DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for myositis — screening already-approved drugs against its 20-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMyositis maps to a 20-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 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
alpha-L-iduronidase (IDUA) — IDUA 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 1~{r},2~{r},3~{r},4~{s},6~{s}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6I6R · 2.02 Å · ligand (1~{R},2~{R},3~{R},4~{S},6~{S})-6-azanyl-2,3,4-tris(oxidanyl)cyclohexane-1-carboxylic acid (H62). Experimental structure, not a prediction.
What the evidence adds up to
In a 2002 cohort of 46 patients with idiopathic inflammatory myositis (excluding malignancy-associated disease and inclusion body myositis), followed for up to 20 years, 15.2% achieved full remission, 17.4% had a monophasic illness, 19.6% had a relapsing-remitting course, 34.8% had chronic progressive illness, and 13.04% died. The five-year survival rate was 95% and the 10-year survival rate 83.8%. All patients scored significantly lower on the SF-36 health survey across all domains compared to the general population. Patients with chronic progressive illness reported more bodily pain than those with relapsing-remitting illness, but no significant difference in SF-36 scores was found between patients with active disease and those with inactive disease.
A 2023 observational study of 175 idiopathic inflammatory myositis patients (mean age 40.9 years, 76.6% female) reported that among 94 patients followed for a median of 65 months, 74.1% had a complete clinical response and 11.8% had a partial clinical response at last follow-up. 40.2% were off steroids and 13.8% were in clinical remission. Complete clinical response was associated with better functional outcomes and less damage compared to partial response. The overall mortality rate was 13.7%, with disease-specific mortality at 9.1%; most deaths occurred early and were associated with active disease. Baseline parameters and myositis subsets did not significantly influence functional outcome or damage.
A 2022 historical cohort of 80 inflammatory myositis patients found that 40.0% achieved complete remission, 3.8% had incomplete remission, and 13.8% had no response to treatment; 23.8% did not return for follow-up at least six months after starting treatment. No deaths were reported during follow-up. An association was found between treatment response and baseline factors including intravenous immune globulin use, time of symptom onset, sex, disease pattern, and subtype. A 2001 prospective study of 11 untreated inclusion body myositis patients over six months found a mean decline of 4% in predicted normal muscle strength, but one third of patients showed no change or slight improvement.
What is still missing is a clear understanding of why a substantial minority of patients do not respond to current treatment, and why early active disease drives mortality. No randomised controlled trials are reported here, and the observational data do not control for treatment regimens or patient stratification by autoantibody status or muscle biopsy subtype. Larger, prospectively designed studies with standardised outcome measures and longer follow-up are needed to identify which patients are at risk of poor outcomes.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Lara D. Veeken · 2002 · 273 citations
Outcome in patients with idiopathic inflammatory myositis: morbidity and mortality
AbstractOBJECTIVE: To assess the long-term outcome of a cohort of 46 patients with idiopathic myositis by assessing both health status, as measured by the SF-36, and cumulative survival probability over a 20-yr follow-up period at a single rheumatology centre. Methods and results. Forty-six patients under long-term follow-up from 1978 to 1999 were identified from our database. All patients fulfilled three out of four of the Bohan and Peter criteria for myositis. We excluded those with malignancy-associated disease and those with inclusion body myositis. Twenty-three patients (50%) had adult-onset polymyositis, 14 (30.4%) had adult-onset dermatomyositis, one had childhood-onset dermatomyositis and eight (17.4%) had an overlap syndrome (associated with either systemic lupus erythematosus or rheumatoid arthritis). During the course of the disease, seven patients (15.2%) went into full remission, eight (17.4%) had monophasic illness, nine (19.6%) had a relapsing-remitting course, 16 (34.8%) had chronic progressive illness and six (13.04%) died. All patients had significantly lower SF-36 scores in all aspects of health compared with the general population (P< or =0.001). Patients with chronic progressive illness had significantly greater bodily pain (P< or =0.05, t-test) than those with a relapsing-remitting illness, but did not differ in other aspects of health. There was no significant difference in the scores in the different domains of the SF-36 between the patients with active disease and those with inactive disease (0.05<P<0.1). Six of the 46 patients died. Cumulative survival probability was calculated. The five-year survival rate was 95% and the 10-yr survival rate 83.8%. CONCLUSION: Patients with myositis report significantly poorer health compared with the general population. Health status and disease activity are important outcome measures in the assessment of patients with myositis.
A prospective natural history study of inclusion body myositis
AbstractEleven patients with untreated inclusion body myositis (IBM) were prospectively studied during a 6-month period that included muscle strength, lean body mass, and muscle mass measurements. There was an overall quantifiable mean decline in percent of predicted normal muscle strength of 4% from baseline in a 6-month period, but one third of patients showed no change or slight improvements in strength. Short-term treatment trials in IBM will require large numbers of patients to detect slowing, arrest, or even slight improvement in muscle strength.
