CBL0137 is primarily investigated for its anticancer properties, particularly in preclinical and early clinical studies. It functions as a small molecule that modulates chromatin structure by inhibiting the activity of FACT (Facilitates Chromatin Transcription), a protein complex involved in DNA repair and gene expression. By targeting FACT, CBL0137 disrupts cancer cell proliferation and induces apoptosis in tumor cells. It has shown promising activity against a range of solid tumors and hematological malignancies, including glioblastoma, pancreatic cancer, and leukemia. Its ability to activate the innate immune response against tumors adds another layer to its therapeutic potential, making it a candidate for combination therapies with immunotherapies such as checkpoint inhibitors. CBL0137 is also being explored for its ability to sensitize resistant cancer cells to other treatments, enhancing the efficacy of chemotherapy and radiation. Due to its unique mechanism of action, it represents a novel approach in oncology drug development, especially for tumors with limited treatment options.