DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for optic nerve disorder — screening already-approved drugs against its 6-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleOptic nerve disorder maps to a 6-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 optic nerve disorder 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
DENN domain containing 1A (DENND1A) — DENND1A 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 gdpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6EKK · 1.82 Å · ligand GUANOSINE-5'-DIPHOSPHATE (GDP). Experimental structure, not a prediction.
What the evidence adds up to
In experimental models of optic neuropathies, major clinical trials such as the Optic Neuritis Treatment Trial and Ischemic Optic Neuropathy Decompression Trial have shown that currently available treatments for demyelinating and ischaemic optic neuropathies are ineffective and can even be harmful. A 2010 review states that ischaemic damage of the optic nerve has no proven effective treatment. For the common idiopathic form (nonarteritic anterior ischaemic optic neuropathy, NAION), multiple attempted therapies including systemic corticosteroids, anticoagulants, antiplatelet agents, diphenylhydantoin, hyperbaric oxygen, and optic nerve sheath decompression have been unsuccessful. Levodopa has been proposed but is unproven, and megadose intravenous corticosteroid therapy has not been studied systematically. Prophylaxis in NAION is unproven.
For hereditary optic neuropathies (HONs), a 2024 review notes that treatment targeting various pathogenic mechanisms has been investigated, but studies of clinical applicability remain nascent. Present management largely remains supportive. The two most prevalent HONs are Leber Hereditary Optic Neuropathy and Dominant Optic Atrophy. Despite major advances linking Leber Hereditary Optic Neuropathy to mitochondrial DNA mutations, a 2000 review observed relatively little focus on applying basic scientific methodologies to optic neuropathies other than glaucoma. The relative absence of detailed scientific knowledge about basic mechanisms in optic nerve injury has contributed to the use of empiric therapies.
Neural regeneration and repair in the central nervous system are active research topics, and a 2007 article considered the potential to restore function to the damaged optic nerve, outlining technical issues and a strategy for research progress. Optic nerve regeneration studies are ongoing in animals. Neuroprotective strategies are under intense investigation for optic neuropathies including NAION, with clinical trials in humans in progress as of 2010. A 2022 review catalogued rare genetic syndromes causing secondary optic atrophy but did not report any effective treatments.
What is still missing are completed, positive human clinical trials for any pharmacological treatment of these optic neuropathies. The clinical applicability of treatments targeting pathogenic mechanisms in hereditary optic neuropathies remains nascent. No neuroprotective or regenerative strategy has yet been proven in patients. Adequate funding for trials, better patient stratification by genetic or ischaemic subtype, and validated animal models that translate to human disease are all lacking.
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 · 1979 · 110 citations
Peripheral neuropathy in arsenic smelter workers
AbstractWe conducted a double-blind controlled study of individuals exposed to arsenic trioxide in a copper-smelting factory. Subjects fell into three categories of peripheral neuropathy: none, subclinical, and clinical. The subclinical group had no symptoms or signs of numbness or reduced reflexes, but did have reduced nerve conduction velocity and amplitude measurements. Clinical neuropathy groups had signs and symptoms of neuropathy and electrophysiologic abnormalities. The clinical and subclinical groups correlated with increased content of arsenic in urine, hair and nails. The incidence of subclinical and clinical neuropathy was greater in arsenic workers than in unexposed controls.
Clinical ophthalmology · 2012 · 34 citations · open access
Current options for the treatment of optic neuritis
AbstractOptic neuritis can be defined as typical (associated with multiple sclerosis, improving independent of steroid treatment), or atypical (not associated with multiple sclerosis, steroid-dependent improvement). Causes of atypical optic neuritis include connective tissue diseases (eg, lupus), vasculitis, sarcoidosis, or neuromyelitis optica. In this manuscript, updated treatment options for both typical and atypical optic neuritis are reviewed. Conventional treatments, such as corticosteroids, therapeutic plasma exchange, and intravenous immunoglobulin therapy are all discussed with commentary regarding evidence-based outcomes. Less commonly used treatments and novel purported therapies for optic neuritis are also reviewed. Special scenarios in the treatment of optic neuritis - pediatric optic neuritis, acute demyelinating encephalomyelitis, and optic neuritis occurring during pregnancy - are specifically examined.
