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All trans-retinoic acid
All-trans-retinoic acid (ATRA) binds to retinoic acid receptors to promote differentiation of acute promyelocytic leukemia cells and induce apoptosis of leukemic blasts.
All-trans-retinoic acid (ATRA) binds to retinoic acid receptors to promote differentiation of acute promyelocytic leukemia cells and induce apoptosis of leukemic blasts. Used for Acute promyelocytic leukemia (APL), particularly t(15;17) positive disease.
At a glance
| Generic name | All trans-retinoic acid |
|---|---|
| Also known as | Vesanoid |
| Sponsor | Mahmoud Ramadan mohamed Elkazzaz |
| Drug class | Retinoid; differentiation agent |
| Target | Retinoic acid receptor-alpha (RAR-alpha) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
ATRA is a vitamin A derivative that activates retinoic acid receptors (RARs), particularly RAR-alpha, which are abnormally fused to the PML gene in acute promyelocytic leukemia (APL). By binding these receptors, ATRA triggers degradation of the PML-RARA fusion protein, restoring normal differentiation pathways and causing leukemic cells to mature and undergo programmed cell death rather than proliferate.
Approved indications
- Acute promyelocytic leukemia (APL), particularly t(15;17) positive disease
Common side effects
- Retinoid acid syndrome (RAS) / differentiation syndrome
- Headache
- Fever
- Dry skin and mucous membranes
- Bone pain
- Elevated triglycerides
- Hepatotoxicity
- Pseudotumor cerebri
Key clinical trials
- All-Trans Retinoic Acid (ATRA) Plus PD-1 Inhibition in Recurrent IDH-Mutant Glioma (PHASE2)
- Tretinoin and Arsenic Trioxide in Treating Patients With Untreated Acute Promyelocytic Leukemia (PHASE3)
- All-Trans Retinoic Acid (ATRA) and Atezolizumab for the Treatment of Recurrent or Metastatic Non-Small Cell Lung Cancer (PHASE1)
- A Phase II Study of the Combination of Pembrolizumab and ATRA Combination Treatment of Relapsed HL and B-NHL (PHASE2)
- Drug Screening Using Novel IMD in Salivary and Head and Neck Cancers (EARLY_PHASE1)
- A Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma (PHASE2)
- 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)
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:
- All trans-retinoic acid CI brief — competitive landscape report
- All trans-retinoic acid updates RSS · CI watch RSS
- Mahmoud Ramadan mohamed Elkazzaz portfolio CI