DeCure for Developmental and epileptic encephalopathy, 13
DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for developmental and epileptic encephalopathy, 13 — 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 moduleDevelopmental and epileptic encephalopathy, 13 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
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Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Structures already discussed alongside developmental and epileptic encephalopathy, 13 in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
aldehyde dehydrogenase 7 family member A1 (ALDH7A1) — ALDH7A1 is one of the genes in this disease's Open Targets module — part of the target space DeCure's repurposing candidates point at. The protein backbone is drawn as a cartoon. The structure has nicotinamide-adenine-dinucleotide bound in it, shown as sticks.
Loading structure…
helix sheet naddrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6O4C · 1.7 Å · ligand NICOTINAMIDE-ADENINE-DINUCLEOTIDE (NAD). Experimental structure, not a prediction.
What the evidence adds up to
Developmental and epileptic encephalopathy 13 is one of a group of heterogeneous paediatric epilepsies defined by drug-resistant seizures and neurodevelopmental delay, typically with onset in infancy or early childhood and an overall poor prognosis. The condition is mainly driven by genetic factors, and no currently available drug has succeeded in suppressing the epileptiform activity in these disorders. A 2025 systematic review of pharmaceutical cannabidiol in children with developmental and epileptic encephalopathies identified 13 eligible studies covering 656 children. Cannabidiol was given at a maximum dose of 50 mg/kg/day. In 10 of those studies, at least 20% of patients achieved a 50% or greater reduction in seizure frequency. Adverse events were relatively common and included somnolence, loss of appetite, diarrhoea, fatigue, and increased serum aminotransferases; most were mild to moderate and reversible. The review concluded that cannabidiol was well tolerated and effectively reduced seizure frequency in children whose seizures were uncontrolled with concomitant antiepileptic medications.
The broader literature confirms that developmental and epileptic encephalopathies constitute a sizeable group of rare epileptic conditions with multiaxial neurological morbidities. Many affected children develop pharmacoresistant epilepsy beginning in early infancy, do not achieve remission, and require comprehensive medical care into adulthood. The conceptual framework has evolved from initial genetic underpinnings to a clinically driven term encompassing a broader range of aetiologies, and recognition of treatable or modifiable entities within this group is considered especially important. Current reviews summarise well-established DEE syndromes and available precision therapies, but the evidence base for most individual DEE subtypes, including DEE13, remains limited to small case series and genetic reports.
What is still missing are large, randomised controlled trials specifically for DEE13, which would require substantial funding and international collaboration given the rarity of the condition. Long-term data on cannabidiol’s effects beyond seizure frequency—on neurodevelopment, cognition, behaviour, and quality of life—are lacking. Patient stratification by genetic subtype will be essential to determine whether any treatment benefit is uniform across the DEE spectrum or confined to particular aetiologies.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Neuropsychiatric Disease and Treatment · 2009 · 18 citations · open access
Update on the management of Lennox-Gastaut syndrome with a focus on rufinamide
AbstractUpdate on the management of Lennox-Gastaut syndrome with a focus on rufinamide Carl E StafstromSection of Pediatric Neurology, Departments of Neurology and Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, USAObjective: This review summarizes the treatment of Lennox-Gastaut syndrome, an intractable epileptic encephalopathy of early childhood. In particular, the review focuses on rufinamide, a recently released anticonvulsant medication with reported effectiveness in this epilepsy syndrome.Methods: A systematic literature search (PubMed) was performed to review the existing literature pertaining to the treatment of Lennox-Gastaut syndrome as well as studies involving rufinamide as an anticonvulsant medication.Results: The published literature to date documents a beneficial effect of rufinamide on children over 4 years old with Lennox-Gastaut syndrome. Studies indicate a significant decrease in tonic and atonic seizure frequency as well as total seizure frequency compared to placebo-treated children. Rufinamide appears to be well tolerated and a safe medication, somnolence and vomiting being the most common side effects.Conclusions: Rufinamide is a promising adjunctive therapy for Lennox-Gastaut syndrome, an intractable childhood epilepsy. To ensure its optimal effectiveness, clinicians must be familiar with the medication’s clinical response profile and potential for adverse effects.Keywords: pediatric, epilepsy, epileptic encephalopathy, Lennox-Gastaut syndrome, rufinamide
Epileptic Encephalopathies: Clinical Aspects, Molecular Features and Pathogenesis, Therapeutic Targets and Translational Opportunities, and Future Research Directions
AbstractEpileptic encephalopathies encompass a heterogeneous group of epilepsy syndromes that manifest with cognitive, behavioral, and neurologic deficits, seizures that are often intractable and multiform, aggressive electroencephalographic paroxysmal activity, and sometimes early death. As more is learned about the etiologies and manifestations of epileptic encephalopathies, progress has been made toward better treatment options. However, there is still a great need for further randomized controlled trials and research to help create clinically effective therapies. The 2015 Neurobiology of Disease in Children symposium, held in conjunction with the 44th annual meeting of the Child Neurology Society, aimed to (1) describe the clinical concerns involving diagnosis and treatment, (2) review the current status of understanding in the pathogenesis of epileptic encephalopathy, (3) discuss clinical management and therapies for epileptic encephalopathy, and (4) define future directions of research. This article summarizes the presentations and includes an edited transcript of question-and-answer sessions.
