DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for pathological myopia — screening already-approved drugs against its 34-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePathological myopia maps to a 34-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 pathological myopia 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
cell division cycle 7 (CDC7) — CDC7 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 2sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4F9C · 2.08 Å · ligand 8-chloro-2-[(2S)-pyrrolidin-2-yl][1]benzofuro[3,2-d]pyrimidin-4(3H)-one (0SX). Experimental structure, not a prediction.
What the evidence adds up to
In a 2009 series of 16 eyes given three monthly intravitreal ranibizumab injections for myopic choroidal neovascularisation, mean logMAR visual acuity improved from 0.58 (20/76) at baseline to 0.28 (20/37) at 12 months, a mean gain of 3.0 lines. Twelve eyes (75.0%) gained 2 or more lines. Angiographic closure was seen in 15 eyes (93.8%) at 3 months; one eye required further treatment for persistent leakage, and two eyes (12.5%) had recurrence between 3 and 9 months. No ocular or systemic side effects were reported. A 2010 multicentre case series of 40 eyes treated on demand with ranibizumab 0.5 mg reported a mean baseline logMAR of 0.68 (20/131) and a final mean of 0.27 (20/42) after a median follow-up of 13.3 months. Visual acuity improved in 82.5% of patients, and 60% gained 3 or more lines. The mean number of injections was 2.8. Fifteen eyes (37.5%) had previously received photodynamic therapy, but age and prior PDT did not influence the results. No side effects were observed. A 2012 study of ten aphakic eyes treated with three trans-corneal injections of ranibizumab reported mean decimal visual acuity improving from 0.19 to 0.33 (logMAR 0.48) at 6 months, a mean gain of 2.86 lines. Mean central macular thickness fell from 340 μm to 212 μm. No injection complications or drug-related side effects were noted.
A 2014 UK cost-effectiveness analysis used a Markov model based on the phase III RADIANCE trial and the VIP study. Over a lifetime horizon, ranibizumab treatment cost £12,866 and produced 12.99 quality-adjusted life-years, compared with £14,421 and 12.60 QALYs for verteporfin photodynamic therapy, and £8,163 and 12.45 QALYs for observation. Ranibizumab was therefore dominant over vPDT (greater health gain at lower cost) and cost-effective versus observation, with an incremental cost-effectiveness ratio of £8,778 per QALY. At a willingness-to-pay threshold of £20,000 per QALY, ranibizumab had a 100% probability of being cost-effective compared with vPDT and an 88% probability compared with observation.
Two review papers from 2014 and 2015 on the pathogenesis of progressive myopia discuss growth factors, protein metabolism, and hormonal and neural regulation, and suggest that future systemic or local medications might target connective tissue disorders, hormonal shifts, and autonomic imbalance. These reviews do not present clinical trial data or test any specific drug. The evidence for ranibizumab in myopic CNV is limited to small, non-randomised series with short follow-up (the largest has 40 eyes, the longest follow-up is 18 months). No randomised controlled trial comparing ranibizumab to placebo or to other anti-VEGF agents in this specific population is included. What is still missing is a large, masked, randomised trial with adequate power to confirm the visual gains seen in these small series, and to establish the optimal dosing regimen and long-term safety in pathological myopia.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Retina · 2009 · 93 citations
INTRAVITREAL RANIBIZUMAB FOR THE PRIMARY TREATMENT OF CHOROIDAL NEOVASCULARIZATION SECONDARY TO PATHOLOGIC MYOPIA
AbstractPURPOSE: To evaluate the efficacy of intravitreal ranibizumab for the primary treatment of myopic choroidal neovascularization (CNV). METHODS: Sixteen eyes of 16 consecutive patients who received 3 monthly injections of intravitreal ranibizumab for primary treatment of myopic CNV were reviewed. Additional ranibizumab injections were performed in eyes with persistent or recurrent CNV after 3 months. RESULTS: The mean age of the patients was 60.8 years, and the spherical equivalent refractive error was -10.9 D. The mean logMAR best-corrected visual acuity at baseline was 0.58 (20/76). At 1 month and 12 months, the mean logMAR best-corrected visual acuity improved significantly to 0.39 (20/49) and 0.28 (20/37), respectively (P = 0.001 and P < 0.001, respectively). The mean improvement at 12 months was 3.0 lines, and 12 (75.0%) eyes had improvement of 2 or more lines. Fifteen (93.8%) eyes had angiographic closure at 3 months and 1 (6.2%) required further treatment because of persistent leakage at 3 months. Two (12.5%) patients had recurrence of CNV and required retreatment between 3 months and 9 months. Optical coherence tomography showed significant reduction in the mean central foveal thickness after treatment (P < 0.001). None of the patients developed any ocular or systemic side effects associated with intravitreal ranibizumab. CONCLUSION: Intravitreal ranibizumab appeared to be effective for the primary treatment of myopic CNV, with a high proportion of patients sustaining visual gain after treatment.
