Gold compounds are a class of metallodrugs with great potential for
cancer treatment. During the last two decades, a large variety of
gold(I) and
gold(III) compounds are reported to possess relevant antiproliferative properties in vitro against selected human tumor cell lines, qualifying themselves as excellent candidates for further pharmacological evaluation. The unique chemical properties of the
gold center confer very interesting and innovative pharmacological profiles to
gold-based metallodrugs. The primary goal of this review is to define the state of the art of preclinical studies on anticancer
gold compounds, carried out either in vitro or in vivo. The available investigations of anticancer
gold compounds are analyzed in detail, and particular attention is devoted to underlying molecular mechanisms. Notably, a few biophysical studies reveal that the interactions of cytotoxic
gold compounds with
DNA are generally far weaker than those of
platinum drugs, implying the occurrence of a substantially different mode of action. A variety of alternative mechanisms were thus proposed, of which those involving either direct mitochondrial damage or
proteasome inhibition or modulation of specific
kinases are now highly credited. The overall perspectives on the development of
gold compounds as effective anticancer drugs with an innovative mechanism of action are critically discussed on the basis of the available experimental evidence.