Human malignant brain tumor response to herpes simplex thymidine kinase (HSVtk)/ganciclovir gene therapy.

Growing cells from human brain tumors have been treated in vitro and in vivo with murine therapeutic retroviral producer cells. The therapeutic retrovirus carried the potential suicide gene thymidine kinase (tk) from the herpes simplex virus (HSV). After a few days, in which a large proportion of the tumoral cells had the opportunity to acquire a copy of the retrovirus, treatment with ganciclovir was initiated and considered responsible for considerable cell death both in vitro and in vivo. The in vivo experiments were performed in five adult patients who had failed standard therapy and were expected to survive only a few weeks.