Apaziquone as an intravesical therapeutic agent for urothelial non-muscle-invasive bladder cancer

Introduction: Urothelial carcinoma of the bladder is a disease prone to recurrence. A new cytotoxic drug, apaziquone, is an analog of mitomycin C. Given via intravesical instillations it has the ability to specifically target cancer cells. Areas covered: This article reviews the discovery and pharmacological properties of the agent and the first steps of its application in human disease. The poor performance of the drug as a systemic medication is discussed along with in vivo and in vitro studies that have led to current intravesical applications with encouraging results. In detail the limited number of clinical studies on the drug and compare them with relevant series on currently used agents. In addition, different strategies to enhance the efficacy of other agents are discussed and possible application of this experience to apaziquone is proposed. Expert opinion: Apaziquone is a promising local agent for the treatment and prevention of recurrent urothelial carcinoma of the bladder. The limited number of Phase II studies has demonstrated potency and relatively low toxicity. In light of the dearth of randomized controlled trials, Phase III studies are urgently needed before any conclusive pronouncements on this agent can be made.