DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for inclusion body myositis — screening already-approved drugs against its 9-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 myositis maps to a 9-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 myositis 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
FKBP prolyl isomerase 1A (FKBP1A) — FKBP1A 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 4~{s},5~{r},6~{z},9~{s},10~{s},12~{e}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6I1S · 1.52 Å · ligand (4~{S},5~{R},6~{Z},9~{S},10~{S},12~{E})-16-(ethylamino)-4,5-dimethyl-9,10,18-tris(oxidanyl)-3-oxabicyclo[12.4.0]octadeca-1(14),6,12,15,17-pentaene-2,8-dione (E26). Experimental structure, not a prediction.
What the evidence adds up to
No established treatment improves, arrests, or slows progression of inclusion body myositis. Many anti-inflammatory, immunosuppressant, or immunomodulating agents have been tried, but trial design only allows the conclusion that none produced rapid improvement. High-dose prednisone worsens strength while decreasing inflammation but increasing amyloid accumulation. The natural history is that a third of patients stabilise or improve for six months or more, so some agents that did not produce dramatic benefit may have been prematurely abandoned. Immunosuppressive treatment is in most instances ineffective, and the disease is resistant to corticosteroids and other immunotherapies.
A 1989 review of ten histopathologically defined cases reported that two patients had excellent responses to treatment, but the authors noted that inclusion body myositis is generally refractory to treatment and that from a clinical perspective it must be considered a nonspecific designation. A 2011 case report described a male patient with a ten-year history who responded well to intravenous immunoglobulin therapy, with a slowly progressive and rather stable course. A 2022 case report described a 70-year-old female patient who received hyperbaric oxygen therapy and platelet-rich plasma injections as adjunctive therapy and had improvement in muscle function and ambulation.
Recent multiomic analyses of IBM tissues have identified new pathogenic pathways and biomarkers that need validation in larger cohorts. Ongoing clinical trials are targeting T cells and autophagy. New diagnostic criteria and outcome measures have been proposed to improve clinical trial design. Exercise remains the primary therapeutic modality available. What is still missing are validated biomarkers, larger cohort studies to confirm new pathogenic pathways, and effective treatment targets that go beyond the current reliance on exercise and adjunctive case reports.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Neurology · 2006 · 57 citations
The current status of treatment for inclusion-body myositis
AbstractThere is no established treatment that improves, arrests, or slows the progression of inclusion-body myositis (IBM). Many anti-inflammatory, immunosuppressant, or immunomodulating agents have been administered to patients with IBM but the design of clinical trials was such that it can only be concluded that none produced rapid improvement. The natural history of the disease is for stabilization or improvement in a third of patients for 6 months or more. Thus some agents that did not produce dramatic benefit may have been prematurely abandoned. However, because high-dose prednisone worsens strength while decreasing inflammation but increases amyloid accumulation, alternative targets for intervention and novel treatment strategies are needed.
Clinical heterogeneity and treatment response in inclusion body myositis
AbstractInclusion body myositis has been described as an inflammatory myopathy with distinctive clinical and pathologic features that is refractory to treatment. Ten cases of inclusion body myositis, as defined by histopathologic findings, were reviewed to determine whether the clinical characteristics are different in patients whose disease has been defined by light and electron microscopic studies compared with those whose disease has been defined by light microscopic studies alone. The clinical characteristics of both groups of patients were similar, and 2 patients have had excellent responses to treatment. Although inclusion body myositis represents a histologic subset of polymyositis, from a clinical perspective, it must be considered a nonspecific designation. Despite a generally poor prognosis, therapeutic intervention is still warranted.
Current Opinion in Rheumatology · 2001 · 22 citations
Inclusion body myositis: genetic factors, aberrant protein expression, and autoimmunity
AbstractSporadic inclusion body myositis (s-IBM) is an inflammatory myopathy mainly affecting elderly individuals. It has a chronic progressive course leading to severe disability. Immunosuppressive treatment is in most instances ineffective. S-IBM is morphologically characterized by mononuclear cell infiltrates and vacuolated muscle fibers with pathologic accumulation of a large number of different proteins. Recent research has focused on the expression of various factors that may contribute to the inflammatory reaction and the typical inclusions. This review summarizes the new information on genetic factors, abnormal protein expression and inflammation, which provides a basis for linking the different typical morphologic features of s-IBM to a cascade of pathogenic events.
