DeCure for Megalencephalic leukoencephalopathy with subcortical cysts
DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for megalencephalic leukoencephalopathy with subcortical cysts — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMegalencephalic leukoencephalopathy with subcortical cysts maps to a 1-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 megalencephalic leukoencephalopathy with subcortical cysts 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.
What the evidence adds up to
The 2022 supplemental material from a study of 22 Egyptian children with megalencephalic leukoencephalopathy with subcortical cysts (MLC) provides clinicoradiologic correlations but does not report any drug treatment or clinical trial. The 2025 case report describes a 10-month-old female from a nonconsanguineous Agarwal family in North India who presented with macrocephaly and developmental delay. MRI showed classic features of MLC, including diffuse white matter changes and subcortical cysts, especially in the anterior temporal lobes. The report emphasises early diagnosis in genetically predisposed communities but offers no therapeutic intervention or outcome data beyond the initial presentation.
No abstract in this set tests or even mentions a drug. There is no evidence of any pharmacological treatment being administered, no survival data, no response rates, and no sample size for any intervention. The only concrete numbers are the 22 children in the Egyptian series and the single case in the Indian report.
The available literature on MLC remains purely descriptive. What is missing is any funded clinical trial, any drug-repurposing study, any attempt to stratify patients by genotype or disease stage, and any systematic collection of outcome measures after a treatment. Without these, no claim about efficacy can be made.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Sage Journals Data · 2022 · 0 citations · open access
sj-docx-1-jcn-10.1177_08830738221078683 - Supplemental material for Clinicoradiologic Correlation in 22 Egyptian Children With Megalencephalic Leukoencephalopathy With Subcortical Cysts
AbstractSupplemental material, sj-docx-1-jcn-10.1177_08830738221078683 for Clinicoradiologic Correlation in 22 Egyptian Children With Megalencephalic Leukoencephalopathy With Subcortical Cysts by Abdelrahim A. Sadek, Mohammed A. Aladawy, Tarek M. M. Mansour, Mohamed F. Ibrahim, Montaser M. Mohamed, Eman F. Gad, Amr A. Othman, Hosny A. Ahmed, Abdin K. Kasim, Wael M. Wagdy, Mohamed H. T. Hasan and Elsayed Abdelkreem in Journal of Child Neurology
INDIGO (University of Illinois at Chicago) · 2022 · 0 citations · open access
sj-docx-2-jcn-10.1177_08830738221078683 - Supplemental material for Clinicoradiologic Correlation in 22 Egyptian Children With Megalencephalic Leukoencephalopathy With Subcortical Cysts
AbstractSupplemental material, sj-docx-2-jcn-10.1177_08830738221078683 for Clinicoradiologic Correlation in 22 Egyptian Children With Megalencephalic Leukoencephalopathy With Subcortical Cysts by Abdelrahim A. Sadek, Mohammed A. Aladawy, Tarek M. M. Mansour, Mohamed F. Ibrahim, Montaser M. Mohamed, Eman F. Gad, Amr A. Othman, Hosny A. Ahmed, Abdin K. Kasim, Wael M. Wagdy, Mohamed H. T. Hasan and Elsayed Abdelkreem in Journal of Child Neurology
Sage Journals Data · 2022 · 0 citations · open access
sj-docx-2-jcn-10.1177_08830738221078683 - Supplemental material for Clinicoradiologic Correlation in 22 Egyptian Children With Megalencephalic Leukoencephalopathy With Subcortical Cysts
AbstractSupplemental material, sj-docx-2-jcn-10.1177_08830738221078683 for Clinicoradiologic Correlation in 22 Egyptian Children With Megalencephalic Leukoencephalopathy With Subcortical Cysts by Abdelrahim A. Sadek, Mohammed A. Aladawy, Tarek M. M. Mansour, Mohamed F. Ibrahim, Montaser M. Mohamed, Eman F. Gad, Amr A. Othman, Hosny A. Ahmed, Abdin K. Kasim, Wael M. Wagdy, Mohamed H. T. Hasan and Elsayed Abdelkreem in Journal of Child Neurology
Annals of African Medicine · 2025 · 0 citations · open access
Agrawal Disease (Megalencephalic Leukoencephalopathy with Subcortical Cysts): A Rare Neurological Case Report
AbstractABSTRACT: Megalencephalic leukoencephalopathy with subcortical cysts (MLC) is a rare leukodystrophy marked by early-onset macrocephaly, delayed milestones and progressive motor decline. It shows characteristic magnetic resonance imaging (MRI) findings, including diffuse white matter changes and subcortical cysts, especially in the anterior temporal lobes. We report a case of a 10-month-old female from a nonconsanguineous Agarwal family in North India, presenting with macrocephaly and developmental delay. MRI revealed classic features of MLC. The case highlights the importance of early diagnosis even in nonconsanguineous families within genetically predisposed communities.
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.
