DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for brain sarcoma — screening already-approved drugs against its 43-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleBrain sarcoma maps to a 43-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 brain sarcoma 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
Brain sarcoma is a heterogeneous group of rare primary intracranial tumours, and the available literature consists almost entirely of single case reports and small retrospective series. A systematic review of primary intracranial DICER1-mutant sarcoma covering 2000 to 2024 identified only eight studies with ten patients, mean age 15.9 years, with headaches and seizures the most common presenting symptoms. Gross total resection was achieved in half of the cases, and five patients (50%) had stable disease after surgery plus adjuvant radiotherapy and chemotherapy, with a mean follow-up of 17.5 months. The authors describe the tumour as aggressive with non-specific clinical features, and note that large-scale trials are needed to develop optimised protocols.
Other subtypes show equally sparse and often discouraging data. A 2012 case of primary central nervous system histiocytic sarcoma in a 55-year-old woman progressed despite radiotherapy, with consciousness deteriorating within two months; the report states there are no effective therapeutic methods and the prognosis is very poor. A 2019 case of CIC-rearranged intracranial sarcoma in a 57-year-old man recurred rapidly seven months after initial gross total resection and adjuvant temozolomide, but showed partial response to two cycles of vincristine, ifosfamide, doxorubicin and etoposide plus GammaKnife radiosurgery, with no further recurrence noted one year after diagnosis. A 2012 report of intracerebral myeloid sarcoma in a 22-year-old man described the mass disappearing after eight cycles of chemotherapy, but noted there is no standard treatment and that conversion to systemic haematological disease must be monitored.
The broader context is that brain metastases from systemic sarcomas are increasingly recognised, not less. A 1980 series of 114 sarcoma patients found brain metastases in 11 cases, with remarkably high incidences in rhabdomyosarcoma (26%) and malignant fibrous histiocytoma (27%), possibly related to longer survival and the inability of then-standard drugs to cross the blood-brain barrier. A 2008 review similarly notes that as long-term survival in childhood sarcoma improves, late neurologic complications including brain metastases are reported more frequently. A 2025 review of primary intracranial DICER1-mutant sarcoma confirms that molecular profiling is essential for diagnosis, but the evidence base remains too thin to support any standard treatment recommendation.
What is missing is not enthusiasm but data: prospective registries or trials large enough to stratify by molecular subtype, age, and extent of resection; consistent reporting of progression-free and overall survival beyond short follow-up; and any comparative evidence on whether adjuvant chemotherapy or radiotherapy improves outcomes over surgery alone. Without those, every treatment decision in brain sarcoma will remain a case-by-case judgement based on a handful of published anecdotes.
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 · 1980 · 79 citations
Increased incidence of brain metastases in sarcoma patients
AbstractEleven cases of brain metastases that developed in 114 sarcoma patients are presented. Two of 11 patients presented with brain metastasis at the time of diagnosis and the other nine developed them later. The high incidence of brain metastases in patients with rhabdomyosarcoma (26%) and malignant fibrous histiocytoma (27%), two types of tumor which supposedly metastasize rarely to the brain, is remarkable. The increased incidence of brain metastases may be related to longer survival of sarcoma patients and to the inability of AMN and other drugs used in the treatment of sarcomas to cross the blood-brain barrier. Preventive treatment of brain metastases with drugs active in the CNS or with radiotherapy following the diagnosis of pulmonary metastases, could be useful, especially in patients with rhabdomyosarcoma and malignant fibrous histiocytoma.
AbstractBACKGROUND: Myeloid sarcoma rarely presents in the absence of systemic myeloid disease. CASE REPORT: In this study, we present a case of intracerebral myeloid sarcoma with no diagnosis of any hematological disease in a 22-year-old male patient in whom brain magnetic resonance image revealed a meningioma. However, biopsy showed myeloid sarcoma. No myeloid disease was determined. The mass disappeared following 8 cycles of chemotherapy. In the literature, we determined only 8 similar cases cited between 1970 and 2011. CONCLUSION: Intracerebral myeloid sarcoma has currently no standard treatment and may be confused with a primary brain disease. Chemotherapy and/or radiotherapy are the most viable and widely used treatment modalities. Potential occurrence of hematological disease should also be closely followed due to conversion risks.
AbstractSarcomas are a heterogeneous group of tumors that rarely involve the nervous system. Neurologic effects of sarcoma are more often due to tumors outside of the central nervous system. However, as long-term survival rates in childhood sarcoma improves, reports of late neurologic complications have increased. With recent advances in treating local sarcomas with targeted molecular therapies, the incidence of late neurologic complications, such as brain metastases, will probably continue to increase.
