DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for syndromic intellectual disability — screening already-approved drugs against its 42-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleSyndromic intellectual disability maps to a 42-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
approvedMedroxyprogesterone AcetateApproved drug
Structures already discussed alongside syndromic intellectual disability in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
bromodomain PHD finger transcription factor (BPTF) — BPTF 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 isopropyl alcohol bound in it, shown as sticks.
Loading structure…
helix sheet ipadrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2RI7 · 1.45 Å · ligand ISOPROPYL ALCOHOL (IPA). Experimental structure, not a prediction.
What the evidence adds up to
No drug treatment is tested or reported in any of these abstracts. One 2011 review states that intellectual disability has been considered an immutable condition and that current medical practice aims only at relieving symptoms, not altering cognitive deficits. The same review notes that scientific advances have raised the possibility that intellectual disability may become treatable, and that fragile X syndrome is the leading candidate for mechanism-based therapy. A 2011 paper on Down syndrome states that the field of cognitive pharmacology for children with intellectual disability does not exist, that no physician could name a single medication used for that purpose in children, and that the mechanisms supporting cognition are still in a discovery phase.
A 2020 review of outcome measures reports that tools assessing intellectual and adaptive functioning have rarely been used as primary outcome measures in treatment trials, and that further study is needed on their usefulness for measuring change. A 2022 paper on Bardet-Biedl syndrome mouse models states there are limited pharmacological interventions for intellectual disability, partly due to poor understanding and the heterogeneous nature of the condition, and that there are also limited mouse models. A 2021 study of a specialist intellectual disability inpatient unit in the UK reports that Health of the Nation Outcome Scales for people with learning disabilities scores decreased significantly from admission to discharge for all patient categories, but this study does not involve any drug intervention.
What is still missing: no drug has been shown to improve core intellectual or adaptive functioning in any human trial for syndromic intellectual disability. The field lacks validated outcome measures sensitive to change, lacks consensus on clinical trial design for children, and lacks sufficient animal models that recapitulate the human condition. Funding for mechanism-based drug development remains limited, and no FDA-sanctioned indication exists for any cognitive-enhancing medication in this population.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
American Journal on Intellectual and Developmental Disabilities · 2020 · 35 citations
Outcome Measures for Core Symptoms of Intellectual Disability: State of the Field
AbstractIntellectual disability (ID) is defined by impairments in intellectual and adaptive functioning. As such, tools designed to assess these domains would theoretically be ideal outcome measures for treatment trials targeting core symptoms of ID. However, measures of intellectual and adaptive functioning have rarely been used as primary outcome measures to date and further study is needed regarding their usefulness to measure change. This area of inquiry is important because promising, mechanism-modifying treatments for conditions leading to ID are being initiated. To show efficacy, these treatments need to demonstrate an impact on core features of ID. After reviewing literature on this topic, we suggest solutions to several problems outlined, including use of out-of-age-range testing, alternative metrics, and development of new measures.
ACS Chemical Neuroscience · 2011 · 19 citations · open access
Fragile X Syndrome: An Update on Developing Treatment Modalities
AbstractIntellectual disability (ID; mental retardation) is considered an immutable condition. Current medical practices are aimed at relieving symptoms and not at altering the underlying cognitive deficits. Scientific advancements from the past decade have led to the exciting possibility that ID may now be treatable. Moreover, pharmaceutical therapies targeting the most common form of inherited ID, Fragile X syndrome (FXS), may become the new benchmark for central nervous system (CNS) drug discovery: seeking cures for neurodevelopmental disorders.
