DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for familial hemiplegic migraine — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleFamilial hemiplegic migraine maps to a 2-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 familial hemiplegic migraine 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
sodium voltage-gated channel alpha subunit 1 (SCN1A) — SCN1A 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 3beta,14beta,17beta,25rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7DTD · 3.3 Å · ligand (3beta,14beta,17beta,25R)-3-[4-methoxy-3-(methoxymethyl)butoxy]spirost-5-en (9Z9). Experimental structure, not a prediction.
What the evidence adds up to
Migraine is a syndrome with multiple causes. In families with hemiplegic migraine, mutations have been found in two different genes. Familial aggregation and twin studies in families with migraine without aura and migraine with aura show that genetic factors are important, although the mode of inheritance is not always clear. Genome-wide scans have identified susceptibility loci for these common syndromes, but none of the studies performed to date provide convincing evidence for a gene responsible for the common varieties of migraine. The heterogeneity of familial hemiplegic migraine underscores the likely heterogeneity of the more common types of migraine.
A study at the Danish Headache Center compared the clinical characteristics of 105 patients with sporadic hemiplegic migraine with those of patients with migraine with typical aura and patients with familial hemiplegic migraine. No drug was tested in any of these studies. No treatment was evaluated.
Migraine prevalence is highest between the ages of 25 and 55, often peaking in the late 30s and early 40s. The American Migraine Study showed that migraine prevalence was lower in African-Americans than Asians. Migraine is comorbid with depression, anxiety disorders, manic depressive illness, and epilepsy. Diagnosis is complicated by the episodic and heterogeneous nature of the illness.
What is still missing is any drug-repurposing trial for familial hemiplegic migraine, any patient stratification based on the known gene mutations, and the funding to conduct such work.
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 Neurology Neurosurgery & Psychiatry · 1996 · 20 citations · open access
Familial hemiplegic migraine in the west of Scotland: a clinical and genetic study of seven families.
AbstractOBJECTIVES: Clinical and genetic characterisation of families in the west of Scotland with familial hemiplegic migraine. METHODS: Families with familial hemiplegic migraine were identified via probands attending the regional paediatric neurology and child development centre. All available family members were assessed clinically and genetic linkage studies for the known familial hemiplegic migraine gene locus on chromosome 19 were carried out on three families. RESULTS: Seven unrelated kindreds with familial hemiplegic migraine were identified. Clinical information was obtained on 138 family members, 27 of whom fulfilled the International Headache Society criteria for familial hemiplegic migraine. Whereas the severity, duration, frequency, and temporal progression of acute hemiplegic migrainous attacks showed pronounced variability within and between families, and even in the same individual over time, no true clinical heterogeneity of the condition was apparent. Genetic linkage analysis gave results consistent with linkage to the familial hemiplegic migraine gene locus on chromosome 19p in one family. In the other two families, evidence against linkage was obtained. There was no significant clinical difference between these three families. CONCLUSIONS: This study provides characterisation of the clinical features of familial hemiplegic migraine in a British population. Significant variability was found in the frequency and character of migraine attacks within and between families, and no true clinical heterogeneity was identified. On the other hand, further evidence for genetic heterogeneity of the condition was found.
AbstractMigraine is not a disease but a syndrome that undoubtedly has multiple causes. Several migraine syndromes have a clear autosomal dominant pattern of inheritance, and recently, mutations have been found in two different genes in families with hemiplegic migraine. Familial aggregation and twin studies in families with other more common migraine syndromes (i.e., migraine without aura and migraine with aura) show that genetic factors are important, although the mode of inheritance is not always clear. Numerous candidate genes have been identified based on clinical features of migraine and the known functions of the different genes. Recently genome-wide scans in families with migraine without aura and migraine with aura have identified susceptibility loci for these common syndromes. However, none of the numerous studies performed to date provide convincing evidence for a gene responsible for the common varieties of migraine.
Galter Health Sciences Library, Northwestern University · 2003 · 0 citations · open access
Sporadic Hemiplegic Migraine: A Separate Entity
AbstractThe clinical characteristics of 105 patients with sporadic hemiplegic migraine (SHM) were compared with those of patients with migraine with typical aura (MA) and patients with familial hemiplegic migraine (FHM) in a study at the Danish Headache Center, Glostrup Hospital, Gentofte Hospital, University of Copenhagen, and the John F Kennedy Institute, Denmark.
Cambridge University Press eBooks · 2001 · 0 citations
Migraine
AbstractMigraine headache is an extremely common and temporarily disabling headache disorder. Most studies on migraine prevalence have reported variation by age and gender. Prevalence is generally highest between the ages of 25 and 55, often with a peak in the late 30s and early 40s. Migraine was once considered to be a disease of the affluent. The American Migraine Study showed that migraine prevalence was lower in African-Americans than Asians. Familial aggregation of migraine has long been recognized and genetic studies have generally supported a role for both genetic and environmental risk factors in the etiology of migraine. The heterogeneity of familial hemiplegic migraine (FHM) underscores the likely heterogeneity of the more common types of migraine. Diagnosis of migraine is complicated by the episodic and heterogeneous nature of the illness. Population-based studies demonstrate that migraine is comorbid with depression, anxiety disorders, and manic depressive illness as well as epilepsy.
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.
