DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for hemangioblastoma — 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 moduleHemangioblastoma 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
No approved-drug candidate for 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
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
Hemangioblastoma is a rare, benign central nervous system tumour with an overall incidence of 0.141 per 100,000 person-years according to a SEER database analysis. In that analysis of prognostic factors, age between 60 and 79 years (HR 3.697, p<0.001) and age over 80 years (HR 12.318, p<0.001) were associated with worse overall survival, while African American race (HR 1.857, p=0.003), multiple tumours (HR 1.715, p<0.001), and having had surgery (HR 0.638, p=0.013) were associated with better overall survival. Patients who received surgery alone had better overall survival than those receiving no treatment (p=0.008) and those receiving both surgery and radiotherapy (p=0.002). Surgery is described as the most common and effective treatment, and isolated sporadic hemangioblastomas typically respond well after surgery, with neurological deficits sometimes reversed post-operatively.
The tumours can occur sporadically or in association with von Hippel-Lindau disease, a germline mutation in the VHL tumour suppressor gene. A 2023 review states that surgical resection and radiotherapy are effective, while conventional chemotherapies are not commonly used due to limited blood-brain barrier penetration. That review notes that recent chemotherapies have shown promise but that further research is needed to determine efficacy, and it describes new advances in brachytherapy and immunotherapy as promising but not yet established.
A 2025 study examined 139 hemangioblastoma samples from 111 patients for fibroblast growth factor receptor (FGFR) expression. Immunohistochemistry showed positive staining for FGFR2 in 95% of samples and FGFR4 in 61%, while FGFR1 was negative in all samples and FGFR3 positive in only 12%. FGFR2 expression was significantly increased in VHL-mutated tumours (75%, p=0.034) and in male patients (68%, p=0.020). Tumours located in the cerebrum had a higher likelihood of positive FGFR4 staining (100% of 6 cerebral tumours, p=0.009), and larger tumour diameter was associated with FGFR4 expression (median 12.0 mm vs 17.5 mm, p=0.018). The authors propose FGFRs as potential therapeutic targets, but no clinical trial data on FGFR inhibitors in hemangioblastoma patients are presented.
What is still missing: prospective clinical trials testing any targeted therapy or immunotherapy specifically in hemangioblastoma patients, validated biomarkers to stratify sporadic versus VHL-associated cases for treatment, and funding to move beyond retrospective case series and immunohistochemistry studies into interventional trials.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Frontiers in Oncology · 2020 · 46 citations · open access
Incidence, Prognostic Factors and Survival for Hemangioblastoma of the Central Nervous System: Analysis Based on the Surveillance, Epidemiology, and End Results Database
AbstractBackground: Hemangioblastomas are uncommon, benign neoplasms of the central nervous system (CNS). This study aims to evaluate the incidence, demographics, clinical characteristics, and prognosis of CNS hemangioblastomas using the data from the Surveillance, Epidemiology, and End Results (SEER) Program. Methods: Univariate and multivariate analyses using the Cox proportional hazards model were employed to identify prognostic factors of overall survival. The Kaplan-Meier method was utilized to evaluate overall survival distribution by treatment modality. A nomogram was further built to predict survival at 3 and 5 years. Results: The overall incidence rate of CNS hemangioblastomas was 0.141 per 100,000 person-years. Through univariate analysis and multivariate analyses, age between 60-79 years (HR=3.697, p<0.001), age greater than 80 years (HR=12.318, p<0.001), African American race (HR=1.857, p=0.003), multiple tumors (HR=1.715, p<0.001), and prior surgery (HR=0.638, p=0.013) were significantly associated with better overall survival. Patients receiving surgery alone had better overall survival compared with patients receiving no treatment (p=0.008) and patients receiving both surgery and radiotherapy (p=0.002). The calibration plots demonstrated an excellent agreement between nomogram-predicted and actual survival. Conclusions: In conclusion, age, race, tumor location, number of tumors, and prior surgery are prognostic factors for survival. Surgery was the most common modality and was suggested as an effective and optimal treatment. The proposed nomogram can predict the prognosis of patients with CNS hemangioblastomas and help clinicians in making decisions.
AbstractOBJECTIVE: Although reported to occur throughout the central nervous system, hemangioblastomas are principally found in the cerebellum and spinal cord. Pineal region tumors comprise approximately 1% of central nervous system neoplasms. A wide variety of tumors can affect this region, the most common being germ cell tumors, gliomas, and pineal cell tumors. In the literature, we found only one case of hemangioblastoma in the pineal region in association with von Hippel-Lindau disease. CLINICAL PRESENTATION: We describe the case of a patient with a symptomatic hemangioblastoma in the pineal region with no clinical criteria for von Hippel-Lindau disease. The patient had a 1-month history of short-term memory loss, headache, difficulty concentrating and writing, disturbed balance, and loss of bladder function. At the time of physical examination, she was awake, alert, and oriented. An ophthalmoscopic examination revealed nystagmus with conjugate upward gaze and papilledema. Radiological images showed a mass in the pineal region with obstructive hydrocephalus. INTERVENTION: A lateral suboccipital infratentorial supracerebellar approach was used to remove the tumor, which was attached to the quadrigeminal plate. Histological examination showed the lesion to be a hemangioblastoma. The clinical findings for von Hippel-Lindau disease were negative. CONCLUSION: The patient's neurological deficits were reversed after surgery. This case emphasizes the importance of the differential diagnosis of hemangioblastomas located in this region. These tumors can be safely removed through surgery.
