DeCure for Central nervous system rhabdomyosarcoma
DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for central nervous system rhabdomyosarcoma — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCentral nervous system rhabdomyosarcoma maps to a 2-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 central nervous system rhabdomyosarcoma 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
dicer 1, ribonuclease III (DICER1) — DICER1 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7XW2 · 3.04 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
In a single institution series of 59 paediatric rhabdomyosarcoma patients, the prevalence of central nervous system involvement was 11.9% (7 patients), higher than earlier reports. Among the 6 deaths from disease, all had rapid progression, with a median survival of 14 days. The authors suggest the higher incidence could be secondary to treatment modifications or more sensitive detection. No effective intervention for CNS involvement is described in this series.
A separate case report describes a primary rhabdomyosarcoma arising from the filum terminale in a 48-year-old man, diagnosed by immunocytochemistry. The authors review 22 previously reported cases of primary CNS rhabdomyosarcoma and discuss a possible origin from neuroectoderm. No treatment outcomes or survival data are given for this case beyond the histological diagnosis.
In a 20-month-old child with neurofibromatosis type 1 and orbital embryonal rhabdomyosarcoma, the tumour regressed completely with chemotherapy and external beam radiotherapy. This single case demonstrates that orbital rhabdomyosarcoma can be associated with neurofibromatosis type 1, but it does not address CNS parenchymal involvement or the rapidly fatal course seen in the larger series.
What is still missing is any prospective trial designed specifically for CNS rhabdomyosarcoma, a reliable stratification of patients by molecular subtype or site of CNS involvement, and funding to move beyond single-institution retrospective reports and isolated case descriptions.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of Pediatric Hematology/Oncology · 2018 · 20 citations
Central Nervous System Involvement of Rhabdomyosarcoma: A Single Institution Experience
AbstractThe incidence of central nervous system (CNS) involvement in patients with rhabdomyosarcoma (RMS) is low, and the outcome is dismal. We present a single institution analysis of CNS involvement of pediatric RMS. In 59 patients, the prevalence of CNS involvement was 11.9% (7 patients), higher than prior reports. Of the 6 deaths from disease, all had rapid progression, with a median survival of 14 days. The higher incidence could be secondary to treatment modifications or more sensitive detection. These findings are useful for decisions at the time of CNS involvement and could lead to modifications for future RMS clinical trials.
Primary Rhabdomyosarcoma of the Central Nervous System: Case Report
AbstractA case of a primary rhabdomyosarcoma that appeared to be arising from the filum terminale in a 48-year-old man is reported. The histological diagnosis of rhabdomyosarcoma was supported using immunocytochemical methods. Twenty-two cases of primary rhabdomyosarcoma of the central nervous system previously reported in the literature are reviewed briefly. The possible origin of these tumors from neuroectoderm is discussed.
Ophthalmic Plastic and Reconstructive Surgery · 2007 · 9 citations
Orbital Embryonal Rhabdomyosarcoma in Association with Neurofibromatosis Type 1
AbstractIn Brief A 20-month-old child with systemic features of neurofibromatosis type 1 presented with sudden-onset proptosis. Biopsy and histopathology confirmed the diagnosis of an embryonal rhabdomyosarcoma. The tumor regressed completely with chemotherapy and external beam radiotherapy. This case highlights the association of rhabdomyosarcoma with neurofibromatosis type 1. Orbital rhabdomyosarcoma may be associated with neurofibromatosis type 1.
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.
