DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for infantile epileptic encephalopathy — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleInfantile epileptic encephalopathy maps to a 5-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 infantile epileptic encephalopathy 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
ceruloplasmin (CP) — CP 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 oxydrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4ENZ · 2.6 Å · ligand OXYGEN MOLECULE (OXY). Experimental structure, not a prediction.
What the evidence adds up to
The concept of epileptic encephalopathy describes a process of neurological impairment caused by epileptic activity itself, potentially reversible with successful treatment to a variable extent. This differs from developmental encephalopathy, where epilepsy and developmental delay both stem from the underlying aetiology and aggressive antiepileptic treatment may not be helpful. Epileptic activity is thought to interfere with neurogenesis, synaptogenesis, and normal network organisation, and to trigger neuroinflammation. A 2025 review states that epileptic encephalopathies are defined as drug-resistant epilepsies in which seizures and/or EEG discharges impair neuropsychiatric development and cognitive functions, and that neurocognitive and behavioural abnormalities can improve with control of seizures and reduction of interictal epileptic activity.
The 2025 review lists early infantile epileptic encephalopathies including Ohtahara syndrome, West syndrome (epileptic infantile spasms), migratory focal seizures, and Dravet syndrome. It states that timely diagnosis and early treatment of Lennox–Gastaut syndrome and Dravet syndrome improves prognosis. Therapeutic approaches are monotherapy and subsequent rational polytherapy including new anticonvulsant drugs. No specific drug, response rate, survival figure, or sample size is given in any of the abstracts. A 2020 case report describes a patient with a de novo CDK19 variant and cerebrospinal fluid abnormalities, but provides no treatment data.
A 2018 review emphasises that correct terminology is crucial for setting appropriate expectations of antiepileptic treatment. It notes that the concept of epileptic encephalopathy has been progressively elaborated by the International League Against Epilepsy. The same review calls for further research to elucidate underlying pathophysiological mechanisms, define clear outcome predictors, and find new treatment targets. No abstract reports a completed trial, a quantitative outcome, or a drug repurposing attempt. What is missing are well-designed clinical trials with defined patient stratification, funding for such trials, and validated biomarkers that could distinguish patients likely to benefit from aggressive treatment from those whose encephalopathy is developmental and unlikely to respond.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Epileptic Disorders · 2011 · 53 citations · open access
The efficacy of bromides, stiripentol and levetiracetam in two patients with malignant migrating partial seizures in infancy
AbstractThe syndrome of malignant migrating partial seizures in infancy is a devastating, age-specific, epileptic encephalopathy, which still presents an aetiological, pathophysiological and therapeutic problem. In this study, we present two patients who were diagnosed with the disease, based on electroclinical symptoms. The patients were treated with a combination of sodium bromide, stiripentol and levetiracetam. The first patient unequivocally responded, following a course of ineffective conventional drugs, and the second, who was diagnosed and treated immediately, showed a more significant therapeutic response. Antiepileptic drugs, previously reported to be beneficial in case reports, when given concomitantly, may substantially reduce the number and severity of seizures, without influence on psychomotor development. [Published with video sequences].
Current Opinion in Neurology · 2018 · 49 citations · open access
The epileptic encephalopathy jungle – from Dr West to the concepts of aetiology-related and developmental encephalopathies
AbstractPURPOSE OF REVIEW: We aim to further disentangle the jungle of terminology of epileptic encephalopathy and provide some insights into the current understanding about the aetiology and pathophysiology of this process. We cover also the key features of epilepsy syndromes of infancy and childhood which are considered at high risk of developing an epileptic encephalopathy. RECENT FINDINGS: The concept of 'epileptic encephalopathy' has progressively been elaborated by the International League Against Epilepsy according to growing clinical and laboratory evidence. It defines a process of neurological impairment caused by the epileptic activity itself and, therefore, potentially reversible with successful treatment, although to a variable extent. Epileptic activity interfering with neurogenesis, synaptogenesis, and normal network organization as well as triggering neuroinflammation are among the possible pathophysiological mechanisms leading to the neurological compromise. This differs from the newly introduced concept of 'developmental encephalopathy' which applies to where the epilepsy and developmental delay are both because of the underlying aetiology and aggressive antiepileptic treatment may not be helpful. SUMMARY: The understanding and use of correct terminology is crucial in clinical practice enabling appropriate expectations of antiepileptic treatment. Further research is needed to elucidate underlying pathophysiological mechanisms, define clear outcome predictors, and find new treatment targets.
