ARQ531: The therapy that targets multiple pathways in acute myeloid leukemia

So far this century we have witnessed the introduction of a number of targeted therapies, developed through rational drug design, which have changed cancer treatment and resulted in improved outcomes for many patients, including those with a spectrum of chronic lymphoid and myeloid malignancies.1,2 However, despite improved understanding of the biology of acute myeloid leukemia (AML), similar scale benefits by targeting kinases and other intracellular and surface proteins have yet to be realized, and the prognosis for patients with AML remains poor. Moreover, cytotoxic drugs and therapies developed in the last century currently remain the backbone of AML treatment, and as AML primarily affects the elderly, many of whom are therefore frail with multiple co-morbidities, the clinical application of such curative therapies is somewhat limited.3 Furthermore, even in those fit enough for intensive chemotherapy, both relapse and treatment resistance are common, due to the aggressive nature of the disease. The search therefore continues for biology-driven targeted treatments for patients with AML which can be delivered to all, and at the same time increase remission rates, reduce relapses and prevent treatment resistance. The expectation is that these therapies will come from advances in the understanding of the biology of AML.