MUC1 immunotherapy is here to stay.

INTRODUCTION: Success of HBV vaccines in reducing the incidence of liver cancer, and HPV vaccines in reducing preneoplastic cervical lesions, demonstrate the potential of cancer reduction by harnessing the immune system. For most human cancers, infectious etiology is not known but other tumor antigens, candidates for vaccines, have been identified. AREAS COVERED: The authors discuss knowledge accumulated the last two decades on the tumor antigen MUC1 that has put it at the top of the list as an immunotherapy reagent. They examine evidence that anti-MUC1 immunity affects tumor development and prognosis. Finally, they review two decades of immunotherapy trials targeting MUC1, focusing primarily on vaccines but also adoptive antibody and T-cell therapies. EXPERT OPINION: Most approaches targeting MUC1 have been immunotherapies administered to date to more than 1200 patients in clinical trials. Even though these trials focused on advanced cancer, encouraging results were reported particularly for less immunosuppressed patients. Furthermore, spontaneous anti-MUC1 immune responses are associated with better prognosis or with a reduced lifetime risk of developing MUC1+ cancers. MUC1 is abnormally expressed in over 80% of all cancers. Successfully targeting this molecule could benefit over a million patients diagnosed yearly with MUC1+ tumors just in the USA.