DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Kennedy disease — screening already-approved drugs against its 11-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleKennedy disease maps to a 11-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 kennedy disease 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
androgen receptor (AR) — AR 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 1r,2rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5CJ6 · 2.07 Å · ligand 2-chloro-4-{[(1R,2R)-2-hydroxy-2-methylcyclopentyl]amino}-3-methylbenzonitrile (51Y). Experimental structure, not a prediction.
What the evidence adds up to
A 58-year-old Chinese man with limb numbness, progressive proximal weakness, lymph node and thyroid enlargement, oedema, lower-limb pigmentation and gynaecomastia was initially diagnosed with POEMS syndrome and treated with dexamethasone and small-dose cyclophosphamide for six months without improvement. Gene analysis later confirmed Kennedy disease. The authors note this as the first reported case of Kennedy disease misdiagnosed as POEMS syndrome and advise clinicians to consider the differential diagnosis.
In a Chinese family from Wenzhou, the proband showed proximal limb weakness, fasciculation, muscle atrophy, gynaecomastia, sexual dysfunction and raised serum creatine kinase. Electromyography identified both myopathy and neuropathy. Two other affected males and two female carriers carried expanded CAG repeats in the androgen receptor gene. The repeat numbers were 43 in the proband, and 43 and 42 in the other two affected males. One of these had symptoms similar to the proband.
Two further Chinese pedigrees were studied. Family A had 58 individuals across four generations; the proband had onset at age 39. Family B had 61 individuals across five generations, with two patients whose onset was at 39 and 41 years. All three patients had limb and bulbar muscular weakness from lower motor neuron damage, signs of androgen insensitivity, and mild to moderate elevation of serum creatine kinase. Electromyography showed widespread anterior horn damage; muscle biopsy showed neurogenic atrophy. CAG repeat numbers were 49, 48 and 47. Pedigree analysis confirmed X-linked recessive inheritance.
The abstracts provide no treatment trial data, no survival statistics, and no response rates. What is missing is any controlled study of a drug that slows progression or improves symptoms in Kennedy disease, as well as any systematic effort to stratify patients by CAG repeat length or disease stage for future trials.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Medical Principles and Practice · 2015 · 2 citations · open access
Kennedy Disease Misdiagnosed as Polyneuropathy, Organomegaly, Endocrinopathy, Monoclonal Gammopathy, and Skin Changes (POEMS) Syndrome: A Case Report
AbstractOBJECTIVE: The aim of this paper was to report the first case of Kennedy disease misdiagnosed as polyneuropathy, organomegaly, endocrinopathy, monoclonal gammopathy, and skin changes (POEMS) syndrome. CLINICAL PRESENTATION AND INTERVENTION: A 58-year-old Chinese man presented with limb numbness, progressive limb proximal weakness, lymph node and thyroid enlargement, edema, pigmentation in the lower limb, and obvious gynecomastia, which was initially diagnosed as POEMS syndrome and was treated with dexamethasone and small doses of cyclophosphamide without any improvement after 6 months. Finally, the patient diagnosis was confirmed as Kennedy disease (KD) by gene analysis. CONCLUSION: This case suggests that clinicians should pay more attention to the differential diagnosis between KD and POEMS syndrome. Gene analysis was helpful in detecting this rare confusing disease in this patient.
[Investigation of a family with Kennedy disease by genetic analysis].
AbstractOBJECTIVE: To report on a Chinese family from Wenzhou with genetically confirmed Kennedy disease and describe its clinical and genetic features. METHODS: The clinical phenotype and the level of relevant biochemical markers were assessed. To determine the number of CAG repeats in the exon 1 of androgen receptor (AR) gene, genomic DNA was extracted from peripheral blood samples of the family members, amplified by PCR and identified by DNA sequencing. RESULTS: The proband showed predominantly proximal limb weakness, fasciculation, muscle atrophy, gynecomastia, sexual dysfunction and increased serum creatine kinase. Myopathy and neuropathy were identified by electromyography. Two other affected males and 2 affected female carriers were identified to carry an expanded CAG repeat in the AR gene. The numbers of CAG repeats were found to be 43 in the proband, 43 and 42 in the other two affected males, one of which had similar clinical symptoms to the proband. CONCLUSION: The family was diagnosed with Kennedy disease by analysis of the AR gene.
[Study on clinical manifestation, genotype and genetic characteristics of two Kennedy disease pedigrees].
AbstractOBJECTIVE: To investigate the clinical manifestations, genotypes, and genetic characteristics of two pedigrees with Kennedy disease. METHODS: The clinical data of the patients from two Kennedy disease families were collected. The numbers of trinucleotide CAG repeats in exon 1 of the androgen receptor gene were determined by DNA sequencing and repeat fragment analysis. RESULTS: Family A was composed of 58 individuals in 4 generations. The proband had onset at 39 years old. There were two Kennedy disease patients in family B which included 61 individuals in 5 generations. The two patients had onset at 39 and 41 years old, respectively. All the three patients displayed limbs and bulbar muscular weakness because of the damage of lower motor neurons. They had androgen insensitivity syndrome in common, and showed mild or moderate increase in serum creatine kinase level. The electromyogram showed wild damage in anterior horn of spinal cord. Muscle biopsy displayed neurogenic muscular atrophy. The numbers of the CAG repeat expansion in the androgen receptor gene of the three patients were 49, 48, and 47, respectively. X-linked recessive mode of inheritance was demonstrated by pedigree analysis in the two families. CONCLUSION: Kennedy disease usually occurs in mid-adulthood man. The clinical features are the weakness and wasting of limbs and bulbar muscles. Genetic analysis contributes to diagnosis and identification of carriers, and is beneficial to genetic counseling and prenatal diagnosis.
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.
