DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hereditary fructose intolerance — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHereditary fructose intolerance maps to a 1-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 hereditary fructose intolerance 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
aldolase, fructose-bisphosphate B (ALDOB) — ALDOB 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8D44 · 2.8 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
Hereditary fructose intolerance is an uncommon metabolic disorder first described in 1956 by Chambers and Pratt. By 1963, 23 cases in 14 families had been reported from European clinics. The condition produces nausea, vomiting, malaise, substernal pain, excessive sweating, tremor, confusion, coma and convulsions after eating foods that contain fructose. Continued ingestion of fructose can lead to cirrhosis and mental retardation.
A 1988 review by T. M. Cox at Hammersmith Hospital covers the condition but the abstract provides no patient numbers, survival data, or treatment outcomes.
In 1990, five children with hereditary fructose intolerance developed neurological impairment. In three of these children the neurological problems were linked to the acute hepatic toxicity of fructose — hypoglycaemia, abnormal coagulation, and cardiovascular collapse. In the other two children no such link could be shown. The authors state that neurological impairment is not a classic feature of the disease but can occur during the acute phase and does not rule out the diagnosis.
No controlled trials, no survival statistics, and no response rates are reported in these abstracts. What is missing is any prospective study of long-term outcomes with strict dietary avoidance, any biomarker for neurological risk beyond acute hepatic crisis, and any trial design that stratifies patients by age or severity of initial presentation.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
New England Journal of Medicine · 1963 · 86 citations
Hereditary Fructose Intolerance
AbstractHEREDITARY fructose intolerance is an uncommon metabolic disorder, characterized by symptoms of nausea, vomiting, malaise, substernal pain, excessive sweating, tremor, confusion, coma and convulsions, that follows the ingestion of foods containing fructose.1 With continued ingestion of fructose cirrhosis2 3 4 and mental retardation4 , 5 may develop. To date, 23 cases in 14 families1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 have been reported from European clinics. The first description of hereditary fructose intolerance was presented by Chambers and Pratt6 in 1956. In the following year, Froesch et al.5 presented several cases in 1 family, fully described the symptoms of this condition and found that in the patients affected, low levels . . .
AbstractJournal Article Hereditary Fructose Intolerance Get access T. M. COX T. M. COX Departments of Medicine and Haematology, The Royal Postgraduate Medical School, Hammersmith HospitalDu Cane Road, London W12 OHS Search for other works by this author on: Oxford Academic PubMed Google Scholar QJM: An International Journal of Medicine, Volume 68, Issue 2, August 1988, Pages 585–594, https://doi.org/10.1093/oxfordjournals.qjmed.a068226 Published: 01 August 1988
Unusual Cerebral Manifestations in Hereditary Fructose Intolerance
AbstractFive children with hereditary fructose intolerance developed symptoms of neurological impairment. In three of them, neurological involvement was related to the acute hepatic toxicity of fructose (hypoglycemia, abnormal coagulation, cardiovascular collapse); in the other two, such a relationship could not be demonstrated. Neurological impairment is not classic in hereditary fructose intolerance, but its occurrence in the acute phase of the disease is possible and does not constitute an argument against the diagnosis.
Oxford University Press eBooks · 2016 · 1 citations
Disorders of Fructose Metabolism
AbstractHereditary fructose intolerance is an autosomal recessive disease which is manifest at weaning but formal diagnosis is often delayed until late childhood or adult life. Fructose, sucrose and sorbitol present in offending foods and drinks induce hypoglycaemia, hypophosphatemia, acidosis, hyperuricemia and hypermagnesemia. If unrecognized, the disease causes failure to thrive, a reno-tubular syndrome with nephrocalcinosis, jaundice, and ultimately liver injury. Parenteral administration of fructose or its congeners can be fatal. Molecular analysis of the aldolase B gene has revolutionized diagnosis. Treatment by a strict dietary exclusion (supplemented by water-soluble vitamins) is successful and, if instituted in a timely manner, is compatible with a normal life span. Early diagnosis and dietary modification are critical for well-being and normal development in affected children.
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.
