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ATRA
ATRA (all-trans retinoic acid) promotes differentiation of acute promyelocytic leukemia cells and induces apoptosis by binding to retinoic acid receptors.
ATRA (all-trans retinoic acid) induces differentiation and apoptosis of acute promyelocytic leukemia cells by binding to retinoic acid receptors and promoting maturation of abnormal myeloid precursors. Used for Acute promyelocytic leukemia (APL), particularly t(15;17) positive disease.
At a glance
| Generic name | ATRA |
|---|---|
| Also known as | all-trans retinoic acid, Tretinoin, Vesanoid®, All-trans retinoic acid, Vesanoid |
| Sponsor | South China Children's Leukemia Group |
| Drug class | Retinoid; differentiation agent |
| Target | Retinoic acid receptor (RAR), particularly RAR-alpha |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
ATRA is a retinoid that acts as a ligand for nuclear retinoic acid receptors (RAR and RXR), triggering transcriptional changes that force leukemic promyelocytes to differentiate into mature granulocytes rather than proliferate. This differentiation therapy approach, often combined with arsenic trioxide, has transformed acute promyelocytic leukemia (APL) from a highly fatal disease into one of the most curable acute leukemias.
Approved indications
- Acute promyelocytic leukemia (APL)
Common side effects
- Retinoic acid syndrome (RAS)
- Headache
- Fever
- Dry skin and mucous membranes
- Bone pain
- Elevated triglycerides
- Hepatotoxicity
Key clinical trials
- Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma (PHASE3)
- Lenalidomide and Dinutuximab With or Without Isotretinoin in Treating Younger Patients With Refractory or Recurrent Neuroblastoma (PHASE1)
- Metronomic Chemotherapy in Wilms Tumor (MetroWilms-1906) (PHASE1, PHASE2)
- A Study Comparing Tretinoin Gel Microsphere, 0.04% and RETIN-A MICRO ® Gel Microsphere, 0.04% in the Treatment of Acne Vulgaris (PHASE1)
- Isotretinoin With or Without Dinutuximab, Aldesleukin, and Sargramostim Following Stem Cell Transplant in Treating Patients With Neuroblastoma (PHASE3)
- Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) (PHASE3)
- 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)
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 CI brief — competitive landscape report
- ATRA updates RSS · CI watch RSS
- South China Children's Leukemia Group portfolio CI