Mucin 1 (MUC1) is particularly well suited as a cancer immunotherapy target due to the elevated protein expression and aberrant forms associated with malignancy. A variety of therapeutic strategies have been explored, including antibodies intended to induce cancer cell destruction, and vaccinations with peptides, tumor extracts, and gene expression systems.

MUC1 immunotherapeutic strategies have included vaccination with peptide sequences, glycan molecules, viruses, and dendritic cells, monoclonal antibodies and monoclonal antibody conjugates. Here we review the relevant clinical trials in each field of immunotherapy with particular focus on large and recently published trials.

Long clinical experience in the trial setting has reduced concerns of immunotherapy associated toxicities and inappropriate immune responses, with the main limitation (common to many experimental approaches) being a lack of clinical efficacy. However, there have been sufficient treatment-associated responses to justify continued pursuit of MUC1 targeted immunotherapies. The focus now should be on application to the relevant cancers under appropriate circumstances and combination with the emerging non-specific immunotherapy approaches such as the PD-1 pathway inhibitors.