IDH1 Inhibitor 1 is primarily used in the field of oncology, specifically for targeting cancers associated with mutations in the isocitrate dehydrogenase 1 (IDH1) gene. These mutations are commonly found in certain types of gliomas and acute myeloid leukemia (AML). The inhibitor works by blocking the abnormal activity of the mutated IDH1 enzyme, which is responsible for producing an oncometabolite called 2-hydroxyglutarate (2-HG). By inhibiting this enzyme, the compound helps to reduce the accumulation of 2-HG, thereby restoring normal cellular differentiation and potentially slowing tumor growth. This makes IDH1 Inhibitor 1 a promising candidate for precision medicine approaches in treating IDH1-mutated cancers. Additionally, it is being studied in combination therapies to enhance its efficacy and overcome resistance mechanisms in cancer treatment.