The Development and Clinical Applications of Oral Arsenic Trioxide for Acute Promyelocytic Leukaemia and Other Diseases

Appreciation of the properties of arsenic trioxide (ATO) has redefined the treatment landscape for acute promyelocytic leukaemia (APL) and offers promise as a treatment for numerous other diseases. The benefits of ATO in patients with APL is related to its ability to counteract the effects of PML::RARA, an oncoprotein that is invariably detected in the blood or bone marrow of affected individuals. The PML::RARA oncoprotein is degraded specifically by binding to ATO. Thus ATO, in combination with all-trans retinoic acid, has become the curative treatment for ATO. The multiple mechanisms of action of ATO has also paved the way for application in various condition encompassing autoimmune or inflammatory disorders, solid organ tumours, lymphomas and other subtypes of AML. The development of oral formulation of ATO (oral ATO) has reduced costs of treatment and improved treatment convenience allowing widespread applicability. In this review, we discuss the mechanisms of action of ATO, the development of oral ATO, and the applications of oral ATO in APL and other diseases.

Automated summary

Appreciation of the properties of arsenic trioxide (ATO) has revolutionized the treatment of acute promyelocytic leukaemia (APL) and holds promise for numerous other diseases. ATO's ability to counteract the effects of the oncoprotein PML::RARA, found in APL patients, has made it a curative treatment when combined with all-trans retinoic acid. Additionally, ATO's multiple mechanisms of action have opened up possibilities for its use in various autoimmune or inflammatory disorders, solid organ tumors, lymphomas, and other subtypes of AML. The development of oral ATO has improved treatment convenience and accessibility.