The management of Acute Myeloid Leukemia (AML) (with the exception of acute promyelocytic leukemia) has remained largely unchanged over the past 40 years. In particular, patients defined as high-risk, according to the 2017 European Leukemia Net recommendations, represent a subgroup with poor response to current therapies that are frequently associated with high-grade toxicity and potentially fatal complications. Areas covered: Preliminary results from an ongoing phase III clinical trial suggest that CPX-351 could represent an interesting treatment option in both induction and 'bridge-to-transplant' settings. In particular, 60- to 75-year-old patients with secondary AML, when treated with CPX-351, exhibit superior overall survival (HR = 0.69; P = 0.005; median OS 9.56 vs. 5.95 months), event free survival (HR = 0.74; P = 0.021), and composite response rate (47.7% vs. 33.3%; P = 0.016) as compared to standard '7 + 3' therapy. Herein, we detail the main pharmacological features of CPX-351 and review updated results of clinical trials investigating its employment in AML. Expert commentary: Novel liposome-based drugs display a high therapeutic index and represent a promising alternative to unencapsulated drugs, especially when high-risk features complicate the use of standard treatments. Further efforts in both understanding AML biology and improving nanodrug design are needed.
Keywords: Acute myeloid leukemia; CPX-351; cytarabine; daunorubicin; drug delivery; liposome; nanomedicine; nanoparticles.