British Journal of Ophthalmology · 2010 · 46 citations · open access
Is ranibizumab effective in stopping the loss of vision for choroidal neovascularisation in pathologic myopia? A long-term follow-up study
AbstractAIM: To assess the efficacy and safety of ranibizumab in the treatment of choroidal neovascularisation (CNV) caused by pathologic myopia (PM). DESIGN: Prospective, multicentre, interventional case series. METHODS: 40 eyes of 39 consecutive patients with PM and CNV were treated with 'on demand' intravitreal injection of ranibizumab 0.5 mg. Final best corrected visual acuity (BCVA) and its change from baseline were the main outcome measures. Changes in optical coherence tomography (OCT) central retinal thickness (CRT) were a secondary outcome. RESULTS: Mean age was 53±13 years and mean refractive error -13.5±6.5 D. Median follow-up was 13.3±2 (range 12-18) months. Fifteen eyes (37.5%) had previously been treated with photodynamic therapy (PDT). The mean baseline logarithm of the minimum angle of resolution (logMAR) BCVA (Early Treatment Diabetic Retinopathy Study (ETDRS) vision chart) was 0.68±0.34 (Snellen equivalent 20/131) and 21±16 letters. The final mean logMAR BCVA was 0.27±0.2 (p = 0.008) (20/42) and 40.5±14 letters (p = 0.01). Mean final VA improved in 82.5% of patients, in 60% by 3 or more lines (median number of lines gained 2.9). Even six out of seven cases of low vision (≤1.1 logMAR) at the final examination has improved vision. Mean OCT CRT reduced from 218±70 to 175±46 μm (p 0.02). Age and previous PDT did not influence the results (p>0.05). The mean number of injection was 2.8±1.2 (range 1-6). No ocular or systemic side effects were observed. CONCLUSION: Ranibizumab was an effective treatment for stabilising and improving vision with a low number of injections in 92.5% of patients with myopic CNV in a long-term follow-up.
Ranibizumab, Verteporfin Photodynamic Therapy or Observation for the Treatment of Myopic Choroidal Neovascularization: Cost Effectiveness in the UK
AbstractPURPOSE: The aim of this study was to evaluate the cost effectiveness of ranibizumab compared with verteporfin photodynamic therapy (vPDT) or no treatment (observation) in patients with visual impairment due to myopic choroidal neovascularization (CNV). METHODS: A Markov model with health states defined by best-corrected visual acuity and a 3-month cycle length was developed. It had a healthcare provider (UK National Health Service and personal social services) perspective, a lifetime time horizon, and was based on 2011 prices; future costs and health outcomes were discounted at 3.5 % per annum. Baseline characteristics were based on the phase III RADIANCE (Ranibizumab and vPDT Evaluation in Myopic CNV) study, and year 1 health-state transitions were based on this and the VIP (Verteporfin in Photodynamic Therapy) study. Extensive sensitivity analyses tested the robustness of the model. RESULTS: The lifetime cost of treating myopic CNV with ranibizumab was £12,866, whereas vPDT and observation were associated with total costs of £14,421 and £8,163, respectively. Ranibizumab treatment produced higher cumulative quality-adjusted life-years (QALYs; 12.99) than vPDT (12.60) or observation (12.45). Ranibizumab treatment was therefore dominant, with greater health gains and lower overall costs than vPDT. Ranibizumab was cost effective compared with observation, with an incremental cost-effectiveness ratio of £8,778/QALY. In the probabilistic sensitivity analysis, ranibizumab had a 100 % and 88 % probability of being cost effective compared with vPDT and observation, respectively, at a willingness-to-pay threshold of £20,000/QALY. CONCLUSION: This study indicates that ranibizumab therapy is dominant over vPDT for the treatment of visual impairment due to CNV secondary to pathologic myopia in the UK healthcare setting and cost effective compared with observation.
