Phase I/Ib Trial Of The Muc1 Inhibitor Go-203-2c Alone And In Combination With Decitabine For Acute Myeloid Leukemia

MUC1, an oncogenic protein aberrantly expressed on AML and leukemic stem cells, supports critical aspects of the malignant phenotype including resistance to apoptosis, cell proliferation and autonomous self renewal. GO-203-2C is a novel cell-penetrating peptide that blocks homodimerization of the MUC1-C subunit required for nuclear translocation and downstream signaling. In murine AML models, treatment with GO-203-2C resulted in specific eradication of myeloid leukemia. GO-203-2C is synergistic with hypomethylating agents in pre-clinical models of AML. Based on these findings, we are conducting a phase I/Ib study of GO-203-2C alone and in combination with decitabine in patients with AML.