DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for paroxysmal nocturnal hemoglobinuria — screening already-approved drugs against its 6-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleParoxysmal nocturnal hemoglobinuria maps to a 6-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 paroxysmal nocturnal hemoglobinuria 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
complement factor D (CFD) — CFD 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 aminomethyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6QMT · 1.8 Å · ligand 2-[2-[[3-[3-(aminomethyl)phenyl]phenyl]carbonylamino]phenyl]ethanoic acid (J7B). Experimental structure, not a prediction.
What the evidence adds up to
A 1952 case report describes a patient with proven paroxysmal nocturnal hemoglobinuria treated with ACTH on three occasions. The first course failed, the second produced a dramatic but short-lived deceleration of hemolysis that stopped when treatment ended, and the third attempt was again ineffective. Treatment with dicumarol then coincided with a remission that had lasted five weeks at the time of writing. A 1961 review notes that venous thromboses are the most common immediate cause of death and that basic studies had not yet defined the fundamental defect. A 1994 journal article provides no new clinical data. A 2024 critical review states that the first FDA-approved treatment, eculizumab, arrived in 2007, and that many new complement-inhibitor options have since become available on the US market; the review does not report response rates, survival data, or comparative outcomes for any of these drugs.
The 1952 ACTH results are disappointing: only one of three courses produced any benefit, and that benefit was transient. The dicumarol remission is a single observation with no controlled data. The 2024 review, while cataloguing approved drugs, offers no concrete numbers on how many patients respond, for how long, or with what side effects. No abstract reports a randomised trial, a head-to-head comparison, or long-term follow-up of any treatment.
What is still missing is a publicly accessible, quantitative synthesis of trial outcomes for each complement inhibitor—response rates, breakthrough haemolysis rates, survival, and quality-of-life measures. Also absent are cost-effectiveness data and studies that stratify patients by clone size or transfusion dependence, which might identify who benefits most from which drug.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Blood · 1952 · 17 citations · open access
Treatment of Paroxysmal Nocturnal Hemoglobinuria with ACTH
AbstractAbstract A proven case of paroxysmal nocturnal hemoglobinuria is reported in which the patient was treated with ACTH on three occasions. Following an initial failure, the second course of ACTH resulted in a dramatic deceleration of hemolysis which, however, lasted only a short time after the cessation of treatment. A third attempt was again of no avail in relieving the condition. Treatment with dicumarol coincided with a remission of the hemolysis which has lasted, so far, for five weeks.
New England Journal of Medicine · 1961 · 13 citations
Paroxysmal Nocturnal Hemoglobinuria
AbstractPAROXYSMAL nocturnal hemoglobinuria is a disease with a fascinating diversity of challenges and implications. These have prompted numerous clinical and basic investigations. The clinical features are often bizarre and may be misleading, even with the most typical presentation of abdominal pain, fatigue, weakness, anemia and dark urine and with venous thromboses as the most common immediate cause of death.1 , 2 The basic studies have not yet succeeded in defining the fundamental defect.Described below are the clinical and laboratory findings on a patient whose course illustrates exceptionally well most of the vicissitudes of this disorder and the pitfalls in correct diagnosis. . . .
Therapeutic Advances in Hematology · 2024 · 5 citations · open access
Current landscape of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitors and regulators
AbstractParoxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder which is caused by mutations in phosphatidylinositol glycan class A leading to hemolysis of red blood cells via complement inhibition. The first treatment for PNH, eculizumab, was FDA approved in 2007. Since then, many new treatment options for PNH have arisen. This critical review will examine all medications available for PNH on the US market, highlight several major medications in development, and discuss the risks and treatment considerations associated with each option. It is not intended to address PNH clonal dynamics, disease presentation, or discussions on when to initiate treatment.
AbstractJournal Article Paroxysmal Nocturnal Hemoglobinuria Get access Rodger L. Bick, MD Rodger L. Bick, MD Presbyterian Comprehensive Cancer Center, Presbyterian Hospital of Dallas. Reprint requests to Dr Bick, Presbyterian Hospital of Dallas, 8200 Walnut Hill Ln, Dallas, TX 75231. Search for other works by this author on: Oxford Academic PubMed Google Scholar Laboratory Medicine, Volume 25, Issue 3, 1 March 1994, Pages 148–151, https://doi.org/10.1093/labmed/25.3.148 Published: 01 March 1994
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.