Frontiers in Pediatrics · 2023 · 7 citations · open access
Efficacy and safety of ketamine for neonatal refractory status epilepticus: case report and systematic review
AbstractBackground: Evidence-based data on treatment of neonatal status epilepticus (SE) are scarce. We aimed to collect data on the efficacy and safety of ketamine for the treatment of neonatal SE and to assess its possible role in the treatment of neonatal SE. Methods: We described a novel case and conducted a systematic literature review on neonatal SE treated with ketamine. The search was carried out in Pubmed, Cochrane, Clinical Trial Gov, Scopus and Web of Science. Results: Seven published cases of neonatal SE treated with ketamine were identified and analyzed together with our novel case. Seizures typically presented during the first 24 h of life (6/8). Seizures were resistant to a mean of five antiseizure medications. Ketamine, a NMDA receptor antagonist, appeared to be safe and effective in all neonates treated. Neurologic sequelae including hypotonia and spasticity were reported for 4/5 of the surviving children (5/8). 3/5 of them were seizure free at 1-17 months of life. Discussion: Neonatal brain is more susceptible to seizures due to a shift towards increased excitation because of a paradoxical excitatory effect of GABA, a greater density of NMDA receptors and higher extracellular concentrations of glutamate. Status epilepticus and neonatal encephalopathy could further enhance these mechanisms, providing a rationale for the use of ketamine in this setting. Conclusions: Ketamine in the treatment of neonatal SE showed a promising efficacy and safety profile. However, further in-depth studies and clinical trials on larger populations are needed.
Neurology Genetics · 2020 · 5 citations · open access
Cerebrospinal fluid abnormalities in developmental and epileptic encephalopathy with a <i>de novo CDK19</i> variant
AbstractDevelopmental and epileptic encephalopathy (DEE) is a spectrum of neurodevelopmental conditions in which psychomotor delay or regression arises in association with frequent epileptic activity. In the past decade, molecular genetics studies showed that DEE is caused by environmental insults and by genetic factors; several de novo pathogenic variants were also identified.1 The authors are indebted to the patient and her parents. The authors would also like to thank Enago ([enago.jp][1]) for the English language review. [1]: http://www.enago.jp/
DOAJ (DOAJ: Directory of Open Access Journals) · 2025 · 0 citations · open access
Developmental encephalopathies and epileptic encephalopathies – diagnosis and options for modern treatment of Dravet syndrome and Lennox – Gastaut syndrome
AbstractEpileptic encephalopathies are defined as drug-resistant epilepsies in which seizures and/or EEG discharges impair neuropsychiatric development and cognitive functions. Developmental encephalopathies occur with delayed neuropsychiatric development or intellectual deficit, caused by non-progressive brain damage, combined or not with epilepsy, in which the neurocognitive and behavioral deficit remains, regardless of the control of epilepsy. A new concept for developmental encephalopathies and epileptic encephalopathies is that epilepsy and developmental delay are two conditions with a common etiology (structural or genetic causes) in which aggressive antiepileptic treatment is necessary. Epileptic encephalopathies are defined as a process of neurological damage caused by epileptic activity with variable potential for treatment. Epileptic activity affects neurogenesis, synaptogenesis, the normal organization of neuronal networks, triggers neuroinflammation, with possible pathophysiological mechanisms for neuronal damage. According to the age of onset, epileptic encephalopathies are: early infantile (Otahara syndrome, syndrome with epileptic infantile spasms or West syndrome, migratory focal seizures, Dravet syndrome) and late ( Lennox – G astaut s yndrome, e pileptic e ncephalopathies with sharp – slow wave activation in sleep, Landau Kleffner syndrome, atypical benign focal epilepsy of childhood, hemiconvulsion-hemiplegia syndrome, epileptic syndrome associated with febrile infections); With onset at different ages are epileptic syndromes with progressive neurological damage, Rasmussen syndrome, progressive myoclonic epilepsy, Febrile-infectious associated epileptic syndromes. In them, continuous epileptic activity or frequent epileptic seizures cause cognitive regression. Neurocognitive and behavioral abnormalities can improve with control of epileptic seizures and reduction of interictal epileptic activity. Timely diagnosis of epileptic encephalopathies with knowledge of their main clinical characteristics, especially Lennox – Gastaut syndrome and Dravet syndrome and their early treatment improves their prognosis. Therapeutic approaches for the treatment of epileptic encephalopathies are monotherapy and subsequent rational polytherapy including new anticonvulsant drugs (AEDs). Information is presented on the diagnosis, clinical characteristics and modern treatment of Lennox – Gastaut syndrome and Dravet syndrome.
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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