# Arsenic trioxide

Source: https://onco.cc/drugs/arsenic-trioxide/  
OnCo record `arsenic-trioxide` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

An ancient poison turned cure: with retinoic acid it cures more than 95% of acute promyelocytic leukaemia without conventional chemotherapy.

## Summary

Developed from traditional Chinese medicine in Harbin (1990s); approved 2000 for relapsed APL and 2018 first line with tretinoin after APL0406 (Lo-Coco, NEJM 2013) showed ATRA-ATO superior to ATRA-chemotherapy in standard-risk APL. Toxicities: differentiation syndrome, QT prolongation, hepatotoxicity. Oral arsenic (Realgar-Indigo naturalis) is used in China.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-08
- Tags: gap-fill
- Brand: Trisenox
- Modality: Inorganic arsenical (differentiation agent)
- Mechanism: Binds PML moiety of PML-RARA, inducing SUMOylation and degradation of the fusion oncoprotein; triggers differentiation and apoptosis of APL blasts.
- Approvals: US 2000: Relapsed/refractory APL; US 2018: Newly diagnosed low-risk APL with tretinoin; EU 2002: APL

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Arsenic_trioxide
- APL0406 (NEJM 2013): https://doi.org/10.1056/NEJMoa1300874
- Label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=arsenic%20trioxide

## Connected records

- technologies: [Epigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)](https://onco.cc/technologies/epigenetic-drugs/), [Traditional Chinese herbal medicine alongside treatment](https://onco.cc/technologies/traditional-chinese-herbal-medicine/)
- cancers: [Acute myeloid leukaemia](https://onco.cc/cancers/aml/), [Acute promyelocytic leukaemia](https://onco.cc/cancers/apl/)
- companies: [Teva Pharmaceuticals](https://onco.cc/companies/teva/)
- terms: [Cancer stem cell theory and phenotypic plasticity](https://onco.cc/terms/cancer-stem-cell-theory/), [Differentiation syndrome](https://onco.cc/terms/differentiation-syndrome/), [QT prolongation](https://onco.cc/terms/qt-prolongation/)
- targets: [PML](https://onco.cc/targets/pml/), [Retinoic acid receptor alpha (RARA)](https://onco.cc/targets/rara/), [Thioredoxin reductase 1](https://onco.cc/targets/txnrd1/)
- people: [Saijuan Chen](https://onco.cc/people/chen-saijuan/), [Zhu Chen](https://onco.cc/people/chen-zhu/)
- key papers: [APL0406: retinoic acid plus arsenic trioxide without chemotherapy for low- and intermediate-risk acute promyelocytic leukaemia](https://onco.cc/key-papers/paper-apl0406-lo-coco-nejm-2013/)
- institutions: [Ruijin Hospital, Shanghai Jiao Tong University](https://onco.cc/institutions/ruijin-hospital/)
- pathways: [Acute myeloid leukaemia (KEGG map)](https://onco.cc/pathways/aml-signalling/)
- trials: [APL0406](https://onco.cc/trials/apl0406/)

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