Pakistan Journal of Medical Sciences · 2025 · 0 citations · open access
Primary Intracranial DICER-1 Mutant Sarcoma: A Systematic Review of Existing Literature from 2000 to 2024
AbstractBackground and Objective: Primary Intracranial DICER1-Mutant Sarcoma (PIDMS) is a rare brain tumor with limited data available on its clinical presentation, treatment, and prognosis. This review aimed to analyze the literature on PIDMS, focusing on its presenting features, imaging findings, genetic profiling, surgical treatment, and outcomes. Methodology: Our systematic review was conducted following the Preferred Reporting Items for Systematic Review and Meta-Analyses (PRISMA) guidelines. PubMed and Google Scholar were searched to identify the studies published between 2000 and 2024. Only the studies with biopsy-proven PIDMS cases were included. Studies on animals, metastatic, and extracranial sarcomas were excluded. The Joanna Briggs Institute (JBI) critical appraisal tools were used for the quality assessment. Results: Eight studies comprising 10 patients met the inclusion criteria. Five of the cases (50%) occurred in the pediatric group, while five (50%) in the adult age group (mean age: 15.91 ± 17.71 years). Six patients (60%) were males, and the most common symptoms included headaches and seizures. The frontal and frontoparietal lobes were the most common tumor locations, and the molecular profiling in all 10 cases revealed DICER1 mutation. Gross total resection (GTR) was achieved in 50% of the cases. The mean follow-up duration was 17.5 months, and seven patients underwent combined adjuvant treatment with radiotherapy and chemotherapy. Five patients (50%) had stable disease after undergoing surgical resection and adjuvant therapy. Conclusion: PIDMS is an aggressive neoplasm with non-specific clinical features overlapping with other brain tumours. Extensive genetic profiling and large-scale clinical trials are needed to develop optimized treatment protocols.
Clinical analysis of primary central nervous system histiocytic sarcoma: one case report
AbstractObjective
To provide insight into the clinical manifestation, histopathology characteristics, diagnostic and therapeutic methods of primary histiocytic sarcoma (HS) of central nervous system.
Methods
The clinical, auxiliary examination and pathological data of one patient with intracranial primary histiocytic sarcoma were presented, and relevant literature were reviewed.
Results
A 55-year-old female had an 2-month history of hypomnesia and unsteady gait. MRI scans showed multiple mass lesions in brain. Biopsies of the brain lesion showed the tumor cells had polymorphism, and abundant cytoplasm was eosinophilic or clear, with large, pleomorphic nuclei. Immunohistochemically, the tumor cells stained positively with CD45,CD68, CD163, Ki67 and Vimentin, but negatively with pan-cytokeratin, epithelial membrane antigen, CD3, CD20, CD79a, estrogen receptor, progesterone receptor, Melan-A and glial fibrillary acidic protein. Though she had received radiotherapy, the disease progressed and her consciousness deteriorated within 2 months.
Conclusions
HS should be considered if multiple or single intracranial unexplained lumps are found and the earliest pathological biopsy is required for early diagnosis. There are no effective therapeutic methods for HS and the prognosis is very poor.
Key words:
Histiocytic sarcoma; Central nervous system neoplasms; Diagnosis
Neuro-Oncology Advances · 2019 · 0 citations · open access
CS-14 A CASE OF CIC-REARRANGED INTRACRANIAL SARCOMA
AbstractAbstract INTRODUCTION Intracranial sarcoma is extremely rare among primary brain tumors and often misdiagnosed. Its standard treatment is yet to be established, and treatment options are discussed on a case-by-case basis. Here we report our recent case of intracranial sarcoma review the relevant literature. CASE ILLUSTRATION A 57-year-old right-handed man presented with headache and was found to have a 5cm mass in the right frontal lobe. Gross total resection was achieved without complications. Given the local pathological diagnosis being glioblastoma, adjuvant radiotherapy with concurrent temozolomide was administered. Further pathological examination revealed Capicua (CIC) rearrangement on FISH, which lead to the diagnosis of sarcoma. No further treatment was pursued at that time. However, he noticed rapid decline in the right visual acuity 7 months from the initial diagnosis. MRI demonstrated a rapidly-growing mass in the right optic nerve sized 1.5cm, which was depicted as a high uptake area on FDG-PET, suggestive of recurrence. Two cycles of chemotherapy with vincristine, ifosfamide, doxorubicin, and etoposide as well as GammaKnife stereotactic radiosurgery were performed with partial response. Sustained myelosuppression and debilitating constitutional symptoms precluded additional chemotherapy. No further recurrence was noted 1 year after diagnosis. CONCLUSION We have recently experienced a case of CIC-rearranged intracranial sarcoma. FISH was useful in detecting CIC rearrangement and reaching the correct pathological diagnosis. Rapid recurrence of the tumor was noted, but well controlled with radiochemotherapy.
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works using Disease Ontology synonyms, 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.