Exploration of neuroscience · 2023 · 4 citations · open access
Emerging therapies of hemangioblastomas
AbstractHemangioblastoma are benign, vascularized cranial tumors caused by autosomal dominant inherited von Hippel-Lindau disease or can appear sporadically. This review will investigate current and emerging treatments for cerebral tumors. It will focus on the current and, more importantly, developing hemangioblastoma treatments. Surgical resectioning and radiotherapy are effective treatment options for cerebral tumors, whereas chemotherapies are not commonly used due to their limited ability to penetrate the blood-brain barrier. Recent chemotherapies have shown promise, but further research is needed to determine the efficacy as a treatment for hemangioblastomas. New advances in brachytherapy and immunotherapy are considered promising treatment options for hemangioblastoma. This review aims to offer valuable insights into the latest developments in hemangioblastoma treatments.
PHOTODYNAMIC THERAPY AND VITRECTOMY FOR A LARGE OPTIC NERVE HEMANGIOMA WITH NEOVASCULARIZATION AND RETINAL DETACHMENT: A CLINICOPATHOLOGIC CORRELATION
AbstractIn Brief Purpose: To report the clinicopathologic correlation of a young man with a von Hippel-Lindau disease–associated peripapillary hemangioblastoma and its satisfactory response to a combination of photodynamic therapy (PDT) and vitrectomy. Design: Clinicopathologic correlation. Methods: We studied the case of a 14-year-old boy with an optic nerve mass and large inferior exudative retinal detachment complicated by a significant tractional component from extensive secondary neovascularization over the lesion. Results: A juxtapapillary hemangioblastoma with secondary neovascularization was documented by clinical examination, fundus photography, and optical coherence tomography. A von Hippel-Lindau gene mutation was detected. The patient responded satisfactorily to a combination of PDT and vitrectomy. Conclusions: A staged approach to treatment of peripapillary hemangioblastoma with a combination of PDT and vitrectomy may be favorable to therapy with one modality. The authors describe the clinicopathologic correlation of a 14-year-old boy with a large von Hippel-Lindau disease-associated peripapillary hemangioblastoma with secondary neovascularization and a large inferior exudative and tractional retinal detachment. The patient responded favorably to a combination of photodynamic therapy and vitrectomy, and histopathologic analysis of the neovascular membrane showed closure of blood vessels after photodynamic therapy.
International Journal of Surgery Case Reports · 2016 · 2 citations · open access
Isolated hemangioblastoma of the cervical spinal cord
AbstractINTRODUCTION: Hemangioblastomas are benign, slow growing but highly vascularized tumors of the central nervous system, with the most common location of occurrence in the posterior fossa. Hemangioblastomas usually have an associated with patients that have Von-Hippel Lindau disease, resulting a germline mutation in the VHL tumor suppressor gene. Isolated or sporadic occurrences of hemangioblastomas are much more infrequent and typically respond well after surgery. PRESENTATION OF CASE: We present case of a 22year old female with worsening shoulder pain, decreased sensation in the hands and feet, and decreasing strength and was found to have a hemangioblastoma of the cervical spine. DISCUSSION: The patient was treated with surgery and responded well to treatment. We also present a review of the literature on isolated occurrences of hemangioblastomas of the spinal cord. CONCLUSION: Isolated hemangioblastoma are a rare tumor of the central nervous system and can be managed with surgery.
Fibroblast growth factor receptor expression in hemangioblastomas: A novel therapeutic target
AbstractHemangioblastoma is a highly vascularized, benign tumor in the central nervous system, frequently associated with von Hippel-Lindau (VHL) disease. Hemangioblastoma may cause tumor-associated hemorrhage or exert pressure on nearby structures, leading to life-threatening complications. Although surgical resection is the primary treatment, complete removal is not always feasible. Accordingly, there is a need to explore targeted or anti-angiogenic therapies. The fibroblast growth factor receptor (FGFR) family has roles in tumorigenesis and angiogenesis, making it a potential target in personalized therapy. The distribution and significance of FGFRs in hemangioblastoma have yet to be investigated. We examined 139 formalin-fixed, paraffin-embedded hemangioblastoma samples from 111 patients, including sporadic cases and those associated with VHL disease. Immunohistochemistry revealed positive staining for FGFR2 (95%) and FGFR4 (61%), while FGFR1 (0%) and FGFR3 (12%) were mainly negative. FGFR2 expression was significantly increased in VHL-mutated tumors (75%, p = 0.034) and in male patients (68%, p = 0.020). Tumors located in the cerebrum (n = 6, 5%) had a higher likelihood of positive FGFR4 staining (100%, p = 0.009). Additionally, a larger tumor diameter was associated with a higher likelihood of FGFR4 expression (median 12.0 mm vs 17.5 mm, p = 0.018), suggesting its contribution in tumor growth. Our study revealed the expression of FGFR2 and FGFR4 in a significant number of hemangioblastomas. This finding demonstrates the potential of FGFRs as promising therapeutic targets for patients with hemangioblastoma.
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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