DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for rhabdomyosarcoma — screening already-approved drugs against its 44-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleRhabdomyosarcoma maps to a 44-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
approvedVincristineApproved drug
Structures already discussed alongside rhabdomyosarcoma in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
bovine ABCC1 — Vincristine has a real, experimentally solved structure in complex with this target (PDB 9LGC, 2.95 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet r1qdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9LGC · 2.95 Å · ligand Vincristine (R1Q). Experimental structure, not a prediction.
What the evidence adds up to
In a prospective series of 39 adults with rhabdomyosarcoma treated between 1973 and 1996, the overall 5‑year survival was 31% and the 10‑year survival 27%. Five‑year survival fell from 60% for tumours smaller than 5 cm to 14% for tumours 5–10 cm and 0% for tumours larger than 10 cm. Patients with localised or locoregional disease at presentation had a 44% 5‑year survival; no patient with metastatic disease survived five years. A complete response to chemotherapy gave a 57% 5‑year survival, compared with only 7% for poor responders. Metastatic disease at presentation and poor chemotherapy response were independent predictors of death. Age, tumour location, nodal status and histological subtype were not associated with survival in this adult cohort.
A 2010 review states that rhabdomyosarcoma, though a typical childhood tumour with high malignancy and a marked tendency to metastasise, generally responds well to chemotherapy and radiotherapy. Multimodal therapy is considered essential, but the intensity and combination of surgery, radiotherapy and chemotherapy must be selected according to patient risk groups. The review notes that open and debated issues remain, and that novel strategies are being considered for the near future.
A 2011 in vitro study of novel DNA‑binding drugs observes that chemotherapy protocols for rhabdomyosarcoma continue to rely on the same general cytotoxic compounds regardless of tumour subtype, progression or stage. These agents, such as vincristine, actinomycin D and cyclophosphamide, depend on rapid tumour‑cell proliferation for selectivity. Other agents used in clinical trials include topoisomerase poisons (etoposide, doxorubicin, epirubicin, topotecan, irinotecan) and alkylating agents (ifosfamide, carboplatin). The study notes that no agents targeting specific molecular pathways, such as the PAX3‑FKHR chimeric protein, are yet in routine use.
What remains missing is a prospective trial designed specifically for adults, given that the 2001 series spans 23 years and includes only 39 patients. No targeted therapy has been validated against the PAX3‑FKHR fusion or other molecular drivers. Stratification by tumour size and chemotherapy response is possible, but no funding or trial design has yet addressed the adult population separately from paediatric protocols.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Annals of Surgery · 2001 · 167 citations · open access
Response to Chemotherapy and Predictors of Survival in Adult Rhabdomyosarcoma
AbstractOBJECTIVE: To assess outcome and identify predictors of survival of adults with rhabdomyosarcoma. SUMMARY BACKGROUND DATA: The literature on adult rhabdomyosarcoma is limited. Few studies have identified predictors of long-term survival in this patient population. METHODS: Thirty-nine adults with rhabdomyosarcoma were treated between 1973 and 1996 and prospectively followed. Outcomes were assessed with respect to patient and tumor characteristics, local treatment, and response to chemotherapy. RESULTS: Twenty-six patients had localized/locoregional disease and 13 patients had metastatic disease at presentation. Twenty-one patients underwent attempted curative resection, 27 received radiotherapy, and 37 received chemotherapy. Median follow-up for surviving patients was 152 months. The overall 5- and 10-year survival rates were 31% and 27%, respectively. Five-year survival rates for patients with tumors less than 5 cm, 5 to 10 cm, and more than 10 cm were 60%, 14%, and 0%, respectively. Patients with localized/locoregional disease at presentation had a 44% 5-year survival rate; there were no 5-year survivors among patients with metastatic disease. Patients who had a complete response to chemotherapy had a 5-year survival rate of 57%, compared with a rate of only 7% for poor responders. Metastatic disease at presentation and poor response to chemotherapy were independent predictors of death on multivariate analysis. CONCLUSIONS: Age, location, nodal status, and histologic subtype do not appear be associated with survival in adults with rhabdomyosarcoma treated with multimodal therapy. Metastatic disease at presentation and poor response to chemotherapy are strongly associated with poor prognosis. Future systemic therapies should be targeted to patients with localized/locoregional disease and partial responders to conventional chemotherapy.
Expert Review of Anticancer Therapy · 2010 · 50 citations
Selecting multimodal therapy for rhabdomyosarcoma
AbstractRhabdomyosarcoma is a typical tumor of childhood, characterized by a high grade of malignancy, local invasiveness and a marked propensity to metastasize, but also a generally good response to chemotherapy and radiotherapy. Multimodal therapy is essential to cure rhabdomyosarcoma patients, but different uses of surgery, radiotherapy and chemotherapy, and their intensity, need to be selected and modulated to different patient risk groups. This article attempts to give an account of the current treatment options, the open and debated issues and the potential novel strategies for the near future.
Considerations for Treatment Development in Rhabdomyosarcoma: In Vitro Assessment of Novel DNA Binding Drugs
AbstractSoft Tissue Tumors 4 without relapse and despite this drastic difference in tumour response, chemotherapy protocols continue to utilize the same compounds regardless of tumour subtype, progression or stage Without agents to target specific molecular pathways and proteins of RMS, such as the PAX3-FKHR chimeric protein, chemotherapy protocols continue to utilize general cytotoxic compounds that rely on the rapid proliferation of tumour cells for selectivity and optimal efficacy. Most of these agents bind to DNA and disrupt key molecular processes involved in DNA transcription and replication. Treatment usually involves the vinca alkaloid vincristine, the transcription inhibitor actinomycin D and the alkylating prodrug cyclophosphamide Several other general cytotoxic agents, including the topoisomerase poisons etoposide, doxorubicin, epirubicin, topotecan and irinotecan as well as the alkylating agents ifosfamide and carboplatin have also been used in alternative treatment protocols and large scale clinical trials (Table Many agents included in RMS clinical trials and standard treatment protocols can be broadly classified as general cytotoxic agents, a large proportion, including etoposide, doxorubicin and topotecan, specifically target and poison the function of the topoisomerase enzymes, whilst actinomycin D is a transcription inhibitor that has been successful in the treatment of a wide variety of tumours, including RMS.
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.
