Anderson and Cornetta have considerable experience 
in RMGT in primates. In these experiments gene 
transfer into normal bone marrow was attained in 
the majority of animals (51,52). While the 
transduction efficiency in these experiments was 
too low for clinical applications of gene therapy, 
the RMGT protocol demonstrated that RMGT could be 
performed in the setting of AuBMT without 
significant effects on the viability of bone marrow 
cells and subsequent hematopoietic reconstitution. 
RMGT is currently being used in a human clinical 
trial in which tumor infiltrating lymphocytes (TIL) 
are marked with the LNL-6 vector (Appendix 3) . LNL- 
6 is a vector developed in the laboratory of A. 
Dusty Miller and is based on the Moloney murine 
leukemia virus (MoMLV) and contains a bacterial 
gene for neomycin resistance (neo R ) . LNL-6 is a 
safety-modified version of the vector N2 . The neo R 
gene product, neomycin phosphotransferase, is a 
selectable marker since it protects cells from the 
neomycin analogue G418, (which is normally toxic to 
mammalian cells) . LNL-6 is manufactured by Genetic 
Therapy Inc. and has been approved for limited use 
by the U.S. Food and Drug Administration. An IND 
will be submitted to the FDA regarding the use of 
LNL-6 in this protocol. 
This protocol is designed to study the feasibility 
of using RMGT in the setting of AuBMT. The patient 
population eligible for study is a high risk 
population with a recurrence rate as high as 70-80% 
in the first year. Accrual goals are 10 patients 
with AML and 10 patients with ALL. 
2 . 0 OBJECTIVES 
1.) To use RMGT to mark bone marrow cells used for 
autologous bone marrow transplantation (AuBMT) in 
patients with acute leukemia and to assess whether 
relapsed patients have leukemic blasts which 
contain the marker gene. If leukemic cells are 
shown to contain the retroviral-mediated 
transferred gene, it will establish that leukemic 
cells were contained in the bone marrow used for 
transplantation and these cells contributed to 
relapse. It will confirm the importance of marrow 
purging and may increase our understanding of 
leukemia relapse. 
Recombinant DNA Research, Volume 15 
[471] 
