DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for orbit embryonal rhabdomyosarcoma — screening already-approved drugs against its 16-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleOrbit embryonal rhabdomyosarcoma maps to a 16-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 orbit embryonal 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
fibroblast growth factor receptor 4 (FGFR4) — FGFR4 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~{r}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8KH8 · 1.49 Å · ligand 1-[4-[(1~{R})-1-[3,5-bis(chloranyl)pyridin-4-yl]ethoxy]-5-cyano-pyridin-2-yl]-3-[6-methanoyl-5-[(4-methyl-2-oxidanylidene-piperazin-1-yl)methyl]-3-(2-morpholin-4-ylethoxy)pyridin-2-yl]urea (VVW). Experimental structure, not a prediction.
What the evidence adds up to
A population-based study of 102 patients with orbital embryonal rhabdomyosarcoma diagnosed between 1988 and 2012 reported a median age of 6 years, with 78.4% white and 56.9% male. Median tumour size was 30 mm. Among 92 patients with known surgical and radiotherapy status, 54.3% received both surgery and radiotherapy, 39.1% received primary radiotherapy, and 6.5% received primary surgery alone. Ninety-three percent of the entire cohort received chemotherapy. The 5- and 10-year cause-specific survival rates were 94.3% and 92.2%, respectively; overall survival at 5 and 10 years was 93.3% and 91.3%. In the 95 patients followed for at least 12 months, no significant prognostic factors for cause-specific or overall survival were identified, and the local treatment strategy (surgery versus radiotherapy) did not significantly affect either outcome (P=0.29 for cause-specific survival, P=0.468 for overall survival). The authors concluded there is no local treatment of choice in terms of survival, though radiotherapy is a reasonable alternative to surgery.
An experimental study using transplanted tumours from a paratesticular embryonal rhabdomyosarcoma found no significant difference in therapeutic effect between a combination chemotherapy of vincristine, actinomycin D and cyclophosphamide (the VAC regimen) and radiotherapy alone. Morphologic analysis suggested VAC is effective when undifferentiated rhabdomyoblasts predominate, while radiotherapy is preferable for tumours containing variously differentiated rhabdomyoblasts. This work is from 1989 and used a paratesticular model, not orbital disease.
A 2023 case series of 8 paediatric orbital rhabdomyosarcomas stated that prognosis has improved due to progress in chemotherapy and new radiotherapy techniques, but presented no survival or response rate data. The study aimed to report clinical and therapeutic characteristics and treatment complications.
What is still missing is prospective randomised data comparing surgery and radiotherapy for orbital embryonal rhabdomyosarcoma, given the population-based study found no survival difference between strategies. The experimental data suggesting histology-based treatment selection (VAC versus radiotherapy) has not been validated in orbital disease. No trial has stratified patients by differentiation grade or molecular subtype. Funding for a multi-centre trial large enough to detect survival differences in this rare tumour remains absent.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Cancer Management and Research · 2018 · 13 citations · open access
The prognosis and effects of local treatment strategies for orbital embryonal rhabdomyosarcoma: a population-based study
AbstractIntroduction: Orbital embryonal rhabdomyosarcoma is a rare childhood malignancy with a good prognosis, but the optimal treatment remains unclear. Using a population-based cancer registry, we assessed the prognoses and survival outcomes of patients with orbital embryonal rhabdomyosarcoma according to the local treatment strategy. Patients and methods: Patients diagnosed with orbital embryonal rhabdomyosarcoma between 1988 and 2012 as part of the Surveillance Epidemiology and End Results program were included. Univariate and multivariate Cox regression analyses were performed to determine the prognostic factors associated with cause-specific survival (CSS) and overall survival (OS). Results: In total, 102 patients were included; their median age was 6 years, 78.4% were white, and 56.9% were male. The median tumor size was 30 mm. Of 20 patients with an available histologic grade, the tumors of 90% were poorly differentiated/undifferentiated. Of 92 patients with available surgical and radiotherapy (RT) statuses, 50 (54.3%), 36 (39.1%), and 6 (6.5%) received surgery and RT, primary RT, and primary surgery, respectively. Ninety-five patients (93.1%) received chemotherapy. The 5- and 10-year CSSs of the entire cohort were 94.3% and 92.2%, respectively. The 5- and 10-year OSs were 93.3% and 91.3%, respectively. In 95 patients who were followed up for at least 12 months, there were no significant prognostic factors related to CSS and OS. Furthermore, the local treatment strategy did not significantly affect CSS ( P =0.29) or OS ( P =0.468). Conclusion: There is no local treatment of choice for orbital embryonal rhabdomyosarcoma in terms of survival. However, RT is a reasonable alternative treatment to surgery. Keywords: orbital embryonal rhabdomyosarcoma, survival, radiotherapy, surgery, SEER
Experimental Chemotherapy and Radiotherapy to Paratesticular Rhabdomyosarcoma
AbstractExperimental chemotherapy and radiotherapy were tried in transplanted tumors derived from a paratesticular embryonal rhabdomyosarcoma. There was no significant difference on the therapeutic effect between a combination chemotherapy composed of vincristine, actinomycin D and cyclophosphamide, so-called "VAC" regimen, and a single therapy of radiation. However, morphologic analyses suggest that VAC is effective in embryonal rhabdomyosarcomas in which undifferentiated rhabdomyoblasts predominate, while radiotherapy is preferable for those containing variously differentiated rhabdomyoblasts.
Zenodo (CERN European Organization for Nuclear Research) · 2023 · 0 citations · open access
MULTIMODAL TREATMENT IN PEDIATRIC ORBITAL RHABDOMYOSARCOMA: ABOUT 8 CASES
AbstractRhabdomyosarcoma is the most frequent mesenchymal tumor in children, composed of cells with histopathological characteristics of striated muscle at different stages of embryogenesis. The orbital location accounts for about 10% of cases. Its prognosis has improved thanks to the progress of chemotherapy and new radiotherapy techniques.The aim of our study is to report the clinical and therapeutic characteristics of rhabdomyosarcomas of the orbit in children, and to present different complications of the treatment.
Zenodo (CERN European Organization for Nuclear Research) · 2023 · 0 citations · open access
MULTIMODAL TREATMENT IN PEDIATRIC ORBITAL RHABDOMYOSARCOMA: ABOUT 8 CASES
AbstractRhabdomyosarcoma is the most frequent mesenchymal tumor in children, composed of cells with histopathological characteristics of striated muscle at different stages of embryogenesis. The orbital location accounts for about 10% of cases. Its prognosis has improved thanks to the progress of chemotherapy and new radiotherapy techniques.The aim of our study is to report the clinical and therapeutic characteristics of rhabdomyosarcomas of the orbit in children, and to present different complications of the treatment.
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.
