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. Notably, CBL0137 has demonstrated the ability to cross the blood-brain barrier, making it a candidate for treating brain tumors. Additionally, it may enhance the effects of other therapies such as radiation and chemotherapy by sensitizing resistant cancer cells. Due to its immunomodulatory effects, CBL0137 is also being explored for its potential to stimulate antitumor immune responses, possibly improving outcomes in combination with immunotherapies. Its broad mechanism of action and ability to target multiple cancer pathways support its development as a novel oncology therapeutic.