DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for ciliary body disorder — screening already-approved drugs against its 10-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCiliary body disorder maps to a 10-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 ciliary body disorder 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
endoplasmic reticulum aminopeptidase 1 (ERAP1) — ERAP1 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 pgedrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6RQX · 1.68 Å · ligand TRIETHYLENE GLYCOL (PGE). Experimental structure, not a prediction.
What the evidence adds up to
Chronic intestinal pseudo-obstruction, a rare disorder of ciliary-type motility in the gut, was followed prospectively in 74 adults over an eleven-year period. Median age at onset was 38 years, and median body mass index and serum albumin at consultation were 18.3 kg/m² and 3.8 mg/dL respectively. Total parenteral nutrition was required by 23 patients (31%) and invasive decompression by 18 (24%). Intestinal sterilisation was attempted in 51 patients (69%) and was effective in 33 of them (65%); of those who responded, 28 (85%) were taking metronidazole. Seven patients (9%) used opioids. Nine deaths occurred (12%), five from infection (56%) and two from suicide (22%). Of the deceased, six (67%) had received total parenteral nutrition and four (44%) decompression therapy. Fifty-one patients (69%) expressed a wish for palliative care.
The two review articles on ciliopathies describe a broad group of genetic disorders caused by defects in ciliary structure or function, ranging from organ-specific presentations such as renal, hepatic and pancreatic cysts, neural tube defects, polydactyly, situs inversus and retinal degeneration, to pleiotropic syndromes like Bardet-Biedl and Joubert syndrome. Neither review reports any clinical trial data or therapeutic intervention; both are descriptive summaries of phenotype and diagnosis. No drug efficacy is claimed in any of the three abstracts.
The natural history data show a severe disease with substantial morbidity and mortality, but no randomised or controlled treatment evidence is presented. The only drug mentioned with any signal of benefit is metronidazole, used within an intestinal sterilisation regimen that helped two-thirds of those who received it, but the abstract does not separate its effect from the broader procedure, nor does it report response duration or survival benefit. Opioid use was recorded in a small minority, with no outcome data attached.
What is missing is a prospective, controlled trial of intestinal sterilisation or any antimicrobial regimen in chronic intestinal pseudo-obstruction, with stratification by disease subtype and nutritional status. The palliative care needs of these patients are documented but no interventional study addresses them. No data exist on psychiatric support despite two suicides in the cohort. Funding for a multicentre registry with standardised treatment protocols and long-term follow-up is required before any drug can be recommended for this condition.
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 Neurogastroenterology and Motility · 2023 · 13 citations · open access
Natural History of Chronic Intestinal Pseudo-obstruction and Need for Palliative Care
AbstractBackground/Aims: Natural history of chronic intestinal pseudo-obstruction (CIPO), a rare disease characterized by episodes of non-mechanical obstruction, is unclear in adults. This study evaluates the clinical course of CIPO and palliative care needs of patients. Methods: From October 2010 to September 2021, 74 patients who underwent cine MRI and had a definitive diagnosis of CIPO were prospectively included. We investigated disease etiology and outcomes, age at onset, nutritional status at consultation (body mass index and serum albumin), hydrogen breath test results, and total parenteral nutrition (TPN) during the disease course. Results: , 3.8 mg/dL, and 60%, respectively. TPN and invasive decompression therapy were required by 23 (31%) and 18 (24%) patients, respectively. Intestinal sterilization was performed in 51 (69%) patients and was effective in 33 (65%); of these, 28 (85%) were taking metronidazole. Seven (9%) patients used opioids. There were 9 deaths (12%), including 5 (56%) from infection and 2 (22%) from suicide. Of the deaths, 6 (67%) and 4 (44%) underwent TPN management and decompression therapy, respectively. Fifty-one patients (69%) wanted palliative care. Conclusion: CIPO is a rare, severe, and under-recognized disease. Standardization of treatment strategies, including palliative care and psychiatric interventions, is desired.
Oxford University Press eBooks · 2013 · 7 citations
Ciliopathies
AbstractThe ciliopathies are a group of rare diseases that often affect multiple systems within the body, and are caused by defects in the function or structure of cilia. This resource provides a clinical overview and reference to this newly emergent group of disorders ranging from Alström syndrome to putative ciliopathic disorders. Each chapter provides an in-depth discussion on a specific disorder, including the latest scientific research together with a description of its features, and practical guidelines on diagnosis.
Journal of Pediatric Neurology · 2022 · 0 citations
The Function and Role of the Cilium in the Development of Ciliopathies
AbstractAbstract “Ciliopathies” are a group of genetic disorders described by the malformation or dysfunction of cilia. The disorders of ciliary proteins lead to a range of phenotype from organ-specific (e.g., cystic disease of the kidney, liver, and pancreas, neural tube defects, postaxial polydactyly, situs inversus, and retinal degeneration) to sketchily pleiotropic (e.g., Bardet-Biedl syndrome and Joubert syndrome). The mechanism below the disfunction of cilia to reach new therapeutic strategies.
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.
