Anticancer effects of APR-246 have been observed in several in vivo models, including an acute myelogenous leukemia (AML) mouse model that is predictive for the human disease. Similar effects are seen in multiple cancer cell lines, including those for ovarian and breast cancer.

In addition, reactivation of p53 by APR-246 also sensitizes cancer cells to chemotherapy; for example, APR-246 with platinum-based chemotherapeutic drugs provides a high synergistic effect in a cisplatin-resistant ovarian cancer cell line and in primary ovarian cancer cells from patients. Similar results have been observed in other cancer cell lines when APR-246 is used in combination with standard chemotherapy agents. These results not only indicate that APR-246 can boost the efficacy of standard chemotherapy but also suggest that combination therapy with APR-246 may help to overcome chemotherapy resistance in patients.