DeCure for Inclusion body myopathy with Paget disease of bone and frontotemporal dementia type 1
DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for inclusion body myopathy with Paget disease of bone and frontotemporal dementia type 1 — 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 moduleInclusion body myopathy with Paget disease of bone and frontotemporal dementia type 1 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 inclusion body myopathy with paget disease of bone and frontotemporal dementia type 1 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
valosin containing protein (VCP) — VCP 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 adpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 10QQ · 2.13 Å · ligand ADENOSINE-5'-DIPHOSPHATE (ADP). Experimental structure, not a prediction.
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 FASEB Journal · 2008 · 21 citations
Frontotemporal dementia associated with a <i>Valosin‐Containing Protein</i> mutation: report of three families
AbstractObjective: The phenotype of inclusion body myopathy with Paget disease of the bone and frontotemporal dementia (FTD) (IBMPFD), associated with Valosin‐Containing Protein ( VCP ) mutations is described in 3 families. Methods: Probands were identified on the basis of a pathological diagnosis of frontotemporal lobar degeneration with neuronal intranuclear ubiquitin/TDP‐43‐positive inclusions (FTLD‐U type IV). VCP sequencing was carried out. Clinical data on affected family members were reviewed. Results: Family A: 4 subjects had a myopathy (M): 2 showed also Parkinsonism (P) and FTD. Proband's brain had FTLD‐U type IV and brainstem‐type Lewy body pathology. Muscle displayed neurogenic changes and VCP(+)‐rimmed vacuoles. A VCP R191Q mutation was found in one allele. Family B: 8 subjects developed IBMPFD: FTD was seen in 4, M in 3, P in 2 and primary progressive aphasia (PPA) in one. Proband's brain showed FTLD‐U type IV, hippocampal sclerosis (HS) as well as Lewy neurites in the substantia nigra. A novel VCP T262A mutation was found in one allele. Family C: 2 subjects developed IBMPFD. The proband had FTD while a brother presented M and dementia. Proband's brain had FTLD‐U type IV and HS. A VCP R159C mutation was found in one allele. Conclusions: We identified three new families with IBMPFD associated with VCP mutations. A novel T262A mutation was found. One individual had PPA: a novel finding in IBMPFD. Supported by AG10133
Rinsho Shinkeigaku · 2013 · 2 citations · open access
Inclusion body myopathy with Paget’s disease of bone and frontotemporal dementia
AbstractInclusion body myopathy with Paget's disease of bone and frontotemporal dementia (IBMPFD) is an autosomal dominant disease caused by mutations in the VCP gene. VCP encodes a well-conserved multifunctional protein, valosin containing protein (VCP), which has important roles in protein quality control via proteasome and autophagy, protein aggregation, quality control of mitochondria, cell proliferation, and so on. Clinically, muscle weakness is the most common symptom of which disease onset is around 40 years. Affected muscles are variable, and the patients are sometimes diagnosed as limb girdle muscular dystrophy or GNE myopathy. Muscle pathology shows characteristic features including cytoplasmic/nuclear inclusions, rimmed vacuoles, and disorganized myofibrills, together with neurogenic changes. Paget's disease of bone is reported to be observed in a half of the patients around the age of 40 years, but less common in Japanese patients. Frontotemporal dementia is seen around one third of the patients which appears nearly 10 years later than muscle or bone disease. In addition to cognitive dysfunctions, motor neuron involvement and cerebellar signs were also seen in our series. IBMPFD is not so rare disease as previously thought, but complicate clinical findings may make its diagnosis difficult.
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.
