DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for Pineoblastoma — screening already-approved drugs against its 7-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePineoblastoma maps to a 7-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 pineoblastoma 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
lysine demethylase 5C (KDM5C) — KDM5C 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 2-{[(edrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5FWJ · 2.1 Å · ligand 2-{[(2-{[(E)-2-(dimethylamino)ethenyl](ethyl)amino}-2-oxoethyl)amino]methyl}pyridine-4-carboxylic acid (MMK). Experimental structure, not a prediction.
What the evidence adds up to
A systematic review of 108 adult pineoblastoma cases reported a median age at diagnosis of 30 years. The 5-year survival rate was 49.5% (95% CI 0.378–0.602) and the 10-year survival rate was 33.9% (95% CI 0.207–0.476). Gross total resection was associated with better survival than subtotal resection or no surgery (P=0.018). Radiotherapy and chemotherapy were each associated with survival benefit (P<0.001 and P=0.020, respectively). Multivariate Cox analysis identified radiotherapy as an independent favourable prognostic factor (P<0.001); gross total resection showed a trend toward improved survival within five years that did not reach statistical significance (P=0.079).
A single-institution paediatric series of 14 children treated between 1998 and 2017 reported a 5-year event-free survival of 23% and overall survival of 57%; 10-year event-free survival was 23% and overall survival 30.1%. Seven of 14 patients died, one from late radiotherapy sequelae. Ten of 14 patients relapsed. Children under three years old who were treated without radiotherapy had a 5-year overall survival of 0% versus 74.1% in older children who received both chemotherapy and radiotherapy (P=0.014). The authors describe pineoblastoma as a highly malignant childhood tumour with dismal prognosis.
A retrospective analysis of 18 paediatric patients in Beijing treated between 2014 and 2022 reported a 5-year progression-free survival of 42.0% (±13.1%) and 5-year overall survival of 88.1% (±7.9%). Nine patients experienced recurrence; two died. Among the nine recurrent patients, the median time to first relapse was 24.0 months (range 10.0–49.0). The order of treatment appeared to matter: patients who received craniospinal irradiation before chemotherapy had a longer median time to first relapse after surgery than those who received chemotherapy first (29 months versus 13 months, P=0.011). One patient with a germline DICER1 mutation experienced more than three recurrences despite second surgery, second radiotherapy, and multiple chemotherapy cycles, suggesting a particularly poor outcome in that subgroup.
What remains missing are prospective trials large enough to stratify by age, molecular subtype, and extent of resection, as well as dedicated funding for such trials in this ultra-rare tumour. The existing evidence is retrospective, spans decades, and mixes treatment eras, making it difficult to isolate the effect of any single intervention. No standard salvage regimen for recurrent disease has been established.
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 neurosurgery · 1988 · 8 citations
Pineoblastoma with an unusually long survival
AbstractThe authors report a case of pineoblastoma with a 9-year follow-up period after stereotaxic biopsy, a shunting procedure, and radiotherapy. Light and electron microscopic studies of biopsy and autopsy specimens revealed no cell differentiation of the pineoblastoma. The possible factors predisposing to long survival are discussed in comparison with the course in patients with medulloblastoma.
Two cases of pineal anlage tumor with molecular analysis
AbstractPineal anlage tumor is a rare pediatric tumor with clinical and histological features overlapping with pineoblastoma. Two patients with pineal anlage tumor, a 13-month-old female and an 11-month-old male, underwent subtotal resection, high-dose chemotherapy with autologous stem cell rescue, and radiation. Neither had tumor progression 50 months after diagnosis. The tumors underwent next-generation sequencing on a panel of 340 genes. Chromosomal copy gains and losses were present and differed between the tumors. No mutations or amplifications, including none specific to pineoblastoma, were identified.
