DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for cerebellar hemangioblastoma — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCerebellar hemangioblastoma maps to a 1-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 cerebellar hemangioblastoma 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
von Hippel-Lindau tumor suppressor (VHL) — VHL 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 2s,4rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8QVU · 2.24 Å · ligand (2S,4R)-1-[(2S)-2-[4-[4-[(3S)-4-[4-[5-[(4S)-2-azanyl-3-cyano-4-methyl-6,7-dihydro-5H-1-benzothiophen-4-yl]-1,2,4-oxadiazol-3-yl]pyrimidin-2-yl]-3-methyl-1,4-diazepan-1-yl]butoxy]-1,2,3-triazol-1-yl]-3-methyl-butanoyl]-N-[(1R)-1-[4-(4-methyl-1,3-thiazol-5-yl)phenyl]-2-oxidanyl-ethyl]-4-oxidanyl-pyrrolidine-2-carboxamide (WYL). Experimental structure, not a prediction.
What the evidence adds up to
A 51-year-old man developed numerous mass lesions along the entire neuraxis with leptomeningeal enhancement ten years after surgery for a solitary cerebellar hemangioblastoma. No mutation in the VHL gene was found. External beam radiation and radiosurgery were given; the patient died one year after the diagnosis of hemangioblastomatosis. Two women aged 45 and 57, also without VHL mutations, had total resection of cerebellar hemangioblastomas. One had local recurrence at 38 months, spinal cord tumours at 91 months, and dissemination to the entire cerebrospinal cavity at 107 months. The other developed hydrocephalus at 53 months with tumour in the intracranial subarachnoid space. Both received ventriculo-peritoneal shunts and whole-brain and whole-spinal irradiation; both died about two years after the diagnosis of dissemination. The 2014 paper states that molecular targeted therapies including vascular proliferation suppression have been attempted but no effective therapy has been established.
A retrospective review of 19 patients with cerebellar hemangioblastomas larger than 3 cm in diameter reported 20 resections. Preoperative embolisation was used in eight cases (38.1%). One patient had transient neurological complications after embolisation (12.5%). Embolised patients had larger median total, solid, and cystic tumour volumes and were more likely to involve the cerebellopontine angle. Compared with non-embolised patients, embolised patients had less decrease in haemoglobin, lower estimated blood loss, fewer postoperative complications and permanent deficits, and more instances of neurological improvement. A combined cohort of 99 patients (39 embolised) from the same institution and a literature review supported these findings. The authors conclude that improvements in endovascular techniques have made preoperative embolisation a safe and effective procedure with minimal risks for many patients.
The 2009 and 2014 case reports both note that cerebrospinal fluid dissemination of cerebellar hemangioblastoma occurs predominantly in patients without VHL disease. Diagnosis of dissemination was made roughly ten years after initial surgery. Irradiation was given in all cases but did not prevent death within one to two years of the diagnosis of dissemination. The 2022 embolisation study does not report long-term survival or recurrence data for the embolised versus non-embolised groups.
What is still missing is any prospective trial of systemic therapy for disseminated hemangioblastomatosis, a reliable biomarker or imaging method to predict which solitary tumours will disseminate, and a standardised protocol for long-term surveillance after resection of a sporadic cerebellar hemangioblastoma. Patient stratification by VHL mutation status, tumour volume, and histological subtype is not yet incorporated into treatment decisions beyond surgical planning.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of Korean Medical Science · 2009 · 21 citations · open access
Disseminated Hemangioblastomatosis of the Central Nervous System without von Hippel-Lindau Disease: A Case Report
AbstractWe report a very rare case of hemangioblastomatosis that developed after surgical removal of a solitary cerebellar hemangioblastoma (HB). A 51-yr-old man presented with back pain 10 yr after undergoing surgery for cerebellar HB. Magnetic resonance imaging showed numerous mass lesions along the entire neuraxis accompanied by prominent leptomeningeal enhancement. Genomic DNA analysis showed no mutation in the von Hippel-Lindau (VHL) genes. A surgical specimen obtained from a lesion in the cauda equina showed pathological findings identical to those of the cerebellar HB that had been resected 10 yr earlier. External beam radiation therapy and radiosurgery were subsequently performed; however, the patient succumbed one year after receiving the diagnosis of hemangioblastomatosis. The reduction of tumor cell spillage during surgery and regular long-term follow-up are recommended for patients with HBs.
Surgical Neurology International · 2014 · 14 citations · open access
Disseminated cerebellar hemangioblastoma in two patients without von Hippel-Lindau disease
AbstractBACKGROUND: Two patients who had received a total resection of cerebellar hemangioblastoma developed cerebrospinal fluid dissemination during a long-term follow-up period. We present this rare disease with discussion based on the literature. CASE DESCRIPTION: The patients were two women aged 45 and 57 years. In the cerebellar hemisphere, one patient had cystic hemangioblastoma of mural nodule type and the other had solid type. Both the patients successfully underwent total resection by craniotomy. They presented no mutations in the von Hippel-Lindau disease (VHL) gene or lesions in the other organs. One patient developed local recurrence 38 months after the initial surgery, and received stereotactic radiosurgery. Three spinal cord tumors developed 91 months later, and the tumors were disseminated to the entire cerebrospinal cavity 107 months later. The other patient developed hydrocephalus 53 months after the initial surgery with tumor tissues disseminated in the intracranial subarachnoid space. The conditions of the two patients gradually aggravated despite treatment with ventriculo-peritoneal shunt and irradiation to the whole brain and whole spinal cord. CONCLUSION: Cerebrospinal fluid dissemination of cerebellar hemangioblastoma was found dominantly in non-VHL patients. The diagnosis was made 10 years after the initial surgery. Irradiation therapy was performed, but the patients died about 2 years after the diagnosis was given. Molecular targeted therapies including vascular proliferation suppression have been attempted lately, but no effective therapy has been established. Early diagnosis of dissemination as well as combination of aggressive excision and stereotactic radiosurgery are considered to be appropriate for current interventions.
Journal of Neurological Surgery Part B Skull Base · 2022 · 3 citations · open access
Embolization of Large and Giant Posterior Fossa Hemangioblastomas: The Experience of a Single Tertiary Care Center
AbstractAbstract Background Hemangioblastomas pose an inherent surgical risk due to the potential for high intraoperative blood loss, especially in larger tumors. One approach to minimize this risk is to use preoperative embolization. Herein, we present our institutional experience treating large and giant cerebellar hemangioblastomas. Methods We performed a retrospective chart review of 19 patients with cerebellar hemangioblastomas that had a maximal diameter of >3 cm. We performed a literature review and included individual patient-level data that met our >3 cm diameter cerebellar hemangioblastoma inclusion criteria. Results Our cohort consisted of 19 patients that received a total of 20 resections for their cerebellar hemangioblastomas. Preoperative embolization was utilized in eight cases (38.1%). One patient experienced transient neurological complications after embolization (12.5%). Tumors of patients in the embolization group had larger median total, solid, and cystic volumes and were more likely to involve the cerebellopontine angle than those in the non-embolized group. Compared with non-embolized patients, embolized patients had less decrease in their hemoglobin, lower volumes of estimated blood loss, reduced rates of postoperative complications and permanent deficits, and greater instances of neurological improvement. The larger cohort (obtained from the combining our cohort with patients identified during a literature review) consisted of 99 patients with 39 receiving preoperative embolization. Conclusion It is important to examine individual patient characteristics when determining eligibility for preoperative embolization. However, improvements in endovascular techniques have made preoperative embolization a safe and effective procedure with minimal risks that can be performed in many patients.
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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