DeCure's autonomous Psychiatry AI scientist is researching a drug-repurposing hypothesis for autism spectrum disorder — screening already-approved drugs against its 40-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleAutism spectrum disorder maps to a 40-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
approvedAmantadineApproved drugapprovedMemantineApproved drug
Structures already discussed alongside autism spectrum disorder in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
Influenza A M2 transmembrane domain — Amantadine has a real, experimentally solved structure in complex with this target (PDB 6BKK, 1.995 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet 308drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6BKK · 1.995 Å · ligand Amantadine (308). Experimental structure, not a prediction.
What the evidence adds up to
A 1999 selective review of treatment programmes for children with autism found that, according to the American Psychological Association’s Division 12 Task Force criteria, there were no well-established or probably efficacious treatments for autism. The review covered the UCLA Young Autism Project, Project TEACCH, LEAP, applied behaviour analysis programmes, and the Denver Health Science Program, as well as programmes with little or no empirical support such as facilitated communication and auditory integration therapy. Although virtually all programmes showed substantial developmental gains, particularly in measured IQ, none met conventional standards for a well-established treatment.
A 2019 systematic review of 406 controlled clinical trials for autism spectrum disorder published between 1980 and 2016 identified 327 different outcome measures. Only seven scales were used in more than 5% of the studies, and only three of those measured core symptoms: the Autism Diagnostic Observation Schedule, the Childhood Autism Rating Scale, and the Social Responsiveness Scale. Sixty-nine percent of the tools were used in the literature only once. The authors concluded that the evaluation of efficacy in intervention trials relies on heterogeneous and often non-specific tools, and that this fragmentation significantly hampers comparisons between studies and the discovery of effective treatments.
A 2014 paper noted that measuring treatment response in children with autism spectrum disorder is difficult because symptom changes are heterogeneous and can be subtle, especially over short periods. The paper called for continued development of treatment outcome measures to help identify and compare efficacious interventions and to tailor treatments.
What is still missing is a consensus on which outcome measures to use in clinical trials, so that results from different studies can be meaningfully compared. Without agreement on how to measure core symptoms, it remains difficult to determine whether any intervention is effective, and the field lacks the standardised tools needed to identify which subgroups of children might benefit from which treatments.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
School Psychology Review · 1999 · 102 citations
A Selective Review of Treatments for Children with Autism: Description and Methodological Considerations
AbstractAutism is a developmental disorder whose behavioral characteristics range on a continuum from mild to severe. Autism is typically not diagnosed prior to age 2 to 3 years and the prognosis for this pervasive developmental disorder is poor. Although there is no documented “cure” for autism, research suggests that it can be managed effectively using comprehensive behavioral and educational treatment programs. This article reviews and critiques several of the most visible and most frequently cited treatment programs for children with autism: the UCLA Young Autism Project, Project TEACCH, LEAP, applied behavior analysis programs, and the Denver Health Science Program. Treatment programs having little or no empirical support such as facilitated communication, auditory integration therapy, and sensory integration therapy also are briefly reviewed. We evaluate the empirical evidence for the efficacy and effectiveness of these programs using conventional standards of research design and methodology and the Division 12 Task Force on Empirically Supported Treatments for Childhood Disorders of the American Psychological Association. Based on these Task Force criteria, there are no well-established or probably efficacious treatments for autism, although virtually all programs show substantial developmental gains, particularly in measured IQ. Recommendations for future research and practice are offered with guidelines for evaluating treatment programs for children with autism.
What are we targeting when we treat autism spectrum disorder? A systematic review of 406 clinical trials
AbstractThe number of trials aimed at evaluating treatments for autism spectrum disorder has been increasing progressively. However, it is not clear which outcome measures should be used to assess their efficacy, especially for treatments which target core symptoms. The present review aimed to provide a comprehensive overview regarding the outcome measures used in clinical trials for people with autism spectrum disorder. We systematically searched the Web of Knowledge SM database between 1980 and 2016 to identify published controlled trials investigating the efficacy of interventions in autism spectrum disorder. We included 406 trials in the final database, from which a total of 327 outcome measures were identified. Only seven scales were used in more than 5% of the studies, among which only three measured core symptoms (Autism Diagnostic Observation Schedule, Childhood Autism Rating Scale, and Social Responsiveness Scale). Of note, 69% of the tools were used in the literature only once. Our systematic review has shown that the evaluation of efficacy in intervention trials for autism spectrum disorder relies on heterogeneous and often non-specific tools for this condition. The fragmentation of tools may significantly hamper the comparisons between studies and thus the discovery of effective treatments for autism spectrum disorder. Greater consensus regarding the choice of these measures should be reached.
Expert Opinion on Pharmacotherapy · 2001 · 64 citations
Pharmacotherapeutic management of autism
AbstractThere are no medications that are specifically marketed for the treatment of autism. There does exist, however, an extensive body of literature describing both open-label and controlled studies of medications in the treatment of both children and adults with autism. Some of the better-studied medications (including haloperidol and risperidone) are often efficacious in treating associated symptoms of autism but can also cause unacceptable adverse effects. Early studies of serotonin re-uptake inhibitors appear promising but may not be indicated for all age groups. Small, controlled studies of methylphenidate and clonidine indicate a possible role for these medications in the treatment of hyperactivity in autism. No medications have been proven to be efficacious in the treatment of the core social or communication impairment seen in autism. Current pharmacological management is best aimed at target symptoms that have been demonstrated to respond to medication in treatment studies.
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AbstractSignificant advancements have been made in early intervention programs for children with Autism spectrum disorder (ASD). However, measuring treatment response for children with ASD is difficult due to the heterogeneity of changes in symptoms, which can be subtle, especially over a short period of time. Here we outline the challenge of evaluating treatment response with currently available measures as well as newly developed or refined measures that may be useful in clinical trials for young children with ASD. Continued development of treatment outcome measures will help the field identify and compare efficacious interventions and tailor treatments for children with ASD.
AbstractAnecdotal reports and results from open trials have suggested that amantadine, a drug used for influenza prophylaxis and Parkinson's disease and with proven safety in children, lessens hyperactivity and aggressivity in children with various diagnoses. To test the efficacy of amantadine in autism, researchers using a double-blind research protocol randomized 39 …
AUT824103_Supplemental_material_3 – Supplemental material for Efficacy and safety of memantine in children with autism spectrum disorder: Results from three phase 2 multicenter studies
AbstractSupplemental material, AUT824103_Supplemental_material_3 for Efficacy and safety of memantine in children with autism spectrum disorder: Results from three phase 2 multicenter studies by Antonio Y Hardan, Robert L Hendren, Michael G Aman, Adelaide Robb, Raun D Melmed, Kristen A Andersen, Rachel Luchini, Rezwanur Rahman, Sanjida Ali, X Daniel Jia, Madhuja Mallick, Jordan E Lateiner, Robert H Palmer and Stephen M Graham in Autism
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.
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