International Journal of Epilepsy · 2025 · 1 citations · open access
Developmental and Epileptic Encephalopathies: Progress in Understanding and Clinical Implications
AbstractAbstract Developmental and epileptic encephalopathies (DEEs) are a group of complex pediatric epilepsies associated with adverse neurodevelopmental outcomes and multiaxial neurological morbidities. The understanding of DEE has evolved from its initial genetic underpinnings, to a clinically driven term encompassing a broader group of etiologies. Although individually rare, the DEEs constitute a sizeable group of pediatric epilepsies. It is especially important to recognize treatable or modifiable entities in this group. This review attempts to highlight the progress in understanding, along with the evolution of the conceptual framework of DEEs. A summary of the currently well-established DEE syndromes is provided along with available precision therapies, which might help the treating physician in the appropriate evaluation and management of the affected children.
DOAJ (DOAJ: Directory of Open Access Journals) · 2024 · 0 citations
Research progress on developmental and epileptic encephalopathy
AbstractDevelopmental and epileptic encephalopathy (DEE) is a group of heterogeneous disorders characterized by drug-resistant seizures and neurodevelopmental delay, which can hinder brain development and lead to severe cognitive, behavioral, and motor impairments. DEE is mainly driven by genetic factors, typically with an onset in infancy or early childhood and generally with an overall poor prognosis. This article provides a review of the definition, epilepsy syndromes, genetic etiology, and precision treatment of DEE.
Aristotle University of Thessaloniki · 2025 · 0 citations · open access
Efficacy and safety of cannabidiol in children with developmental and epileptic encephalopathies: A systematic review
AbstractDevelopmental and epileptic encephalopathies constitute rare epileptic conditions characterized by treatment-resistant seizures, neurodevelopmental delays, and various comorbidities. None of the currently available drugs succeeded in suppressing the epileptiform activity in those conditions. Cannabidiol has been used as a novel treatment strategy in patients with treatment-resistant epilepsies. We aimed to assess the efficacy and safety of cannabidiol in children with developmental and epileptic encephalopathies through a systematic review. For this systematic review, we searched MEDLINE, Cochrane Central Register of Controlled Trials, trial registries, and reference lists of included studies. All types of studies of pharmaceutical cannabidiol in children with developmental and epileptic encephalopathies were eligible, and we did not set any language or date restrictions. The risk of bias in included studies was assessed using validated tools. Of the 683 records identified, 13 were eligible for inclusion in this review. Studies were of different types and included a total of 656 children with developmental and epileptic encephalopathies. Cannabidiol was administered at a maximum 50mg/kg/day dose. Almost all studies reported positive outcomes with cannabidiol, leading to a reduction of 50% or above in seizure frequency in at least 20% of patients included in 10 studies. Adverse events were relatively common across studies and included somnolence, loss of appetite, diarrhea, fatigue, and increased serum aminotransferases. Most of them were mild to moderate and reversible. Overall, cannabidiol was found to be well tolerated and to effectively reduce seizure frequency in children with developmental and epileptic encephalopathies experiencing seizures uncontrolled with concomitant antiepileptic medications. Future research should investigate the long-term effects of cannabidiol not only in seizure control but also in enhancing the development and quality of life of affected children.
AbstractChildhood epilepsy is characterized by specific epilepsy syndromes that occur during each developmental age. These "age-dependent" epilepsy syndromes are clinically categorized into a self-limited epilepsy group with good prognosis and high prevalence rate and a pharmacoresistant epilepsy group with poor prognosis despite low prevalence rates. Many children develop pharmacoresistant epilepsy beginning in early infancy and progress to developmental epileptic encephalopathy, which is associated with intellectual, behavioral, and/or motor disabilities. These children do not show remission in epilepsy and are transitioned to the adult service and require comprehensive medical care.
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.