Current Opinion in Ophthalmology · 2000 · 17 citations
New therapies for optic neuropathies: development in experimental models
AbstractExperimental models of human diseases have affected the design and direction of both basic and clinical research into understanding the pathogenesis and treatments of demyelinating disease, stroke, and hereditary disorders of the central nervous system. However, in spite of major advances in molecular research that have linked Leber Hereditary Optic Neuropathy to mutations in mitochondrial DNA, there has been relatively little focus in applying basic scientific methodologies to optic neuropathies other than glaucoma. The relative absence of detailed scientific knowledge about the basic mechanisms involved in the pathogenesis of optic nerve injury has contributed to the use of empiric therapies for neuro-ophthalmic optic neuropathies. Over the past decade major clinical trials, such as the Optic Neuritis Treatment Trial and Ischemic Optic Neuropathy Decompression Trial, have proven that currently available treatment options for demyelinating and ischemic optic neuropathies are ineffective and can even be harmful. Although the pathogenesis of visual failure in demyelinating, ischemic, and hereditary optic neuropathies appears diverse, a final common pathway for irreparable optic nerve injury may exist. This article reviews several models of experimental optic neuropathies that may aid in the development of novel treatments for neuro-ophthalmic disorders of the optic nerve during the 21st century.
AbstractNeural regeneration and repair in the central nervous system are currently hot topics in neuroscience. For many years there has been a hope that neurodegenerative diseases which are resistant to current therapies may be treated by the selective replacement of cells. Yet it is only recently that we have started to acquire the knowledge, tools, and techniques that may translate such optimism into new therapies. In this article, we will consider the potential to restore function to the damaged optic nerve. We will consider the technical issues involved and suggest a strategy for research progress.
Journal of Clinical & Translational Ophthalmology · 2024 · 2 citations · open access
Hereditary Optic Neuropathies: An Updated Review
AbstractHereditary optic neuropathies (HONs) are a class of genetic disorders that may lead to vision loss due to either acute or progressive injury to the optic nerve. Although HONs may commonly manifest as isolated optic atrophy, these disorders can also have a variety of characteristic clinical features and time courses that may narrow the differential diagnosis. While the two most prevalent HONs are Leber Hereditary Optic Neuropathy (LHON) and Dominant Optic Atrophy (DOA), the phenotypic spectrum of these conditions, as well as genetic landscape of less common optic neuropathies, have been better characterized through advances in molecular diagnostic testing. Treatment targeting various pathogenic mechanisms has been investigated, although studies of clinical applicability remain nascent. Present management largely remains supportive. In this review, we discuss the clinical features, molecular diagnosis, current treatment, and future directions for HONs.
AbstractIschemic damage of the optic nerve has no proven effective treatment. While ischemia related to vasculitis (arteritic) is treated with systemic corticosteroids, the primary goal is to prevent further damage, either in the affected or fellow eye. Thrombolytic or anticoagulation supplementive therapy may be considerations for the future. In the more common idiopathic (nonarteritic) form (NAION), multiple attempts at therapy, including systemic corticosteroids, anticoagulants and antiplatelet agents, diphenylhydantoin, hyperbaric oxygen, and optic nerve sheath decompression have been unsuccessful. The use of levodopa has been proposed but is unproven. Megadose intravenous corticosteroid therapy has not been studied in a systematic way. Neuroprotective strategies are under intense investigation for optic neuropathies including NAION, and clinical trials in humans are in progress. Optic nerve regeneration studies are ongoing in animals. Prophylaxis in NAION is unproven.
Güncel Retina Dergisi (Current Retina Journal) · 2022 · 0 citations · open access
Other Hereditary Syndromes Associated with Secondary Optic Atrophy
AbstractOptic neuropathy is a cause that seriously impairs vision loss, which we frequently encounter in ophthalmology practice. Although it is frequently seen with eye diseases such as glaucoma, it can also be seen together with systemic neurodegenerative syndromes. In this review, we have included some rare syndromes that cause genetically inherited optic atrophy. In these syndromes, the structure of the encoded proteins is disrupted as a result of mutated genes. The cellular disorder is reflected in the phenotype by affecting the related intracellular chemical reaction.
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.
