A lentiviral vector capable of expressing the HIV-1 vpr gene (Vpr lentivira
l vector) was constructed, and its in vivo anticancer effect was determined
against cutaneous tumors derived from the AT-84 oral cancer cells in immun
ocompetent mice. A single intratumoral injection of the Vpr lentiviral vect
or not only significantly reduced the primary tumor volume but also complet
ely regressed tumors in >40% of animals. More interestingly, the mice of wh
ich the primary tumors were completely regressed by the Vpr lentiviral vect
or were additionally protected from a secondary challenge of AT-84 cells. T
hese data suggest that the Vpr lentiviral vector elicits its anticancer act
ivity in part by the activation of the immune system. The above suggestion
is additionally supported by the failure of the lentiviral vector to demons
trate anticancer activity in immunocompromised nude or SCID mice. The Vpr l
entiviral vector offers a powerful new strategy for cancer gene therapy and
may be useful for the control of solid tumors, such as human oral squamous
cell carcinomas.