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ATRA+Arsenic
ATRA (all-trans retinoic acid) induces differentiation of leukemic cells while arsenic trioxide causes apoptosis and degradation of the PML-RARA fusion protein, together achieving complete remission in acute promyelocytic leukemia.
ATRA (all-trans retinoic acid) induces differentiation of leukemic cells while arsenic trioxide causes apoptosis and degradation of the PML-RARA fusion protein, together achieving complete remission in acute promyelocytic leukemia. Used for Acute promyelocytic leukemia (APL), newly diagnosed and relapsed/refractory.
At a glance
| Generic name | ATRA+Arsenic |
|---|---|
| Also known as | retinoic acid + arsenic trioxide |
| Sponsor | Shanghai Jiao Tong University School of Medicine |
| Drug class | Retinoid and metalloid combination |
| Target | Retinoic acid receptor (RAR) and PML-RARA fusion protein |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
ATRA binds to retinoic acid receptors and promotes differentiation of abnormal promyelocytes into mature granulocytes. Arsenic trioxide induces apoptosis of leukemic cells and triggers degradation of the pathogenic PML-RARA fusion protein characteristic of APL. The synergistic combination achieves high cure rates with reduced toxicity compared to chemotherapy alone.
Approved indications
- Acute promyelocytic leukemia (APL), newly diagnosed and relapsed/refractory
Common side effects
- Differentiation syndrome (APL syndrome)
- Leukocytosis
- Hepatotoxicity
- QT prolongation
- Nausea and vomiting
- Headache
- Peripheral neuropathy
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)
- Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia (PHASE2)
- Venetoclax Combined With ATRA and ATO in Hyperleukocytic Acute Promyelocytic Leukemia (NA)
- Frontline Oral Arsenic Trioxide for APL (PHASE2)
- Oral Arsenic With ATRA for Newly Diagnosed Patients With Acute Promyelocytic Leukemia (PHASE2)
- Efficacy and Safety of ATO Plus ATRA in Nucleophosmin-1 Mutated Acute Myeloid Leukemia (PHASE1, PHASE2)
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 |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- ATRA+Arsenic CI brief — competitive landscape report
- ATRA+Arsenic updates RSS · CI watch RSS
- Shanghai Jiao Tong University School of Medicine portfolio CI