Cancer Lab · DeCure for X

DeCure for Acute promyelocytic leukemia

DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for acute promyelocytic leukemia — screening already-approved drugs against its 24-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module24 genesLead labCancer
All cures
CancerDOID:0060318$DeCureCancer

The disease map

Disease moduleAcute promyelocytic leukemia maps to a 24-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.

Research record

01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash

Current lead

No approved-drug candidate for acute promyelocytic leukemia is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.

Molecular view

retinoic acid receptor gamma (RARG)RARG is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.

Loading structure…
helix sheet 1-adamantyldrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 6FX0 · 1.9 Å · ligand 6-[3-(1-adamantyl)-4-oxidanyl-phenyl]naphthalene-2-carboxylic acid (E9T). Experimental structure, not a prediction.

What the evidence adds up to

The PML protein is widely regarded as a tumour suppressor in the scientific literature, but a 2013 review systematically questions whether the experimental evidence for this assumption is unambiguous. The review notes that the role of PML has been tested in many contexts, yet does not present new clinical data; it examines how the concept of a tumour-suppressor gene has evolved and whether PML meets that standard.

A 2013 clinical trial by Lo-Coco et al, reported in the New England Journal of Medicine, treated low-intermediate risk acute promyelocytic leukaemia (APL) patients with retinoic acid plus arsenic trioxide and no DNA-damaging chemotherapy. The outcome was very favourable for virtually all enrolled patients. The authors describe this as a practice-changing study that opens a new era in leukaemia therapy, confirming results from earlier small pilot studies. A 2006 review notes that the outcome of APL has improved dramatically over the previous 40 years, with insights into genetic and biologic mechanisms leading to specific and effective strategies that transformed APL from one of the most lethal leukaemias to one that is highly curable.

For children with APL, a 2014 review states that treatment includes induced remission, consolidation therapy and maintenance therapy. Prognosis is related to age at onset, initial white blood cell count, negativity of the PML-RARa fusion gene, and mutation rates of the fms-like tyrosine kinase 3 receptor gene. No specific survival or response rates are given for the paediatric population in these abstracts.

What remains missing is a clear demonstration that PML itself acts as a tumour suppressor in humans, rather than simply being a fusion partner in the PML-RARa translocation. The paediatric data lack the large, controlled trials that have been conducted in adults, and no abstract provides long-term follow-up or stratification by the prognostic factors listed. Funding for such paediatric trials and for mechanistic studies of PML function outside the fusion protein is still needed.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Frontiers in Oncology · 2013 · 32 citations · open access

Is PML a Tumor Suppressor?

AbstractThe role of the promyelocytic leukemia (PML) protein has been widely tested in many different contexts, as attested by the hundreds of papers present in the literature. In most of these studies, PML is regarded as a tumor suppressor, a notion on the whole accepted by the scientific community. In this review, we examine how the concept of tumor-suppressor gene has evolved until now and then systematically assess whether this assumption for PML is supported by unambiguous experimental evidence.

https://doi.org/10.3389/fonc.2013.00174
Blood · 2013 · 18 citations · open access

Retinoic acid plus arsenic trioxide, the ultimate panacea for acute promyelocytic leukemia?

AbstractRarely in the field of cancer treatment did we experience as many surprises as with acute promyelocytic leukemia (APL). Yet, the latest clinical trial reported by Lo-Coco et al in the New England Journal of Medicine is a practice-changing study, as it reports a very favorable outcome of virtually all enrolled low-intermediate risk patients with APL without any DNA-damaging chemotherapy. Although predicted from previous small pilot studies, these elegant and stringently controlled results open a new era in leukemia therapy.

https://doi.org/10.1182/blood-2013-06-505115
Journal of the National Comprehensive Cancer Network · 2006 · 9 citations · open access

Strategies for the Treatment of Acute Promyelocytic Leukemia

AbstractThe outcome of acute promyelocytic leukemia (APL) has improved dramatically during the past 40 years. Insights into the genetic and biologic mechanisms of APL lead to the development of specific and effective therapeutic strategies. This article discusses the therapeutic interventions that transformed APL from one of the most lethal leukemias to one that is highly curable.

https://doi.org/10.6004/jnccn.2006.0005
Zhonghua shiyong erke linchuang zazhi · 2014 · 0 citations

Recent advances in the management and prognosis of childhood acute promyelocytic leukemia

AbstractTreatment options for children with acute promyelocytic leukemia include induced remission, conso-lidation therapy and maintenance therapy.The prognosis is related to the age of onset, initial white blood cell count in peripheral blood, negative situation of PML-RARa fusion gene and mutation rates of fms like tyrosine kinase 3 receptor gene. Key words: Acute promyelocytic leukemia; Treatment; Prognosis; Child

https://doi.org/10.3760/cma.j.issn.2095-428x.2014.03.004

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.

DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.