Cancer Lab · DeCure for X

DeCure for Alveolar rhabdomyosarcoma

DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for alveolar rhabdomyosarcoma — screening already-approved drugs against its 47-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module47 genesLead labCancer
All cures
CancerDOID:4051$DeCureCancer

The disease map

Disease moduleAlveolar rhabdomyosarcoma maps to a 47-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 alveolar 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

Two serial case reports describe adult patients with alveolar rhabdomyosarcoma of the extremity who received only subtotal excision or incision biopsy, followed by six cycles of anthracycline-based chemotherapy and then 70 Gy radiotherapy to the local tumour plus 50 Gy prophylactic radiotherapy to regional lymph nodes. Post-radiotherapy contrast CT showed no residual tumour mass, classified as complete response. The sample is two patients, and no follow-up duration or survival data are given.

A separate case report describes a 27-year-old woman with a highly destructive facial lesion diagnosed as alveolar rhabdomyosarcoma on histopathology. The report provides no treatment details or outcome.

A third case involves a 20-month-old boy with recurrent, fusion-positive alveolar rhabdomyosarcoma without metastasis. He had previously achieved complete response with multiagent chemotherapy and external beam irradiation. At relapse he received further chemotherapy, orbital exenteration, and intraoperative brachytherapy. The authors note that customised brachytherapy may limit the sequelae of radiation, but the report gives no survival or recurrence data for this single patient.

Across these three reports the total number of patients is four. No controlled trial, no consistent regimen, and no long-term outcomes are reported. What is missing is any prospective trial with a defined sample size, standardised chemotherapy backbone, stratification by fusion status or age, and sufficient follow-up to measure progression-free or overall survival. Funding for such a trial and a multi-centre collaborative structure would be needed to move beyond single-case observations.

Evidence

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

International Journal of Applied and Basic Medical Research · 2019 · 4 citations · open access

Adult alveolar rhabdomyosarcoma on extremity, successful treatment with radiotherapy following chemotherapy: Serial case report

AbstractAlveolar rhabdomyosarcoma (RMS) is one of the four subtypes of RMS. Alveolar RMS is the rare type found in adults, with the worse prognosis. We report 2 serial cases of alveolar RMS on extremity which was only treated with subtotal excision or incision biopsy. We further gave a total dose of 70 Gy radiotherapy on the local tumor and 50 Gy prophylaxis dose on regional lymph node after 6 cycles of anthracyclines-based chemotherapy. Postradiotherapy, contrast computed tomography scan revealed no tumor mass left (complete response).

https://doi.org/10.4103/ijabmr.ijabmr_100_18
Southern Medical Journal · 2008 · 3 citations

Rhabdomyosarcoma Presenting With Destructive Large Lesion of the Face

AbstractRhabdomyosarcoma (RMS) is the most common soft tissue sarcoma in children and adolescents. RMS may be detected anywhere in the body, although the head and neck are the most involved areas. Prognostic factors of RMS include tumor status, primary tumor site, localization in the organ or tissue of origin, patient age and histopathological type. Alveolar histologic type is more aggressive than the other types and is seen in most patients with locally advanced and metastatic disease. A 27-year-old woman who was admitted to our clinic with a highly destructive lesion on her face is presented. She was diagnosed with alveolar rhabdomyosarcoma on histopathological examination.

https://doi.org/10.1097/smj.0b013e31815d26c0
Plastic & Reconstructive Surgery Global Open · 2022 · 3 citations · open access

Orbital Exenteration and Brachytherapy for the Treatment of Pediatric, Fusion-positive, Recurrent Rhabdomyosarcoma

AbstractA 20-month-old boy presented with biopsy-proven recurrent alveolar rhabdomyosarcoma without metastasis. He was previously treated with multiagent chemotherapy and external beam irradiation showing a complete response. Upon relapse, he was treated with chemotherapy, orbital exenteration, and brachytherapy. Customized, intraoperative brachytherapy has potential to limit the sequelae associated with radiation adjuvant therapy.

https://doi.org/10.1097/gox.0000000000004581
Rhinology online · 2018 · 1 citations · open access

Multidisciplinary approach of a locally advanced adult alveolar rhabdomyosarcoma of paranasal sinuses: a case report and literature review

AbstractAlveolar rhabdomyosarcoma (ARMS) is a rare soft-tissue malignancy constituting less than 1% of soft-tissue sarcomas. In this article we are describing a rare case of ARMS arising in the paranasal sinuses of an adult patient. We emphasize the multidisciplinary treatment administered, thanks to which the patient remains alive and free of disease for six years after the initial diagnosis

https://doi.org/10.4193/rhinol/18.034

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.