Drug intelligence / Profile preview

Arsenol

Development stage
Phase 1
Lead developer
The University of Hong Kong
Modality
Small Molecules
Administration
Oral
01

Overview

Arsenol is an oral formulation of arsenic trioxide (ATO) developed by The University of Hong Kong. While arsenic trioxide is a well-established treatment for acute promyelocytic leukemia (APL), Arsenol is specifically being investigated for its efficacy in other myeloid malignancies, particularly those harboring TP53 mutations which are typically associated with poor prognosis and resistance to standard chemotherapy. The drug's mechanism of action involves the induction of apoptosis, generation of reactive oxygen species (ROS), and inhibition of thioredoxin reductase, leading to cell cycle arrest and the degradation of specific oncogenic proteins. In clinical settings, Arsenol is often studied in combination with ascorbic acid and low-intensity therapies such as hypomethylating agents (azacitidine or decitabine) and venetoclax to enhance its pro-oxidant effects and therapeutic reach in patients with acute myeloid leukemia (AML), myelodysplastic neoplasms (MDS), and chronic myelomonocytic leukemia (CMML).

Brand names
Arsenol
Other names
oral arsenic trioxide
02

Targets

PML-RARA (PML-retinoic acid receptor alpha fusion protein)TXNRD2 (Thioredoxin reductase 2)

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