Journal of Neurology Neurosurgery & Psychiatry · 2000 · 14 citations · open access
A case of inclusion body myositis with benign monoclonal gammopathy successfully responding to repeated immunoabsorption
AbstractA 69 year old woman with inclusion body myositis is described. She presented with benign monoclonal gammopathy. She was resistant to steroid therapy, but responded to repeated immunoabsorption. Up to now, there has been no established therapy for inclusion body myositis, including IVIg. It is suggested that immunoabsorption could be an alternative therapy for inclusion body myositis, when it was accompanied by immunological abnormality.
Current Opinion in Rheumatology · 2024 · 12 citations
Inclusion body myositis: an update
AbstractPURPOSE OF REVIEW: To review recent advances in our understanding of the epidemiology, pathophysiology, and management of inclusion body myositis (IBM). RECENT FINDINGS: Recent epidemiologic studies have highlighted the morbidity and mortality associated with IBM, including the impact of dysphagia. Multiomic analyses of IBM tissues have identified new pathogenic pathways and biomarkers for use in clinical trials. New diagnostic criteria and outcome measures have been proposed to improve clinical trial design. Ongoing clinical trials are targeting T cells and autophagy. SUMMARY: Improvements in our understanding of IBM pathogenesis are identifying new pathways and biomarkers that need validation in larger cohorts. Exercise remains the primary therapeutic modality available, and new treatment targets are needed.
Neurologia i Neurochirurgia Polska · 2011 · 5 citations
Inclusion body myositis: therapeutic approaches. A case report
AbstractInclusion body myositis (IBM) seems to be the most common acquired myopathy among patients of age 50 or over. The characteristic clinical features of IBM include involvement of the quadriceps as well as distal muscles, mainly foot extensors and deep finger flexors. The course of the disease is slow but steadily progressive and most patients after 5 to 10 years require the aid of an assistive device. What distinguishes IBM from other inflammatory myopathies is its resistance to corticosteroids and other immunotherapies. We present a case report, the first in the Polish population, of a male patient with a 10-year history of inclusion body myositis responding well to intravenous immunoglobulin (IVIG) therapy. Despite the almost 10 years duration of the disease, its course was slowly progressive and rather stable. Essential aspects of diagnosis and pathogenesis as well as the therapeutic approach adopted are also discussed. Wtrętowe zapalenie mięśni (inclusion body myositis – IBM) to najczęstsza nabyta miopatia zapalna po 50. roku życia. W obrazie klinicznym najbardziej charakterystyczne jest zajęcie mięśni czworogłowych ud, jak również mięśni dystalnych, zginaczy grzbietowych stopy i zginaczy głębokich palców. Przebieg choroby jest powolny, ale systematycznie postępujący, po kilku latach trwania choroby większość pacjentów przestaje chodzić. W przeciwieństwie do pozostałych miopatii zapalnych, wtrętowe zapalenie mięśni nie odpowiada na leczenie kortykosteroidami i innymi lekami immunosupresyjnymi. Przedstawiana praca to pierwszy w Polsce opis przypadku chorego z dziesięcioletnim wywiadem wtrętowego zapalenia mięśni, u którego leczenie dożylnie podawanymi preparatami immunoglobulin (IVIG) przyniosło dobre efekty. Przebieg choroby w ciągu niemalże 10 lat był powoli postępujący i względnie stabilny. Ponadto omówiono zagadnienia związane z diagnostyką, patogenezą i obecnymi możliwościami leczenia wtrętowego zapalenia mięśni.
International Journal of Advances in Medicine · 2022 · 0 citations · open access
An emerging role of platelet-rich plasma and hyperbaric oxygen in the management of inclusion body myositis: a case report
AbstractInclusion body myositis is an uncommon inflammatory myopathy that causes progressive muscle weakness. Patient management includes immunosuppressant therapy and nonpharmacologic therapies, like physical, occupational, and speech therapy. Standard treatment plans focus on the maintenance of muscle strength and function. Many patients do not respond to pharmacologic therapies and due to the progressive nature of this myopathy, patients eventually become debilitated. Hyperbaric oxygen therapy and platelet-rich plasma injections were provided as adjunctive therapy to a 70-year-old female patient with inclusion body myositis. After treatment, she had improvement in her muscle function and improved ambulation. This case study highlights the impact of adjunctive therapy in a patient with inclusion body myositis.
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.
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