DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for demyelinating polyneuropathy — 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 moduleDemyelinating polyneuropathy 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 demyelinating polyneuropathy 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
kinesin family member 5A (KIF5A) — KIF5A 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 gtpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4UXY · 6.5 Å · ligand GUANOSINE-5'-TRIPHOSPHATE (GTP). Experimental structure, not a prediction.
What the evidence adds up to
Four patients with refractory chronic inflammatory demyelinating polyneuropathy (CIDP) were treated with high-dose cyclophosphamide (200 mg/kg over four days) without stem-cell rescue. All four improved in functional status and muscle strength, and nerve conduction studies improved in three of them. Other immunomodulatory medications were discontinued. The authors report that responses lasted over three years and resulted in long-term remission in this small, uncontrolled series.
A retrospective review of patients treated between 1992 and 2003 at one US centre analysed high-dose intermittent intravenous methylprednisolone as initial and maintenance therapy for CIDP. The abstract gives no numerical results for this analysis.
Two review articles from 2014 and 2019 discuss the biological basis of CIDP treatments, the heterogeneity of the disease—including pure motor forms and variable sensory dysfunction, as well as monophasic, progressive, and relapsing courses—and the outcome measures used in randomised controlled trials over the previous 30 years. Neither review reports new trial data.
What is still missing is any randomised controlled trial of high-dose cyclophosphamide in CIDP, which would require funding and a multi-centre design to recruit a meaningful sample. The 2019 review notes that the existing evidence base relies on trials using different primary and secondary outcomes, and that the relation of those outcomes to routine clinical evaluation remains unclear. No trial has stratified patients by the molecular subtypes or clinical phenotypes described in the 2014 review.
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 · 2002 · 120 citations
High-dose cyclophosphamide without stem-cell rescue for refractory CIDP
AbstractFour patients with chronic inflammatory demyelinating polyneuropathy (CIDP) who were refractory to conventional treatment were treated with high-dose cyclophosphamide (200 mg/kg over 4 days). All improved in functional status and muscle strength. Nerve conduction studies improved in three of four. Other immunomodulatory medications have been discontinued. High-dose cyclophosphamide can be given safely to patients with CIDP and patients with disease persistence after standard therapy may have a response that lasts for over 3 years and results in long-term disease remission.
Autoimmune Diseases · 2014 · 29 citations · open access
Treatment of Chronic Inflammatory Demyelinating Polyneuropathy: From Molecular Bases to Practical Considerations
AbstractChronic inflammatory demyelinating polyneuropathy (CIDP) is an autoimmune disease of the peripheral nervous system, in which both cellular and humoral immune responses are involved. The disease is clinically heterogeneous with some patients displaying pure motor form and others also showing a variable degree of sensory dysfunction; disease evolution may also differ from patient to patient, since monophasic, progressive, and relapsing forms are reported. Underlying such clinical variability there is probably a broad spectrum of molecular dysfunctions that are and will be the target of therapeutic strategies. In this review we first explore the biological bases of current treatments and subsequently we focus on the practical management that must also take into account pharmacoeconomic issues.
Outcome Measures for Chronic Inflammatory Demyelinating Polyneuropathy in Research: Relevance and Applicability to Clinical Practice
AbstractOutcome measures are recommended in the management of chronic inflammatory demyelinating polyneuropathy (CIDP). Various scales have been proposed in recent years, some now commonly utilized in daily clinical practice. The available evidence base relies itself on randomized controlled trial data obtained over the past 30 years, with several studies using different primary and secondary outcomes. We here review the different outcome measures used in CIDP research in relation to those currently recommended for clinical management. We consider the evidence base for CIDP treatment from the primary and secondary outcomes used in these studies and attempt to assess how this may relate to current clinical practice of routine evaluation of treatment effects and long-term monitoring.
S S Korsakov Journal of Neurology and Psychiatry · 2024 · 1 citations
Neuropathy in n-hexane poisoning
Abstract-hexane either through contact with unprotected skin or inhalation can lead to the development of clinical symptoms and electrophysiological changes similar to those of inflammatory demyelinating polyneuropathy which requires careful differential diagnosis. This article presents three cases of severe predominantly motor polyneuropathy with demyelinating features in 15- and 16-year-old adolescents. The results of laboratory tests were within normal limits; electroneuromyography revealed symmetrical involvement of sensory and motor fibers of the nerves of the legs and arms with a decrease in the speed of propagation of excitation and conduction blocks. Sural nerve biopsy revealed intraneural and perineural swelling without any signs of inflammation or fibrosis confirming the genesis of the neuropathy. Despite a relatively favorable prognosis there is no specific therapy for hexane poisoning and the recovery period can last up to several years.
Steroids for Chronic Inflammatory Demyelinating Polyneuropathy
AbstractThe efficacy and safety of high-dose, intermittent IV methylprednisolone (IVMP) as initial and long-term maintenance therapy for chronic inflammatory demyelinating polyneuropathy (CIDP) were analyzed by a retrospective review of outcome data derived from patients’ medical records between 1992 and 2003 at Washington University School of Medicine, St Louis, MO.
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.