Journal of Applied Research in Intellectual Disabilities · 2021 · 16 citations · open access
An 8‐year study of admissions and discharges to a specialist intellectual disability inpatient unit
AbstractBACKGROUND: In the United Kingdom, policy change has led to specialist intellectual disability inpatient bed reduction. Little evidence exists assessing the results for patients admitted to such units. This study evaluates the outcomes of a specialist intellectual disability inpatient unit. METHOD: Gender/age/ethnicity/intellectual disability severity/co-morbid psychiatric/developmental disorders, treatment length and stay data were collected. The health of the nation outcome scales for people with learning disabilities (HoNOS-LD) scores at admission, treatment completion and discharge were recorded. Analysis of these multiple variables and correlations within different patient groups was investigated using various statistical tests. RESULTS: Of 169/176 patients (2010-2018), admission to discharge, HoNOS-LD global and all individual items score decreased significantly, for all patient categories. Treatment completion to discharge duration was significant for the whole cohort. CONCLUSIONS: This is the largest study of intellectual disability inpatient outcomes. Discharge from the hospital appears not associated with duration of treatment. Using HoNOS-LD to demonstrate treatment effectiveness is recommended.
Cambridge University Press eBooks · 2011 · 4 citations
Pharmacotherapy for children with Down syndrome
AbstractThe field of cognitive pharmacology for children with intellectual disability (ID) does not actually exist. As defined by the level of support for clinical trials, or FDA-sanctioned indications, most physicians would be hard pressed to name a single medication used for such a purpose in children. There are few clinical paradigms and little informed consensus about how to navigate these uncharted waters. Despite our advance into the era of genome-based medicine, the mechanisms that support cognition and its neurobiological organization in the brain are still very much in a discovery phase. New appreciation for the biochemical and physiological mechanisms of synaptic dysfunction in neurogenetic disorders holds promise for developing novel therapeutic approaches to ID (Johnston, 2006). Recent advancements using animal models have led to clinical trials for fragile-X syndrome, which is leading the effort to develop therapies for ID based on mechanistic principles (Hagerman et al., 2009).
Revista Brasileira Ginecologia e Obstetrícia · 2025 · 1 citations · open access
Contraception in adolescents with mental disorders: adherence and satisfaction in the use of depot medroxyprogesterone acetate
AbstractObjective: To evaluate the continuation rate, satisfaction, and reasons for discontinuation of depot medroxyprogesterone acetate (DMPA) in adolescents treated in a mental health service. Methods: Prospective cohort study conducted in a reference unit for the care of adolescents with mental disorders (MDs) and intellectual disabilities (IDs). All patients received a gynecological consultation and an educational group on contraceptive methods. Sociodemographic data on age, education and gynecological data (menarche, coitarche, regularity of menstrual cycles and presence of symptoms) were collected. Follow-up was quarterly for 12 months, during which symptoms, desire to continue, and satisfaction with the use of the quarterly injectable were assessed. Results: Eight hundred and sixty-two sexually active adolescents were supported, 532 adolescents chose to use the quarterly injectable, and 69 of these agreed to participate in the study. The mean age of users was 15.5 years (SD=0.91). After 12 months of follow-up, 34 (49.3%) of the 69 adolescents continued to use the method and 36 (52.3%) were satisfied. Among the 33 (47.8%) who discontinued use, the most common reasons were irregular bleeding and weight gain. Conclusions: Adolescents with intellectual disabilities and/or other mental disorders showed a significant rate of continuation and satisfaction with the use of the depot medroxyprogesterone acetate at 12 months, and the most common reasons for discontinuation were irregular uterine bleeding and weight gain.
Iowa Research Online (The University of Iowa) · 2022 · 0 citations · open access
Behavioral and brain phenotypes of mouse models of Bardet-Biedl Syndrome
AbstractIntellectual disability (ID) is one of the most common neurodevelopmental disorders, affecting 1% of the population globally. Clinically, ID is characterized by a deficit in intellectual functioning and adaptive functioning. There are limited pharmacological interventions for ID, partially due to a poor understanding of ID and the heterogeneous nature of ID In addition, there are limited mouse models of ID.
AbstractIntellectual Disability Medicine, Department of General Practice, Erasmus MC, University Medical Centre Rotterdam, Rotterdam, The Netherlands The manuscript submitted does not contain information about medical device(s)/drug(s). No funds were received in support of this work. No benefits in any form have been or will be received from a commercial party related directly or indirectly to the subject of this manuscript.
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.
