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The retinoic acid receptor that, fused to PML in acute promyelocytic leukaemia, blocks white blood cells from maturing. All-trans retinoic acid releases the block and arsenic trioxide destroys the fusion protein, turning a once rapidly fatal leukaemia into one of the most curable. This dossier gathers the 2 products (2 approved), 4 trials, 0 pathways and 0 resistance routes in the corpus that involve it, with external identifiers so it can be joined to UniProt, ChEMBL, Open Targets and the rest of biology.
Ligand-activated nuclear receptor of myeloid differentiation; the PML::RARA fusion in APL is the drug target of tretinoin and arsenic trioxide.
| Cancer | Prevalence | Measure | Note | Source |
|---|---|---|---|---|
| Acute myeloid leukaemia | about 10% | PML::RARA fusion (acute promyelocytic leukaemia subtype) | PML::RARA is present in nearly all cases of acute promyelocytic leukaemia | doi.org |
Approximate, population-level figures; the measure column says what was counted. Ranges show the midpoint as a bar.
| Modality | Approved |
|---|---|
| Small molecule 2 |
| Trial | Setting | Result | Products | ||
|---|---|---|---|---|---|
COG ACNS0332 NCT00392327 | 3 | Mixed | Newly diagnosed high-risk medulloblastoma in children aged 3 to 21: craniospinal radiotherapy with weekly vincristine, with or without daily carboplatin, and with or without isotretinoin maintenance | Carboplatin radiosensitisation improved event-free and overall survival in group 3 high-risk medulloblastoma; isotretinoin maintenance was stopped for futility. | |
ANBL0531 NCT00499616 | 3 | Positive | Intermediate-risk neuroblastoma: response- and biology-based assignment to two, four or eight cycles of carboplatin, etoposide, cyclophosphamide and doxorubicin (with topotecan for poor responders) and surgery, with isotretinoin for the highest-risk subset and no chemotherapy for some stage 4S infants | Three-year event-free survival 83.2 percent and overall survival 94.9 percent with response- and biology-based reduction of chemotherapy; overall survival 100 percent for localised disease. | |
COG ANBL0032 NCT00026312 | 3 | Positive | High-risk neuroblastoma after transplant: ch14.18 (dinutuximab) + GM-CSF + IL-2 + isotretinoin vs isotretinoin alone | 2-year EFS 66% vs 46%; OS 86% vs 75%. | |
| 2 | - | A Phase II Study of Vemurafenib Combined With Acitretin in Patients With Advanced Melanoma | - |
No recorded escape route names this target.
No pathway diagram carries this target as a node.
No companion diagnostic in the registry measures this target.
No model entry for this target yet; check the cancer entries on the models page.
No open questions recorded for this target yet. Suggest one.
Query for this target: (TITLE:"Retinoic acid receptor alpha" OR ABSTRACT:"Retinoic acid receptor alpha" OR TITLE:"RARA" OR ABSTRACT:"RARA") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Retinoic acid receptor alpha (RARA), not a curated reading list.
The dossier as machine-readable JSON, at /api/v1/dossiers/rara.json: identifiers from HGNC, Ensembl, UniProt and ChEMBL, products with status, trials, pathways, hotspots, open questions and assays. The full entity record is in the open API at /api/v1/entities/rara.json. Licence CC BY-NC 4.0.