APR-246 is primarily used in cancer therapy, particularly for tumors with mutations in the p53 gene, which is one of the most common genetic alterations in human cancers. It works by restoring the normal function of mutant p53, enabling the protein to trigger apoptosis (programmed cell death) in cancer cells. This makes it a promising agent in treating cancers such as ovarian, breast, and myeloid malignancies. It has shown synergistic effects when combined with other chemotherapeutic agents or radiation, enhancing tumor cell killing. Clinical trials are exploring its use in both hematologic and solid tumors, especially in patients with TP53 mutations. Its ability to cross the blood-brain barrier also opens potential applications in treating brain tumors.