DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for optic atrophy — screening already-approved drugs against its 30-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleOptic atrophy maps to a 30-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 atrophy 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
ornithine aminotransferase (OAT) — OAT 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 3r,4edrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 10LW · 1.93 Å · ligand (3R,4E)-4-[({3-hydroxy-2-methyl-5-[(phosphonooxy)methyl]pyridin-4-yl}methyl)imino]cyclopent-1-ene-1,3-dicarboxylic acid (RMT). Experimental structure, not a prediction.
What the evidence adds up to
In a 1946 abstract, malaria therapy was reported to control syphilitic primary optic atrophy, with the explanation that the optic nerve degeneration was due to an inflammatory process that malaria therapy arrested. The same abstract stated that routine treatment with trivalent arsphenamine, bismuth and mercury was entirely inefficacious in syphilitic optic atrophy, and that patients receiving it would become blind almost as rapidly as if they had received no treatment at all. No clinical data or details of treatment were given in that abstract. A 1947 report noted that the outlook for vision in syphilitic primary optic atrophy was generally considered extremely poor regardless of therapy, but cited gratifying reports of unusually successful results with malaria treatment by Moore and others. The 1947 report attributed pessimism to excessive delay in starting treatment, which could allow optic atrophy to progress to blindness before adequate treatment was instituted.
A 2010 report described a family with 20 members in three successive generations affected by optic atrophy without other ocular or extraocular manifestations, transmitted as an autosomal-dominant trait. There was a clearly bimodal distribution of severity: 4 male patients complained of severe impairment of vision since childhood, while 16 other subjects (7 males and 9 females) were completely asymptomatic. The authors suggested this could be a new variety of autosomal dominant optic atrophy characterised by sex-influenced severity. A 1985 review of three affected members of a family with autosomal dominant optic atrophy (a father and two daughters) and comparison with 17 additional cases indicated that those with early or congenital onset were at the more severe end of a distribution curve of involvement, while less severely affected patients were older and often had passed many years with little or no visual difficulties. The 1985 authors suggested there was probably only one genetic locus for autosomal dominant optic atrophy.
A 2014 case report described a 13-year-old child presenting with gross visual impairment who was diagnosed with optic atrophy, but radiological investigations revealed osteopetrosis, which the authors noted, though rare, can result in optic atrophy. The aim of that report was to highlight this possibility when evaluating cases of optic atrophy in young patients.
What is still missing is any controlled trial of any drug for non-syphilitic optic atrophy, any modern replication of the malaria therapy results, any genetic stratification that could predict which patients might respond to any treatment, and any funding for such work.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
New England Journal of Medicine · 1947 · 7 citations
Syphilitic Primary Optic Atrophy
AbstractA FEW years ago the outlook regarding vision for patients with syphilitic primary optic atrophy was generally considered extremely poor, regardless of the type of therapy used. This attitude tends to persist in spite of the gratifying reports by Moore et al.1 and others2 , 3 of unusually successful results with malaria treatment. One of the reasons for pessimism is that there is too often an excessive delay in starting treatment and that, as a result, optic atrophy may progress to blindness before adequate treatment is instituted. This delay often appears to be due to failure to make the proper diagnosis, lack . . .
Familial Optic Atrophy with Sex-Influenced Severity
AbstractA family is described with 20 members in three successive generations affected by optic atrophy without other ocular or extraocular manifestations. The anomaly was transmitted as an autosomal-dominant character. There was a clearly bimodal distribution of severity: 4 male patients complained of severe impairment of vision since childhood while 16 other subjects (7 males and 9 females) were completely asymptomatic. This family could be an example of a new variety of autosomal dominant optic atrophy characterized by sex-influenced severity.
Indian Journal of Ophthalmology · 2014 · 6 citations · open access
An unusual cause of optic atrophy in a child
AbstractA 13-year-old child presenting with gross visual impairment was diagnosed as a case of optic atrophy. However, radiological investigations revealed osteopetrosis, which, though rare, can result in optic atrophy. The aim of this case report is to highlight this possibility while evaluating cases of optic atrophy in young patients.
Ophthalmic Paediatrics and Genetics · 1985 · 4 citations
Variable severity in autosomal dominant optic atrophy
AbstractThere are some indications in the literature on autosomal dominant optic atrophy that there are two genetic types - a congenital and a post-natal. This paper reviews the ocular findings of three affected members of a family with autosomal dominant optic atrophy - a father and two daughters - which appear to fit the criteria for a 'congenital' type of optic atrophy. Comparison with an additional 17 cases indicates that those with an early or congenital onset are at the more severe end of a distribution curve of involvement. The less severely affected patients were older and often had passed many years with little or no visual difficulties. Such variation is a recognized feature of autosomal dominant inheritance and is the basis for suggesting that there is probably only one genetic locus for autosomal dominant optic atrophy.
British Journal of Ophthalmology · 1946 · 0 citations · open access
ABSTRACTS
Abstract1) Good results in the control of syphilitic optic atrophy by malaria therapy are reported.These are explained on the basis that the degeneration of the optic nerves is due to an inflammatory process which is arrested by malaria therapy.Routine treatment with trivalent arsphenamine, bismuth and mercury is entirely inefficaceous in syphilitic optic atrophy, and the patient will become blind almost as rapidly as if he had received no treatment at all.No clinical data or details of treatment are given.
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.
