Seclidemstat is an investigational small molecule inhibitor targeting the enzyme lysine-specific demethylase 1 (LSD1). It is primarily being studied in oncology for its potential to modulate gene expression in cancer cells. By inhibiting LSD1, seclidemstat helps restore normal differentiation pathways and reactivates silenced tumor suppressor genes, particularly in cancers driven by aberrant epigenetic regulation. It has shown promise in clinical trials for treating Ewing sarcoma and other sarcomas characterized by specific genetic translocations, such as those involving the EWSR1-FLI1 fusion protein. The drug’s mechanism enhances the expression of genes that promote cell differentiation and reduces the unchecked proliferation of malignant cells. Seclidemstat is also being explored in combination therapies with other agents, such as hormonal therapies or chemotherapy, to improve anti-tumor responses in solid tumors and hematologic malignancies. Its epigenetic modulation offers a novel approach to cancer treatment, especially in tumors resistant to conventional therapies.