Annals of the Russian academy of medical sciences · 2014 · 8 citations
Modern Trends of Basic Research in Pathogenesis of Progressive Myopia
AbstractThe growing prevalence of progressive myopia and its disabling consequences explains the elaboration of reliable diagnostic markers and new treatment strategies based on the research results of molecular mechanisms underlying the development of the condition. The paper reviews recent basic pathogenetic research studies which have greatly broadened the awareness of the deep causes of progressive myopia associated with the activity of certain growth factors, local and systemic protein metabolism, and regulation of hormonal and neural processes. Practical clinical guidelines for new criteria of diagnosis and control of myopia are published as they could be useful while selecting individual treatment plans including indications to sclera-strengthening therapy and its evaluation. The results may be promising in the elaboration of systemic and local medications for the prevention of myopia progression, which should address the regulation of connective tissue disorders, hormonal shifts, and imbalanced autonomic nervous system.
Annals of the Russian academy of medical sciences · 2015 · 5 citations · open access
MODERN TRENDS OF BASIC RESEARCH IN PATHOGENESIS OF PROGRESSIVE MYOPIA
AbstractThe growing prevalence of progressive myopia and its disabling consequences explains the elaboration of reliable diagnostic markers and new treatment strategies based on the research results of molecular mechanisms underlying the development of the condition. The paper reviews recent basic pathogenetic research studies which have greatly broadened the awareness of the deep causes of progressive myopia associated with the activity of certain growth factors, local and systemic protein metabolism, and regulation of hormonal and neural processes. Practical clinical guidelines for new criteria of diagnosis and control of myopia are published as they could be useful while selecting individual treatment plans including indications to sclera-strengthening therapy and its evaluation. The results may be promising in the elaboration of systemic and local medications for the prevention of myopia progression, which should address the regulation of connective tissue disorders, hormonal shifts, and imbalanced autonomic nervous system.
Use Siena air (University of Siena) · 2012 · 0 citations
Ranibizumab (lucentis) injection by anterior chamber in aphakic eyes with myopic choroidal neovascularization
Abstractpurpose. macular choroidal neovascularization (CNV) is one of the most vision-threatening complications of myopia, which can lead to severe vision loss. Our purpose was to evaluate the safety and efficacy of trans-corneal injection of ranibizumab in the treatment of myopic CNV in aphakic patients.\nMaterials and Methods. ten eyes of 10 aphakic patients with CNV secondary to pathologic myopia treated with three trans- corneal injection of ranibizumab were evaluated. A complete ophthalmologic examination including best-corrected visual acuity (BCVA) and fundus biomicroscopy, specular microscopy, fundus optical coherence tomography (OCT), fluorescein an- giography (FA) were performed at baseline and monthly for all patients. Mean time of follow-up was 6 months.\nresults. The mean axial length was 27,6 mm (range, 25.7-31.3 mm). The mean initial visual acuity (VA) was 0.19 (decimal equivalent). A statistically significant improvement to a mean VA of 0.33 decimal equivalent (log-MAR:0.48) was demon- strated at the final follow-up. VA improved by a mean of 2.86 lines. Mean central macular thickness (CMT) measured with OCT was 340 μm (range, 179-663 μm) at the baseline, and was reduced significantly at the final follow-up to 212μm (range, 125-455 μm). No injection complications or drug-related side effects were noted during the follow-up period.\nConclusions. in this small series of aphakic eyes with limited follow-up, ranibizumab by anterior chamber administration seems to be a safe and effective treatment for CNV secondary to pathologic myopia (PM), without any complications. Further studies to evaluate the safety and efficacy are justified.\nKeywords. Ranibizumab, safety, efficacy, myopic CNVs, aphakia, anterior chamber.
Disease module: DeepOracle (Open Targets). Approved indication: ChEMBL drug_indication (max_phase=4). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works, 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.
