Radioiodine therapy, the most effective form of systemic radiotherapy avail
able, is currently useful only for thyroid cancer because of thyroid-specif
ic expression of the sodium iodide symporter (NIS). Here we explore the eff
icacy of a novel form of gene therapy using adenovirus-mediated in vivo NIS
gene transfer followed by I-131 administration for treatment of prostate c
ancer. 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
I-131, we observed an average tumor volume reduction of 84 +/- 12%. These r
esults show for the first time that in vivo NIS gene delivery into non-thyr
oidal tumors is capable of inducing accumulation of therapeutically effecti
ve radioiodine doses and might therefore represent an effective and potenti
ally curative therapy for prostate cancer.