Human Gene Therapy Subcommittee - 11/30/90 
5. if there is no possibility of an allogeneic transplant because of the age of 
the donor or lack of donor, an autologous transplant is recommended. 
After the patient has been determined that an autologous transplant is the 
recommended treatment, he/she would be presented with the question of whether or 
not they wish to participate in the protocol. 
Dr. Cornetta noted that the patients would not be asked to pay any direct costs 
involved with this particular protocol nor would they be required to supply any 
samples in addition to those required for a standard autologous BMT. 
Dr. Cornetta noted that the Wisconsin IBC limited the protocol to four patients so 
that information could be derived regarding: 
1. patients are not infected with replication-competent amphotropic virus; 
2. no acute toxicity is observed related to the introduction of this procedure 
into the transplant, or any outgrowth of a malignant clone or some other 
unexpected catastrophe that may occur because of the procedure; and 
3. data will be reviewed in regard to PCR in normal bone marrow cells. 
Dr. Cornetta said that he believed it was not the IBCs intention to look at long-term 
data, but merely to look at preliminary data. Within a year, the data would be 
prepared and presented to the IBC. 
Dr. Parkman said that the reviewers perceive the labeling of the bone marrow as the 
crucial experiment. He asked for clarification on what percent of marrow cells were 
labeled in both normal and leukemic cells as well as the clonigenic potential of the 
leukemic cells that were used. 
Dr. Cornetta replied that the clonigenic potential could not be answered in vitro, and 
any such results could be misleading in vivo. There was a 1% infection rate found in 
a blast cell colony assay in one patient. 
Dr. Parkman said that since most circulating blasts are not cycling and since non- 
cycling cells are difficult to infect, crucial information is the percent of leukemic 
progenitors that can be infected. The statement made that pluripotent 
hematopoietic stem cells were not infected was in error; long-term cultures would be 
necessary to prove this result. The St. Jude's data, which looks at committed 
hematopoietic progenitor cells, say nothing about uncommitted progenitor stem cells. 
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Recombinant DNA Research, Volume 14 
