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The Long, Winding Road to Precision Medicine in AML

It is now almost six years since Drs Sekeres and Steensma ruefully described acute myeloid leukaemia (AML) as “the Boulevard of Broken Dreams”, reflecting the catalogue of failed attempts to develop novel treatments for this intractable disease.

While the treatment of many other cancers has been transformed by targeted medicines, AML has stubbornly refused to yield, and the basic principles of treatment have been little changed for decades. However, an increasing understanding of the molecular heterogeneity of AML has identified some of the previously unrecognized barriers to treatment success and has begun to clear a path towards the use of precision medicines for AML.

Here at SciMed, we have been privileged to work on several novel targeted drugs that have helped to transform disease outcomes for patients with a wide range of malignant blood diseases. We look forward with excitement to the results of the Beat AML Master Trial (NCT03013998), a phase 1b/2 umbrella investigation which seeks to match older patients with de novo AML to any of up to 12 treatments arms based on upfront genomic screening. We sincerely hope that the approval and advanced-stage development of an increasing number of targeted agents such as midostaurin (Novartis), CPX-351 (Jazz Pharma) and gilterinib (Astellas Inc) heralds the start of the demolition of the Boulevard of Broken Dreams, so that we can replace it with the Motorway of Precision Medicine! [May 2018]