GO-203-2c + decitabine

GO-203-2c + decitabine is an investigational combination therapy for cancer, specifically studied in relapsed or refractory acute myeloid leukemia (AML). GO-203-2c is a rationally designed D-amino acid cell-penetrating peptide that inhibits the MUC1-C subunit of mucin 1 (MUC1), an oncoprotein over-expressed in many human cancers and associated with poor prognosis. By blocking MUC1-C homodimerization and nuclear translocation, GO-203-2c disrupts downstream oncogenic signaling pathways and induces cancer cell death. Decitabine is a nucleoside analog DNA methyltransferase inhibitor that acts as an epigenetic modulator by hypomethylating DNA, thereby reactivating silenced tumor suppressor genes. The combination aims to enhance anti-leukemic effects through complementary mechanisms—targeting both oncogenic signaling (via MUC1 inhibition) and epigenetic regulation (via DNA demethylation). This regimen has been evaluated in phase I/II clinical trials for patients with relapsed or refractory AML[1][3][6][8].