Long-term rat survival after malignant brain tumor regression by retroviral gene therapy.

Total regression of malignant brain tumors was observed in Wistar rats after retrovirus-mediated gene therapy. Tumors were induced by inoculation of C6 rat glioblastoma cells to a specific location in the rat brain and the tumors that developed were visualized by magnetic resonance imaging (MR). Retroviral vectors were constructed from a defective murine retrovirus to which the thymidine kinase (tk 1) gene from herpes simplex was added (HSV1tk). The vectors produced therapeutic viruses upon their introduction into retrovirus packaging cells. Delivery of the producer cells to the tumor mass and subsequent antiherpetic treatment eradicated the tumors completely, as observed using MRI. Some of the treated animals have been followed for over 8 months and show no signs of recurrence.