DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for familial rhabdoid tumor — 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 moduleFamilial rhabdoid tumor 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 familial rhabdoid tumor 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
SWI/SNF related BAF chromatin remodeling complex subunit B1 (SMARCB1) — SMARCB1 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 befdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7VDV · 3.4 Å · ligand BERYLLIUM TRIFLUORIDE ION (BEF). Experimental structure, not a prediction.
What the evidence adds up to
Familial rhabdoid tumours are driven by biallelic mutations in SMARCB1/INI1/hSNF5 in the majority of cases, with rare mutations in other SWI/SNF core members such as BRG1 also on record. The tumours are highly aggressive, have a predilection for infants and young children, and are often metastatic at presentation. In adults the disease is even rarer: a 2013 review notes that within the last decade prognosis has improved significantly, but at least 50% of patients still relapse and subsequently almost inevitably succumb. A 2022 case report of a 19-year-old African male with metastatic malignant extrarenal, extracranial rhabdoid tumour describes death within a year of diagnosis despite therapy.
No definitive chemotherapy regimen has been identified for this malignancy. The 2013 review summarises rationales for targeted therapy but does not report any clinical trial results showing efficacy. The 2022 case report states that despite identification of a candidate drug target, management remains a therapeutic challenge. No abstract reports a drug that improved survival or response rates in a controlled trial.
What is still missing is a validated molecular stratification that separates familial from sporadic cases, a clinical trial design that can enrol enough patients given the rarity of the disease, and funding to test candidate targeted agents in a setting where at least half of patients still die after relapse.
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 · 1987 · 52 citations
Malignant rhabdoid tumor of the extremity
AbstractTwo cases of malignant rhabdoid tumor (MRT) involving the sciatic nerve are described. Despite the close affiliation of the tumor with neural tissue, staining characteristics in these patients do not suggest a neural origin of MRT. Survival in patients with renal and extrarenal MRT has been poor. Our patients were treated with an aggressive chemotherapy program using cisplatin, Adriamycin (doxorubicin), vincristine, cyclophosphamide, actinomycin D, and DTIC. One child died of progressive disease; the other is well 35 months after diagnosis.
Pediatric Hematology and Oncology · 2013 · 35 citations
Rhabdoid Tumors: Clinical Approaches and Molecular Targets for Innovative Therapy
AbstractRhabdoid tumors are rare but highly aggressive tumors with a predilection for infants and young children. The majority of these tumors harbor biallelic mutations in SMARCB1/INI1/hSNF5. Rather rare cases with mutations in other SWI/SNF core members such as BRG1 are on record. Rhabdoid tumors have only recently been registered and treated according to specifically designed treatment recommendations and in the framework of clinical trials. Within the last decade, prognosis has improved significantly but at least 50% of patients still relapse and subsequently almost inevitably succumb to their disease. This review summarizes past and current clinical approaches and presents an overview of the rationales for targeted therapy with potential for future clinical treatment trials for rhabdoid tumors.
Boletín Médico del Hospital Infantil de México · 2023 · 2 citations · open access
Extrarenal rhabdoid tumor of anterior mediastinal location
AbstractBACKGROUND: Rhabdoid tumors are malignant neoplasms of low prevalence, aggressive behavior, and high mortality. They were initially described as renal tumors, although tumors with the same histopathological and immunohistochemical characteristics have been discovered in other locations, mainly in the central nervous system. Few cases of mediastinal location have been reported internationally. This work aimed to describe the case of a mediastinal rhabdoid tumor. CASE REPORT: We describe the case of an 8-month-old male patient admitted to the pediatric department with dysphonia and laryngeal stridor progressing to severe respiratory distress. Contrast-enhanced computed tomography of the thorax showed a large mass with homogeneous soft tissue density, and smooth and well-defined borders, with suspicion of malignant neoplasm. Due to the oncological emergency compressing the airway, empirical chemotherapy was initiated. Subsequently, the patient underwent incomplete tumor resection due to its invasive nature. The pathology report showed morphology compatible with a rhabdoid tumor, which immunohistochemical and genetic studies corroborated. Chemotherapy and radiotherapy to the mediastinum were administered. However, the patient died three months after the initial treatment due to the aggressive behavior of the tumor. CONCLUSIONS: Rhabdoid tumors are aggressive and malignant entities difficult to control and have poor survival. Early diagnosis and aggressive treatment are required, although the 5-year survival does not exceed 40%. It is necessary to analyze and report more similar cases to establish specific treatment guidelines.
Zenodo (CERN European Organization for Nuclear Research) · 2022 · 0 citations · open access
Soft Tissue Rhabdoid Tumour in a 19 Year Old African Male: A Case Report
AbstractRhabdoid Tumours (RT) is rare, rapidly progressive neoplasms that typically occur in childhood; are often metastatic at presentation and are known for their high mortality. These malignancies are even rarer in adults, occur at a variety of anatomic locations and are classified into three categories. A 19 year old male with no pre-existing illnesses or family history of malignancy, presented with metastatic malignant extrarenal, extracranial rhabdoid tumour (MERT) and after a short period of investigation and therapy demised within a year of his diagnosis. A definitive chemotherapy regimen is yet to be identified for this malignancy and despite the identification of a candidate drug target; the management of rhabdoid tumours remains a therapeutic challenge. Further study is required to underpin the molecular biology of this malignancy and with better understanding, targeted therapy will be discovered and applied; particularly for irresectable and metastatic disease.
Zenodo (CERN European Organization for Nuclear Research) · 2022 · 0 citations · open access
Soft Tissue Rhabdoid Tumour in a 19 Year Old African Male: A Case Report
AbstractRhabdoid Tumours (RT) is rare, rapidly progressive neoplasms that typically occur in childhood; are often metastatic at presentation and are known for their high mortality. These malignancies are even rarer in adults, occur at a variety of anatomic locations and are classified into three categories. A 19 year old male with no pre-existing illnesses or family history of malignancy, presented with metastatic malignant extrarenal, extracranial rhabdoid tumour (MERT) and after a short period of investigation and therapy demised within a year of his diagnosis. A definitive chemotherapy regimen is yet to be identified for this malignancy and despite the identification of a candidate drug target; the management of rhabdoid tumours remains a therapeutic challenge. Further study is required to underpin the molecular biology of this malignancy and with better understanding, targeted therapy will be discovered and applied; particularly for irresectable and metastatic disease.
IP International Journal of Ocular Oncology and Oculoplasty · 2023 · 0 citations · open access
Primary atypical rhabdoid orbital tumor: An entity with aggressive behavior
AbstractMalignant rhabdoid tumors are rare, poorly differentiated tumors which usually affect children under the age of three. These tumors have a predilection for the kidney, central nervous system and soft tissue. The definition classically relies on a characteristic morphology and the inactivation of the hSNF5/INI1 tumor suppressor gene. The diagnosis is based on radiological explorations, as well as anatomopathological and immuno-histochemical studies. Whatever the location of the tumor, the therapeutic protocol is only decided after multidisciplinary consultation meeting, while resorting to a triad of chemotherapy, surgery and radiotherapy. The prognosis remains poor and the survival rate is below 30%. We report a rare case of retro-orbital malignant rhabdoid tumor of a new born girl.
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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