Mediterranean Journal of Rheumatology · 2023 · 5 citations · open access
Long Term Outcomes in Idiopathic Inflammatory Myositis: An Observational Epidemiologic Study over 15 Years
AbstractBackground: We report a longitudinal observational cohort of idiopathic inflammatory myositis (IIM) focusing on the long-term clinical outcome and associated parameters. Methods: IIM patients were classified as per Bohan and Peter criteria. In those with ≥ 24 months of follow-up; the treatment response, functional outcomes, and damage at last follow-up were recorded. Complete clinical response and clinical remission as defined by Oddis et al., was used to define outcomes at last follow-up. Results: The cohort consists of 175 patients, mean age 40.9 (+12.6) years, M:F 1:3.3; and the major subsets were dermatomyositis (44.6%), overlap myositis (25.7%), antisynthetase syndrome (6.3%), polymyositis (14.3%), and juvenile DM/OM (8.6%). Ninety-four patients have followed up for 24 months or more, with the median (IQR) of 65(35,100.7) months. Of them, 74.1% and 11.8% had complete and partial clinical responses respectively at the last follow-up. In our cohort 40.2% were off-steroids and 13.8% were in clinical remission at the last follow-up. Complete clinical response was associated with better functional outcomes and lesser damage as determined by HAQ-DI of 0[OR10.9; 95%CI (3.3,160)], MRS [OR 3.2; 95%CI (1.4,7.3)] and lesser MDI [OR 1.7; 95% CI (1.1,2.7)] respectively as compared to partial response (unadjusted analysis). Baseline parameters and IIM subsets did not significantly influence the functional outcome and damage. The mortality rate in our cohort is 24/175 (13.7%), the disease-specific mortality rate being 9.1%. Large majority of deaths were early, associated with active disease. Conclusion: We report good long-term outcomes in all major myositis subsets. Partial clinical response to treatment is associated with worse functional outcomes and damage accrual. Death occurs early in association with active disease.
Expert Opinion on Investigational Drugs · 2021 · 4 citations
Inflammatory myopathies: shedding light on promising agents and combination therapies in clinical trials
AbstractINTRODUCTION: Due to new insights into the pathogenesis of inflammatory myopathies - in short myositis - and the urgent need for new treatment options in patients who are refractory to standard therapy, multiple novel drugs have been developed and studied in clinical trials. In light of this exciting development, a critical evaluation of the present data is necessary in order to identify the best pathway to future treatment of inflammatory myopathies. AREAS COVERED: This review focuses on the current evidence from clinical trials in myositis and encompasses dermatomyositis, polymyositis, necrotizing myopathy, antisynthetase-syndrome, overlap myositis, and inclusion body myositis. The results of studies on new therapeutic agents are summarized, in particular larger cohort studies and randomized trials from recent years. When such data were not available, earlier and smaller representative studies were included instead. EXPERT OPINION: Current studies in most myositis subtypes have shown positive effects of novel biologicals such as abatacept, sifalimumab, JAK-Inhibitors as well as known agents such as rituximab, but further studies are needed to confirm these observations. In inclusion body myositis, the eagerly awaited recent therapeutic trials have missed their primary endpoints, except for the phase 2 study with rapamycin, which has demonstrated significant improvements in secondary endpoints. Future trials will also need to focus on combination therapies of multiple immunomodulatory agents.
Health Science Monitor · 2022 · 0 citations · open access
Epidemiology and clinical outcome and its main determinants among patients with inflammatory myositis
AbstractBackground & Aims: Despite the proper understanding of pathophysiological aspects and recent development in therapeutic approaches, the outcome of patients suffering from inflammatory myositis remains unsatisfactory. In addition, there is no clinical information and a clear outlook for this disease in the community. This study aimed to evaluate the outcome of patients with inflammatory myositis (response rate to treatment) and to determine the related and predictive factors of this outcome in these patients. Materials & Methods: This historical cohort study was performed on 80 patients suffering from inflammatory myositis. By retrospectively reviewing the patient records in the hospital, basic information was extracted and through telephone calls, the outcome status of the disease, and response to treatment were assessed during follow-up and categorized as complete remission, partial remission, and no remission. Results: Within the follow-up time, 40.0% were completely treated (complete remission), 3.8% had no proper response to treatment (incomplete remission), and 13.8% did not respond to the treatment. Also, 23.8% did not refer for further treatment at least six months from the start of treatment. No death was reported within the follow-up time. We found an association between the quality of treatment response and baseline parameters, including the rate of receiving intravenous immune globulin regimen, time of symptoms onset, gender, different patterns of disease, and disease subtype. Conclusion: A notable number of inflammatory myositis patients still do not respond to routine treatment, and we, in fact, are at the forefront of managing the disease.
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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