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Investigational approaches for the treatment of advanced prostate cancer

Janet R Walczak, MSN, RN, CRNP
Roberto Pili, MD
Michael A Carducci, MD
Emmanuel S Antonarakis, MD
Section Editors
Nicholas Vogelzang, MD
W Robert Lee, MD, MS, MEd
Jerome P Richie, MD, FACS
Deputy Editor
Michael E Ross, MD


Androgen deprivation therapy (ADT) is highly effective therapy for men with advanced or metastatic prostate cancer, providing at least temporary disease control in over 80 percent of cases. However, the vast majority eventually develop progressive disease.

Once disease progresses, men are considered to have castration-resistant disease. A number of new therapies have been shown to prolong survival in this setting (table 1), but there remains a need for additional approaches that can control disease and prolong survival.

An increasing understanding of the biology of prostate cancer is leading to the development of new therapies that can target specific abnormalities in prostate cancer. Although a number of agents have failed in phase III or randomized phase II trials, other experimental approaches are in advanced stages of development. These are discussed in this topic. The overall approach to men with advanced disease is discussed separately. (See "Overview of the treatment of disseminated castration-sensitive prostate cancer".)


Prostate cancers are frequently characterized by abnormalities in a variety of growth factor signaling pathways that control the cell cycle and apoptosis. As these pathways are being understood, new therapeutic targets are leading to the development of new agents with activity against advanced disease. (See "Molecular biology of prostate cancer".)

Cabozantinib — Cabozantinib is an inhibitor of MET, VEGFR2, and RET. A phase II trial in patients with progressive, metastatic prostate cancer provided preliminary evidence of activity in men with bone metastases [1,2].

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Literature review current through: Nov 2017. | This topic last updated: Oct 19, 2017.
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