DeCure's autonomous Dermatology AI scientist is researching a drug-repurposing hypothesis for primary ciliary dyskinesia — screening already-approved drugs against its 17-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePrimary ciliary dyskinesia maps to a 17-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 primary ciliary dyskinesia 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
sperm associated antigen 1 (SPAG1) — SPAG1 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 adpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9HB4 · 3.56 Å · ligand ADENOSINE-5'-DIPHOSPHATE (ADP). Experimental structure, not a prediction.
What the evidence adds up to
In a 2022 participatory study of people with primary ciliary dyskinesia, the combination of tests recommended by guidelines — nasal nitric oxide measurement, genetic testing, and biopsy for electron or video microscopy — was used to diagnose only a few patients in the past. The study assessed the current diagnostic situation internationally but did not report any new treatment or drug.
A 2020 study developed a quantitative super-resolution microscopy workflow to improve diagnosis of motile ciliopathies including PCD. The method uses three-dimensional structured illumination microscopy to build a localisation map of PCD proteins, stochastic optical reconstruction microscopy to analyse axonemal structure, and a high-throughput method to detect motile cilia uncoordination by rotational polarity. The authors concluded that super-resolution methods are powerful tools for improving diagnosis, but the study did not test any drug or therapy.
A 2016 article noted that PCD is a rare disease causing symptoms similar to common respiratory diseases, and that late diagnosis is common after damage to the respiratory system has already occurred. It described available diagnostic methods based on literature searches and clinical experience, but did not report any drug trial or treatment outcome.
What is still missing is a drug or therapy tested in any of these studies. No abstract reports a clinical trial, a repurposed drug, or any measure of treatment efficacy. The research remains focused on diagnostic tools. What is needed is funding for trials that test specific compounds, a trial design that can measure lung function decline or symptom improvement in a rare disease, and better patient stratification to account for the genetic heterogeneity of PCD.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Science Translational Medicine · 2020 · 33 citations
A quantitative super-resolution imaging toolbox for diagnosis of motile ciliopathies
AbstractAirway clearance of pathogens and particulates relies on motile cilia. Impaired cilia motility can lead to reduction in lung function, lung transplant, or death in some cases. More than 50 proteins regulating cilia motility are linked to primary ciliary dyskinesia (PCD), a heterogeneous, mainly recessive genetic lung disease. Accurate PCD molecular diagnosis is essential for identifying therapeutic targets and for initiating therapies that can stabilize lung function, thereby reducing socioeconomic impact of the disease. To date, PCD diagnosis has mainly relied on nonquantitative methods that have limited sensitivity or require a priori knowledge of the genes involved. Here, we developed a quantitative super-resolution microscopy workflow: (i) to increase sensitivity and throughput, (ii) to detect structural defects in PCD patients' cells, and (iii) to quantify motility defects caused by yet to be found PCD genes. Toward these goals, we built a localization map of PCD proteins by three-dimensional structured illumination microscopy and implemented quantitative image analysis and machine learning to detect protein mislocalization, we analyzed axonemal structure by stochastic optical reconstruction microscopy, and we developed a high-throughput method for detecting motile cilia uncoordination by rotational polarity. Together, our data show that super-resolution methods are powerful tools for improving diagnosis of motile ciliopathies.
Tidsskrift for Den norske legeforening · 2016 · 1 citations · open access
Primær ciliedyskinesi
AbstractPrimary ciliary dyskinesia (PCD) is a rare disease, but causes symptoms that resemble far more common respiratory diseases. Late diagnosis is common, when damage to the respiratory system has already occurred. This article aims to elucidate the condition and the diagnostic methods available. The article is based on literature searches in PubMed and the author's own experience of patient treatment and clinical research.
Diagnostic testing in people with primary ciliary dyskinesia around the world: where do we stand?
AbstractIntroduction In the past, only few patients with primary ciliary dyskinesia (PCD) were diagnosed with the test combination recommended by guidelines (nasal nitric oxide (nNO), genetic testing, and biopsy for electron or video microscopy) [Halbeisen, ERJ, 2019]. In a large international participatory study of people with PCD, we assessed the current situation.
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.
