DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for infantile liver failure — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleInfantile liver failure maps to a 2-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 infantile liver failure 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.
What the evidence adds up to
Neonatal liver failure is a rare clinical syndrome distinct from acute liver failure in older children and adults, with different causes, presentation, and treatment interventions. A 1996 review defined the syndrome, provided a literature-supported differential diagnosis, and outlined a diagnostic work-up based on that differential. It reviewed the role of mitochondrial-based diseases and advances in the diagnosis and management of neonatal hemochromatosis, and summarised specific therapies associated with the unique range of diseases that cause neonatal liver failure. A 2010 review presented a case of neonatal hemochromatosis, reviewed genetic and metabolic causes, updated the differential diagnosis, and proposed a comprehensive initial work-up and current treatments for neonatal hemochromatosis. A 2022 report described three cases of liver failure admitted to a neonatal intensive care unit and reviewed the diagnostic approach and management of liver failure in this age group, noting a paucity of literature and several newly identified conditions that merit discussion.
No abstract reports any clinical trial, any drug tested in a controlled setting, or any quantitative outcome such as survival or response rates. The 1996 review mentions that the unique range of diseases causing neonatal liver failure are associated with specific therapies, but does not name any drug, report any efficacy data, or describe any patient outcomes. The 2010 and 2022 reviews similarly discuss diagnostic work-up and management without providing any numerical results from treatment. There is no evidence from these abstracts that any drug has been shown to improve survival or reverse liver failure in this population.
What is missing is any controlled clinical data. No trial has been reported that tests a specific drug for neonatal liver failure, no sample sizes or survival figures are given, and no patient stratification is described. The literature consists entirely of reviews and case reports, with no quantitative evidence to support any particular treatment.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Current Opinion in Pediatrics · 1996 · 38 citations
Neonatal liver failure
AbstractNeonatal liver failure (NLF) is a distinct and unusual clinical syndrome that requires a specialized approach to its management. This review establishes a definition of NLF and provides a literature-supported differential diagnosis. A specific approach to the diagnostic work-up of an infant with NLF based on this differential diagnosis is outlined. The role of mitochondrial-based diseases in the genesis of NLF is reviewed as well as current advances in the diagnosis and management of neonatal hemochromatosis. The unique range of diseases that cause NLF are associated with specific therapies, which are summarized.
Fulminant Hepatitis After 10 Days of Acetaminophen Treatment at Recommended Dosage in an Infant
AbstractAcetaminophen is considered a safe drug for children, although hepatotoxicity may develop after overdosing. Reports of liver failure after repeated therapeutic doses of the drug have been rare. Here we describe the case of an infant who developed acute liver failure after administration of acetaminophen for 10 days at a total dose of 720 mg/day (72 mg/kg per day). The patient had high levels of aspartate aminotransferase (11 735 U/L) and alanine aminotransferase (6611 U/L) accompanied by encephalopathy and an increased ammonium level (266 μg/dL). Intravenous N-acetylcysteine therapy resulted in rapid improvement of the child's clinical condition and laboratory test results. Health care providers should be aware that multiple doses of acetaminophen in infants may lead to acute hepatic failure. N-acetylcysteine therapy should be initiated in cases of drug-induced acute liver failure.
Current Opinion in Pediatrics · 2010 · 9 citations
Neonatal liver failure: a genetic and metabolic perspective
AbstractLiver failure in newborns can present formidable diagnostic challenges. The presentation of neonatal liver failure is variable and the initial assessment is crucial in the determination of potentially treatable causes. We present a case of neonatal hemochromatosis, review genetic and metabolic causes of neonatal liver failure, and outline an updated differential diagnosis of neonatal liver failure. In addition, we propose a comprehensive initial work-up of neonatal liver failure, and review current treatments for neonatal hemochromatosis.
AbstractAcute liver failure is a rare event in the newborn period yet early recognition in the neonatal intensive care setting is essential for best outcome. Neonatal acute liver failure (NALF) is distinct from acute liver failure in older children and adults having different etiologies, presentation, and unique treatment interventions. There is a paucity of literature regarding NALF and several newly identified conditions merit discussion. Herein we report three cases of liver failure who were admitted to our neonatal intensive care unit and review the diagnostic approach and management of liver failure in this age group.
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.
