Human Gene Therapy Subcommittee - 11/30/90 
receiving care and/or other compensation, but merely that the patient should not 
assume there is coverage in place for such events. 
Dr. Cometta noted that many insurance carriers will not cover autologous BMT, 
which can cost the patient as much as $150,000 for both in- and out-patient costs 
during the first year. Generally these matters are solved before the patient is 
admitted to the hospital for a BMT. In some cases, since the State of Wisconsin had 
disability coverage, that patients were asked to leave their current carrier and 
assume coverage by the state if they are in need of autologous BMT. The hospital 
requires that all patients are capable of paying for the transplant prior to admission. 
Dr. Cometta then turned to the issue of preclinical data. Three dry mns had been 
undertaken using samples from patients with acute leukemia to study gene marking 
using the LNL6 vector. The same procedures described in the protocol were used; 
however, the cells were incubated for 72 hours to allow for vector integration into 
the cells. The cells were analyzed by PCR in Dr. Anderson's laboratory. In all three 
patients (one with acute lymphocytic leukemia (ALL) and two with acute 
myelogenous leukemia (AML)), PCR analysis showed insertion of the LNL6 vector. 
Additionally, a mock infection with leukemic cells had been performed in the same 
manner as the treated cells exposed to the vector except that the supernatant was 
vector-free. No evidence of the LNL6 vector was found. This result was confirmed 
by both PCR and Southern blot. Therefore, it is possible to insert the vector into 
the cells of interest without getting false-positives. 
Referring to questions asked by Dr. Zallen about approaching patients to participate 
in the protocol, Dr. Cometta said it was a "Catch-22" situation, because the people 
most likely to be able to explain the risks of the procedure are those directly 
involved with it. However, the patients are approached by: 
1. review of history and physical exam; 
2. discussion of alternatives and risks and benefits of transplantation; 
3. if patients are willing to pursue the possibility of transplant, e.g., 
recommend what would be the best type of transplant, normally 
allogeneic transplant in the case of acute leukemia; 
4. search the patient and his/her siblings for a matched donor and, if not 
available, look for an unrelated donor; 
Recombinant DNA Research, Volume 14 
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