Future Developments: Novel Agents

Rapid increases in genomic insights into the molecular sub-structure of acute myeloid leukaemia (AML) have not been followed by similarly rapid improvements in therapeutic options, until recently. A swathe of new drug approvals by the FDA has resulted in a subset of drugs also receiving approval in the European Union (EMA). These drugs have been discussed elsewhere in this book. This chapter will focus on new, non-immune-based therapies which have potential to make a clinical impact for patients with AML in the near future. These agents and their targets include venetoclax (BCL-2), enasidenib (IDH2), ivosidenib (IDH1), glasdegib (Hedgehog), CC-486 (methylation), quizartinib (FLT3), APR-246 (mutant TP53), idasanutlin (MDM2) and pracinostat (HDAC). We aim to briefly summarise key clinical data in relation to these drugs, with a focus on resistance mechanisms and potential future strategies to overcome these evolutionary hurdles.