DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for familial visceral amyloidosis — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleFamilial visceral amyloidosis maps to a 4-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 familial visceral amyloidosis 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
lysozyme (LYZ) — LYZ 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 tvgdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5LSH · 1.061 Å · ligand propyl beta-D-galactofuranoside (TVG). Experimental structure, not a prediction.
What the evidence adds up to
Familial visceral amyloidosis is a systemic disease with a dismal prognosis for which the only existing surgical treatment is liver transplantation. A 49-year-old man with non-AA amyloidotic infiltration of the heart and intestinal tract was initially misdiagnosed, leading to maltreatment. In a 16-year-old girl, systemic amyloidosis presented as a discrete soft tissue amyloidoma in the abdomen, an uncommon tumour-like mass that can occur in the respiratory, genitourinary, gastrointestinal tracts, internal viscera, central nervous system, skin, breast, and soft tissues.
In one extended kindred, a phenotype was reported for the first time that contrasted with an apparently unrelated family carrying the same mutation who presented with spontaneous hepatic haemorrhage and rupture. The manifestations also differed from a family with the lysozyme Ile56Thr variant, who presented with dermal petechiae before proceeding to fatal visceral amyloidosis. A remarkably wide spectrum of disease can be caused by the same amyloid fibril protein, although renal involvement predominates in all cases except those dying of hepatic rupture.
Increased levels of alkaline phosphatase, triglycerides and cholesterol may distinguish the presence of liver compromise in amyloidosis. No drug treatment is described in any of these reports; the only intervention mentioned is liver transplantation, and that is surgical, not pharmacological. The abstracts contain no response rates, survival data, or sample sizes beyond individual case descriptions.
What is still missing is any randomised trial of a drug for this condition, any validated biomarker that reliably predicts progression before liver failure or rupture, and any stratification of patients by genotype or fibril protein type to guide therapy. Without these, the evidence base remains limited to case reports and expert opinion.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The American Journal of Gastroenterology · 2002 · 0 citations
Increased levels of alkaline phosphatase, triglycerides and cholesterol may distinguish the presence of liver compromise in amyloidosis
AbstractFamilial amyloidotic polyneuropathy is a systemic amyloidosis with a dismal prognosis for which a surgical treatment exists. Awareness of the clinical characteristics of the disease is critical for early genetic diagnosis and timely referral for liver transplantation. In this report we describe the history of a 49-year-old man in whom non-AA amyloidotic infiltration of the heart and the intestinal tract was diagnosed. Initially, inappropriate identification of the etiology of the disease led to maltreatment.
Pediatric Rheumatology · 2013 · 0 citations · open access
P01-036 – Systemic amyloidosis presenting with amyloidoma
AbstractAmyloidosis is a heterogeneous group of disorders characterized by extracellular deposition of unique protein fibrils. The least common presentation of an amyloid deposition is as a discrete mass called amyloidoma or amyloid tumor. It has been reported in many anatomic site including the respiratory, genitourinary, and gastrointestinal tracts, as well as internal viscera, the central nervous system, skin, breast, and soft tissues. We report a case of a soft tissue amyloidoma in the abdomen of an 16-year-old girl diagnosed with systemic amyloidosis.
Педиатрическая фармакология · 2010 · 0 citations · open access
Вирус папилломы человека онкогенный вирус
AbstractThe phenotype, reported for the first time in this extended kindred, contrasts with that of an apparently unrelated family carrying the same mutation who presented with spontaneous hepatic haemorrhage and rupture, and with the manifestations in a family with the lysozyme Ile56Thr variant who presented with dermal petechiae before proceeding to fatal visceral amyloidosis. A remarkably wide spectrum of disease can be caused by the same amyloid fibril protein, although renal involvement predominates in all cases except those dying of hepatic rupture.
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.
