Abstract
Acute myeloid leukemia (AML) is a clinically and genetically heterogenous malignancy of myeloid progenitor cells that affects patients of all ages. Despite decades of research and improvement in overall outcomes, standard therapy remains ineffective for certain subtypes of AML. Current treatment is intensive and leads to a number of secondary effects with varying results by patient population. Due to the high cost of discovery and an unmet need for new targeted therapies that are well tolerated, alternative drug development strategies have become increasingly attractive. Repurposing existing drugs is one approach to identify new therapies with fewer financial and regulatory hurdles. In this review, we provide an overview of previously U.S. Food and Drug Administration (FDA) approved non-chemotherapy drugs under investigation for the treatment of AML.
Highlights
Drug repurposing is the practice of finding new applications for existing drugs outside their initial approval
A randomized phase III study assessing the safety and efficacy of combining valproic acid (VPA) and all-trans retinoic acid (ATRA) with induction therapy for the treatment of elderly Acute myeloid leukemia (AML) patients was terminated early due to lack of clinical improvement in the VPA group compared to standard treatment [70]
cardiac glycosides (CGDs) decreased the viability of AML leukemic stem cells (LSCs) in vitro at a greater sensitivity than normal hematopoietic stem cells [94]
Summary
Drug repurposing is the practice of finding new applications for existing drugs outside their initial approval. Repurposed drugs still need to undergo clinical testing for a new indication, the development timeline can be accelerated with less setbacks and lower cost than traditional drug development. AML affects patients of all ages and poor prognosis is associated with older age and complex cytogenetics Even for those who respond favorably to treatment, relapse is common and leads to poor outcomes. High demand exists for new therapies that are more effective, less toxic and better positioned to target the critical molecular pathways in AML leukemogenesis. 2 (IDH2) inhibitor enasidenib [12], all address specific features or subtypes of AML These drugs provide credence to using AML disease pathogenesis to inform the development of compounds with activity against precise molecular aberrations.
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