¹Department of Endocrinology, Mayo Clinic, Rochester, MN, USA

²Stem Cell Laboratory, Mayo Clinic Cancer Center, Rochester, MN, USA

³Department of Nuclear Medicine, Mayo Clinic, Rochester, MN, USA

⁴Department of Urology, Mayo Clinic, Rochester, MN, USA

Correspondence to: J C Morris, Division of Endocrinology, Mayo Clinic, Guggenheim 625, 200 First Street SW, Rochester, MN 55905, USA

Radioiodine therapy, the most effective form of systemic radiotherapy available, is currently useful only for thyroid cancer because of thyroid-specific expression of the sodium iodide symporter (NIS). Here we explore the efficacy of a novel form of gene therapy using adenovirus-mediated in vivo NIS gene transfer followed by ¹³¹I administration for treatment of prostate cancer. Prostate cancer xenografts in nude mice injected with an adenovirus carrying the NIS gene linked to the cytomegalovirus (CMV) promoter revealed highly active uptake of radioiodine. Following administration of 3 mCi of ¹³¹I, we observed an average tumor volume reduction of 84 ± 12%. These results show for the first time that in vivo NIS gene delivery into non-thyroidal tumors is capable of inducing accumulation of therapeutically effective radioiodine doses and might therefore represent an effective and potentially curative therapy for prostate cancer.

Gene Therapy (2001) 8, 1524-1531.