiple 
daughter cells. The terms "monoclonal" and "polyclonal" are coi^sing. 
Dr. Parkman said the investigators had the ability to perform additional in vitro 
preclinical experiments to develop some quantitation of the efficiency of gene 
marking in leukemic progenitor cells, and this result would strengthen the protocol. 
Dr. Erickson said the investigators had put forward a statement in this protocol that 
normal bone marrow cells would not be transfected; however, other protocols have 
expressed a desire to study transfection of normal bone marrow cells. He asked for 
clarification on this issue. 
Dr. Parkman said there was an issue of selectivity. There were attempts to try to 
transfect true pluripotent hematopoietic stem cells; there is no evidence that 
transfection has been successfully accomplished. If the experiment was successful, it 
would be fortuitous, but that it should not be anticipated. Normal progenitor cells 
could be expected to continue to carry this marked gene, and that they will remain 
viable for some time depending upon their normal life expectancy. 
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Recombinant DNA Research, Volume 14 
