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ABSTRACT
Introduction: Despite uninterrupted efforts made to develop newer drugs and schedules in the treatment of acute myeloid leukemia (AML), the induction of chemotherapy with cytarabine and an anthracycline (the classical ‘7 + 3’) still remains the standard of care. Unfortunately, many patients are not suitable for intensive induction therapy due to poor prognosis, age and comorbidities. For these challenging populations, the clinical outcome is poor and novel approaches are desperately required. One of these approaches is the liposomal formulation of drugs, allowing the delivery of more drug to the disease site and thereby improving efficacy and reducing toxicity. CPX-351 is a liposomal formulation of the cytotoxic drugs cytarabine and daunorubicin at synergistic concentrations.
Area covered: In this article, we review the preclinical and clinical experience to date with CPX-351 for patients with AML. Phase I and II studies have been published and a randomized prospective phase III study has recently been completed and presented as an abstract.
Expert opinion: CPX-351 is exclusively developed for use in AML. It is clearly active, as would be expected from its component makeup, and phase III data suggest that this may be the preferred initial combination therapy, at least for older patients with secondary AML.
Declaration of interest
The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.