Introduction: Ipilimumab is a fully human monoclonal antibody that blocks Cytotoxic T lymphocyte-associated antigen 4 (CTLA-4) to potentiate antitumor T cell response. Ipilimumab is approved for the treatment of advanced melanoma based on improved overall survival. Clinical trials of ipilimumab in patients with metastatic castrate-resistant prostate cancer (mCRPC) have demonstrated some clinical activity, but have largely been disappointing to date.
Areas covered: Results of key clinical studies of ipilimumab in the treatment of prostate cancer, including clinical efficacy and toxicities, are summarized.
Expert opinion: There is likely a clinical benefit to ipilimumab in a subset of mCRPC patients. The development of biomarkers for enrichment treatment strategies that select for patients most likely to benefit from ipilimumab is a top priority. Similarly, an understanding of the factors predictive of toxicity will be important in the development of future treatment approaches.
Keywords: Cancer immunotherapy; Yervoy; immune checkpoint; ipilimumab; prostate cancer.