Frontiers in Oncology · 2024 · 1 citations · open access
A systematic review of adult pineoblastoma
AbstractBackground: Adult pineoblastoma is an extremely rare central nervous system malignancy. Limitations of tumour databases, single institution retrospective analyses and a few case reports are not sufficient to clarify treatment options. Therefore, a systematic review of comprehensive research data provides referenceable treatment options. Methods: A systematic review was performed using MEDLINE and Embase using the terms "pineoblastoma" and "adult". Relevant articles in the references were considered to supplement this systematic review. In addition, data were analysed using Kaplan-Meier survival curves, COX analysis, chi-square tests and log-rank tests. Results: A total of 108 adult cases from 32 articles were included in this study and the median age at diagnosis was 30 years. The 5-year survival rate was 49.5% (95% confidence interval: 0.378-0.602) and the 10-year survival rate was 33.9% (95% confidence interval: 0.207-0.476). During the 10-year follow-up period, Kaplan-Meier survival curves highlighted that the gross total resection was more beneficial than subtotal resection and no surgery (P=0.018). The treatment modality of radiotherapy and chemotherapy was beneficial for survival (P<0.001; P=0.020). In addition, multivariate COX analysis showed that radiotherapy was an independent factor in the beneficial prognosis (P<0.001) and gross total resection tends to improve survival within five years (P=0.079). Conclusion: For adult pineoblastoma, gross total excision and radiotherapy can be beneficial for survival.Systematic Review Registration: [website], identifier [registration number].
CRAN-06. CHILDREN WITH PINEOBLASTOMA. A SINGLE-INSTITUTION SERIES
AbstractPineoblastoma is a rare, highly aggressive supratentorial tumour, more frequently diagnosed in young children. The prognosis remains poor. AIM: To present own experience with children with pineoblastoma. PATIENTS AND 14 pts: 8 girls and 6 boys treated between 1998 and 2017 were analyzed. 5 pts (36%) were under 3 years of age. 7 pts underwent complete tumour resection, 2-partial resection and 5- biopsy. At the onset of treatment six patients had disseminated disease. 9 pts were treated according to protocol for MB HR group (with radiotherapy), 5 pts according to own baby protocol. 9 pts (64%) achieved good response to treatment (CCR-4 pts, CR-4 or PR-1). Four patients experienced tumour progression. 7 out of 14 patients are alive 3 ms to 17years from diagnosis (median 3years 4ms). Seven patients died (10ms to 8years from diagnosis). 1 pt died of long term post- radiotherapy sequelae. 5 yr EFS was 23 %, OS- 57%. 10 yr EFS and OS were 23 and 30,1% consecutively. 10 out of 14 pts relapsed 7ms to 4 years 3ms from diagnosis. 9 pts received second line treatment- 4 are alive. Children younger than 3 years of age treated without radiotherapy had worse outcome with 5 yrs OS estimated at 0% vs 74,1% in older children treated with chemotherapy and radiotherapy (p=0,014). Pineoblastoma is highly malignant CNS neoplasm of childhood with dismal prognosis. In our series children below 3 years of age treated without radiotherapy had poorer responses and worse outcome.
LMIC-19. A RETROSPECTIVE ANALYSIS OF PEDIATRIC RECURRENT PINEOBLASTOMA IN BEIJING
AbstractAbstract OBJECTIVE To explore the clinical characteristics of pediatric recurrent pineoblastoma in Beijing. METHODS Eighteen patients with newly diagnosed pineoblastoma were admitted to Beijing Shijitan Hospital between January 2014 and December 2022. The clinical data were retrospectively analyzed. RESULTS Among the 18 patients (M/F=8:1), all were treated with surgery, and both radiotherapy and chemotherapy were administered. Nine patients experienced recurrence, and 2 patients died at last follow-up. The Median follow-up time were 56 months. The 5-year progression-free survival (PFS) and 5-year overall survival (OS) were (42.0±13.1) %, and (88.1±7.9) %, respectively. Among the 9 recurrent patients, one was &lt; 3 years, and 8 cases ≥ 3 years. One girl and 8 boys relapsed; 7 cases were M0 and 2 with metastases at diagnosis; 7 cases were GTR; Two cases experienced recurrence within one year (10 months each), and one died. The other 7 patients relapsed after one year, the median time to first relapse were 24.0 (range: 10.0~49.0)months. The sex, age group, metastases at diagnosis, GTR or not, recurrent sites were not significantly affects PFS. Although the 9 recurrent patients received both cerebral and spinal irradiation and chemotherapy followed by surgery, the different order of chemotherapy or radiotherapy first were significantly affected the duration of remission. The median time before the first relapse after surgery was longer in those who treated under the order of craniospinal irradiation followed by chemotherapy than those of the inverse order (29m vs 13m, χ2=6.528, P=0.011). In addition, one case with germline mutation of Dicer1, experienced recurrences more than 3 times although received second surgery and second radiotherapy, and multiple cycles of chemotherapy in our center, which indicates poorer outcome. CONCLUSION Pineoblastoma is rare and prone to relapse, the order of chemotherapy first may have shorter remission time before the first recurrence after surgery (P&lt; 0.05).
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.
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.
