DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for embryonal 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 moduleEmbryonal 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 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 1975 case report describes an eight-year survival in a child with embryonal rhabdomyosarcoma of the testis after aggressive multimodal therapy, but the authors acknowledge that the low incidence of the disease prevents any single institution from producing meaningful data on effective treatment. A 2023 case report of prostatic embryonal rhabdomyosarcoma in a 35-year-old man states that the tumour is rare, aggressive, and carries a poor prognosis, with distant metastasis common at presentation and low survival rates in adults.
A 2018 population-based study of 102 orbital embryonal rhabdomyosarcoma patients (median age 6 years, 56.9% male) reported 5-year and 10-year cause-specific survival of 94.3% and 92.2%, respectively, and overall survival of 93.3% and 91.2%. Of 92 patients with known treatment, 54.3% received surgery and radiotherapy, 39.1% received primary radiotherapy, and 6.5% received primary surgery; 93.1% received chemotherapy. The study found no significant prognostic factors for cause-specific or overall survival, and the local treatment strategy (surgery versus radiotherapy) did not significantly affect either outcome. The authors conclude that there is no local treatment of choice for orbital embryonal rhabdomyosarcoma in terms of survival.
A 1989 experimental study on transplanted paratesticular embryonal rhabdomyosarcoma found no significant difference in therapeutic effect between the VAC regimen (vincristine, actinomycin D, cyclophosphamide) and radiotherapy alone. Morphologic analysis suggested VAC is effective when undifferentiated rhabdomyoblasts predominate, while radiotherapy is preferable for tumours containing variously differentiated rhabdomyoblasts. What remains missing are prospective trials large enough to stratify patients by anatomic site, histologic subtype, and stage, as well as funding to overcome the rarity of the disease and the difficulty of enrolling adequate numbers of patients.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The Journal of Urology · 1975 · 15 citations
Case Report of Embryonal Rhabdomyosarcoma of the Testis with an 8-Year Survival
AbstractThe low incidence of embryonal rhabdomyosarcoma prevents any one person or institution from presenting any meaningful data in the form of effective treatment based on clinical experience. We realize that this is an antidotal case but the stakes are high when treating any malignancy of the young. Therefore, we advocate early and aggressive therapy using all modalities of treatments, and cite this case which initially seemed hopeless but to date has all the hallmarks of a cure.
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
International Journal of Surgery Case Reports · 2023 · 4 citations · open access
Embryonal rhabdomyosarcoma of the prostate in a young male: A rare case report
AbstractINTRODUCTION AND IMPORTANCE: Embryonal rhabdomyosarcoma (ERMS) of the prostate is a rare disease, and it has a poor prognosis. Mostly patient come with late stage thus with delayed diagnosis and worse outcomes. CASE PRESENTATION: a 35-year-old Bangladeshi male presented with bladder outlet obstruction in an intermittent pattern. Patient was not complaining from any other urological symptoms, there was no hematuria or dysuria, even no constitutional symptoms, no history of weight loss, no anorexia or night rigors, also there was no history of fever. Patients have an only previous history of spinal screw without any other medical illnesses. Patient came to casualty with frequent visits without any inflammatory markers elevation even with normal biochemical labs. O/E he found to have a prostatic mass on PR examination of which patient admitted to urology department and underwent diagnostic cystoscopy which showed cauliflower mass at the prostatic urethra. Biopsies retrieved and showed embryonal rhabdomyosarcoma (RMS) of the prostate under the histopathological examination. CLINICAL DISCUSSION: Embryonal rhabdomyosarcoma (ERMS) mostly care a poor prognosis and modalities vary according to the presentation and the stage. However distant metastasis is common at the time of presentation. CONCLUSION: embryonal rhabdomyosarcoma of the prostate is a rare and aggressive tumor with a low survival rate in adults. However, more clinical data needed for a better outcome.
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.
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.
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