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ATO
Arsenic trioxide induces differentiation and apoptosis of leukemic cells, particularly in acute promyelocytic leukemia.
Arsenic trioxide induces differentiation and apoptosis of leukemic cells, particularly in acute promyelocytic leukemia. Used for Acute promyelocytic leukemia (APL), including newly diagnosed and relapsed/refractory disease.
At a glance
| Generic name | ATO |
|---|---|
| Also known as | As2O3, arsenic trioxide |
| Sponsor | South China Children's Leukemia Group |
| Drug class | Arsenic compound; differentiation agent |
| Target | PML-RARA fusion protein; mitochondrial apoptotic pathways |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
ATO works through multiple mechanisms including induction of cellular differentiation, triggering apoptosis via mitochondrial pathways, and generation of reactive oxygen species in leukemic blasts. It is particularly effective in acute promyelocytic leukemia (APL) where it targets the PML-RARA fusion protein, leading to degradation of the abnormal fusion protein and restoration of normal hematopoietic differentiation.
Approved indications
- Acute promyelocytic leukemia (APL), including newly diagnosed and relapsed/refractory disease
Common side effects
- Differentiation syndrome (APL differentiation syndrome)
- QT prolongation
- Leukocytosis
- Hepatotoxicity
- Neuropathy
- Hyperglycemia
Key clinical trials
- Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia (PHASE3)
- ATRA and SDK002 in Combination With Chemotherapy and Anti-PD-1 Inhibitor in Patients With Advanced Pancreatic Ductal Adenocarcinoma. (PHASE1)
- A Trial to Investigate Whether Oral Arsenic Trioxide Is Similar to Intravenous Arsenic Trioxide in Pharmacokinetics, Safety, and Efficacy (LATITUDE/SDKARS-301) (PHASE3)
- Endotype DIrected Treatment for OSA in Down Syndrome (PHASE4)
- French Registry of First-line Treatment of Acute Promyelocytic Leukemia
- Evaluate the Pharmacokinetics of Oral Arsenic Trioxide Solution Under Fasting and Fed Conditions, to Compare Intravenous Arsenic Trioxide, in Acute Promyelocytic Leukemia (PHASE1)
- Venetoclax Combined With ATRA and ATO in Hyperleukocytic Acute Promyelocytic Leukemia (NA)
- Effectiveness and Safety Study of Early add-on of Ezetimibe With Atorvastatin in Very High-risk Patients (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |