DeCure's autonomous Respiratory AI scientist is researching a drug-repurposing hypothesis for cystic fibrosis — screening already-approved drugs against its 47-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCystic fibrosis maps to a 47-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
approvedIbuprofenCyclooxygenase inhibitor
Structures already discussed alongside cystic fibrosis in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
Crystal structure of the complex formed between phospholipase A2 and 2-(4-isobutyl-phenyl)-propionic acid at 2.2 A resolution — Ibuprofen has a real, experimentally solved structure in complex with this target (PDB 2PWS, 2.21 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 ibpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2PWS · 2.21 Å · ligand Ibuprofen (IBP). Experimental structure, not a prediction.
What the evidence adds up to
Cystic fibrosis has no known cause and no curative treatment; management has historically been supportive, focused on secondary complications. A 1972 guide to drug therapy in cystic fibrosis, composed of 19 minimonographs by clinic directors and consultants, stated plainly that no absolute answers to drug therapy questions could be provided, and that the sections reflected personal opinions. A 1966 report claimed that a comprehensive treatment program used for more than eight years was effective in preventing irreversible pulmonary changes and delaying disease progression, but only if therapy began before the stage of irreversibility. A 2000 review noted that cystic fibrosis is one of the commonest lethal inherited conditions among Caucasians, that life expectancy had improved as treatment became more intensive, but that current treatments were expensive, time-consuming, and could affect quality of life.
By 2017, standards of care had become so strongly entrenched that traditional placebo-controlled studies in cystic fibrosis populations likely to benefit from new therapies had become less feasible. Three therapeutic areas — pulmonary exacerbations, Pseudomonas aeruginosa airway infections, and reduced CFTR protein function — all faced the same challenge: patients and clinicians were much less likely to accept simple placebo-controlled studies. Active-comparator trial designs introduced their own problems, including selecting valid comparators, estimating a comparator's current clinical efficacy for noninferiority testing, and effectively blinding commercially available drugs. A 2021 first-person account from a person with cystic fibrosis born in 1983 described treatment as central to daily life and burdensome, though hope was noted with continuing treatment advancement.
What is still missing is a trial design that can reliably test new therapies against entrenched standards of care without relying on placebo controls that patients and clinicians will no longer accept. The cost and time burden of existing treatments, and the need to stratify patients by disease stage before irreversible changes occur, also remain unresolved. No drug not mentioned in these abstracts is discussed, and no efficacy claim is made for any specific agent.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Archives of Pediatrics and Adolescent Medicine · 1972 · 15 citations
Guide to Drug Therapy in Patients With Cystic Fibrosis.
AbstractThis valuable guide is a series of 19 minimonographs dealing with various aspects of the treatment of patients with cystic fibrosis, ranging from antibiotic therapy to the use of vaccines. Each of the 25 contributors is either a director of a cystic fibrosis clinic or is associated with one in a consultant capacity. Since the basic cause of cystic fibrosis is yet unknown, and treatment to date has been of necessity supportive and concerned with the secondary complications of the disease, no absolute answer to the many questions pertaining to drug therapy can be provided. The sections reflect the personal opinions of each of the contributors with the influence of the editor, Dr. Huang, with her considerable experience evident throughout. Drug dosages and schedules of administration are very well detailed and the references to support their use are current. The antibiotic therapy section, in particular, is well done. Several minor
BMC Pediatrics · 2004 · 13 citations · open access
High-dose ibuprofen therapy associated with esophageal ulceration after pneumonectomy in a patient with cystic fibrosis: a case report
AbstractBACKGROUND: Lung disease in patients with cystic fibrosis is thought to develop as a result of airway inflammation, infection, and obstruction. Pulmonary therapies for cystic fibrosis that reduce airway inflammation include corticosteroids, rhDNase, antibiotics, and high-dose ibuprofen. Despite evidence that high-dose ibuprofen slows the progression of lung disease in patients with cystic fibrosis, many clinicians have chosen not to use this therapy because of concerns regarding potential side effects, especially gastrointestinal bleeding. However, studies have shown a low incidence of gastrointestinal ulceration and bleeding in patients with cystic fibrosis who have been treated with high-dose ibuprofen. CASE PRESENTATION: The described case illustrates a life-threatening upper gastrointestinal bleed that may have resulted from high-dose ibuprofen therapy in a patient with CF who had undergone a pneumonectomy. Mediastinal shift post-pneumonectomy distorted the patient's esophageal anatomy and may have caused decreased esophageal motility, which led to prolonged contact of the ibuprofen with the esophagus. The concentrated effect of the ibuprofen, as well as its systemic effects, probably contributed to the occurrence of the bleed in this patient. CONCLUSIONS: This report demonstrates that gastrointestinal tract anatomical abnormalities or dysmotility may be contraindications for therapy with high-dose ibuprofen in patients with cystic fibrosis.
Current Opinion in Pulmonary Medicine · 2017 · 10 citations
Innovating cystic fibrosis clinical trial designs in an era of successful standard of care therapies
AbstractPURPOSE OF REVIEW: Evolving cystic fibrosis 'standards of care' have influenced recent cystic fibrosis clinical trial designs for new therapies; care additions/improvements will require innovative trial designs to maximize feasibility and efficacy detection. RECENT FINDINGS: Three cystic fibrosis therapeutic areas (pulmonary exacerbations, Pseudomonas aeruginosa airway infections, and reduced cystic fibrosis transmembrane conductance regulator [CFTR] protein function) differ with respect to the duration for which recognized 'standards of care' have been available. However, developers of new therapies in all the three areas are affected by similar challenges: standards of care have become so strongly entrenched that traditional placebo-controlled studies in cystic fibrosis populations likely to benefit from newer therapies have become less and less feasible. Today, patients/clinicians are more likely to entertain participation in active-comparator trial designs, that have substantial challenges of their own. Foremost among these are the selection of 'valid' active comparator(s), estimation of a comparator's current clinical efficacy (required for testing noninferiority hypotheses), and effective blinding of commercially available comparators. SUMMARY: Recent and future cystic fibrosis clinical trial designs will have to creatively address this collateral result of successful past development of effective cystic fibrosis therapies: patients and clinicians are much less likely to accept simple, placebo-controlled studies to evaluate future therapies.
International Journal of Clinical Practice · 2000 · 8 citations
THE CURRENT MANAGEMENT OF CYSTIC FIBROSIS
AbstractSUMMARY Cystic fibrosis (CF) is one of the commonest lethal inherited conditions among Caucasians. It affects multiple organ systems and exhibits a range of clinical problems of varying severity. Life expectancy has improved in recent years as treatment regimes have become more intensive, but current treatments are expensive, often time consuming and may affect quality of life. This review summarises the treatments currently used in the management of patients with CF, and the evidence for these. ( Int J Clin Pract 2000; 54(3) : 171‐179)
AbstractA comprehensive program of treatment for patients with cystic fibrosis has been used for more than eight years and has been shown to be effective in preventing the development of irreversible pulmonary changes and in delaying progression of the disease. To be optimally effective, therapy must be instituted before the stage of irreversibility is reached.
The treatment burden of cystic fibrosis: a day-to-day experience with treatment as someone with cystic fibrosis
AbstractJessica Maetz, who was born in 1983 and now lives in Paris, talks about her experiences of life with cystic fibrosis (CF) and how treatment is central to many aspects of her life. Jessica has also produced a short film that gives a day in the life view of her treatments (see the supplementary material). Treatment for cystic fibrosis interweaves with daily life and can be burdensome, but hope is ever present with the continuing advancement of treatment. <https://bit.ly/36PZnSL>
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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