DeCure for Neurodevelopmental disorder with cardiomyopathy, spasticity, and brain abnormalities
DeCure's autonomous Psychiatry AI scientist is researching a drug-repurposing hypothesis for neurodevelopmental disorder with cardiomyopathy, spasticity, and brain abnormalities — 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 moduleNeurodevelopmental disorder with cardiomyopathy, spasticity, and brain abnormalities 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 neurodevelopmental disorder with cardiomyopathy, spasticity, and brain abnormalities 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
serine hydroxymethyltransferase 2 (SHMT2) — SHMT2 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 plpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6QVL · 2.28 Å · ligand PYRIDOXAL-5'-PHOSPHATE (PLP). Experimental structure, not a prediction.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
S S Korsakov Journal of Neurology and Psychiatry · 2021 · 6 citations
Neurodevelopmental disorders in children and the possibilities of their pharmacotherapy
AbstractNeurodevelopmental disorders (NDD) are characterized by disturbances of the formation of cognitive functions, communication skills, behavior characteristics and/or motor skills, which are caused by abnormalities in the course of the processes of neuroontogenesis. In the clinical practice of a pediatric neurologist and pediatrician, a significant part consists of patients with NDD without a general decrease in intelligence, primarily with speech development disorders, attention deficit hyperactivity disorder (ADHD), specific learning disorders (dyslexia, dysgraphia, dyscalculia). NDD represent a heterogeneous group of diseases, having multifactorial origin and a neurobiological nature, which are caused by genetic mechanisms and early (perinatal) brain damage. Among children with NDD, there is a higher occurrence of anxiety disorders compared to their peers. With NDD, early intervention is indicated, and its positive effect is possible during the period when the brain is most plastic and capable of changes. The published results of multicenter, double-blind, placebo-controlled, randomized clinical trials of pharmacotherapy with the medication "Tenoten for children" for ADHD, specific learning disorders, anxiety disorders and the consequences of perinatal damage to the central nervous system are reviewed.
Acquired Mutations Link Congenital Heart Disease, Neurodevelopmental Disorders
AbstractBack to table of contents Previous article Next article Clinical and Research NewsFull AccessAcquired Mutations Link Congenital Heart Disease, Neurodevelopmental DisordersNick ZagorskiNick ZagorskiPublished Online:4 Feb 2016https://doi.org/10.1176/appi.pn.2016.1b16AbstractAbout 20 percent of children with severe congenital heart disease have an excess of mutations that are also commonly found in children with developmental problems such as autism.A study published December 4 in Science suggests that neurodevelopmental abnormalities in children with congenital heart disease may arise from the acquisition of harmful de novo gene mutations. These are not inherited but rather spontaneously occur during fetal development. These abnormalities include cognitive, motor, social, and language impairments.Michelle Massi"Finding this strong association between the developing heart and brain was a bit surprising, for while the heart and brain are both complex organs, they do separate early in the development process," said study coauthor Jonathan Kaltman, M.D., who is the administrator of the National Heart, Lung, and Blood Institute's (NHLBI) Bench to Bassinet Program, which funded this study."At the same time, it makes sense clinically, given that there are several inherited disorders such as Down's syndrome or DiGeorge syndrome, which feature both cardiac and neurological problems."Investigators from the NHLBI's Pediatric Cardiac Genomics Consortium used a technique known as exome sequencing to scan all the protein-encoding regions of the genome in over 1,200 affected family trios (a child with congenital heart disease [CHD] and his or her parents) along with 900 control trios without CHD.They uncovered almost 400 potentially harmful de novo mutations that significantly contribute to the risk of co-occurring CHD and neurodevelopmental disorders.While an excess number of these harmful mutations were found in only 2 percent of patients who had isolated CHD, the prevalence rose in children with more complications. The mutations were present in about 10 percent of patients who also had neurodevelopmental disorders or other extracardiac congenital anomalies (such as limb or kidney defects), and 20 percent of patients who had both.When the researchers compared the genetic data from children with CHD and children with neurodevelopmental disorders but no heart problems, they found 85 mutations were present in both groups—more than twice as many as would be expected by random chance."These findings show enough statistical risk of developing neurodevelopmental disabilities when these particular gene mutations are present that we might consider developing a genetic test for patients with CHD," Kaltman told Psychiatric News. Such a test could help to identify high-risk children for early interventions that might help to limit developmental delays and improve functioning, he said.In addition to the NHLBI, this study was supported in part by the National Human Genome Research Institute, the Howard Hughes Medical Institute, and Simons Foundation for Autism Research. ■An abstract of "De Novo Mutations in Congenital Heart Disease With Neurodevelopmental and Other Congenital Anomalies" can be accessed here. ISSUES NewArchived
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.
