DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Lisch epithelial corneal dystrophy — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleLisch epithelial corneal dystrophy maps to a 4-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 lisch epithelial corneal dystrophy 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
diphosphoinositol pentakisphosphate kinase 2 (PPIP5K2) — PPIP5K2 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 atpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3T54 · 1.9 Å · ligand ADENOSINE-5'-TRIPHOSPHATE (ATP). Experimental structure, not a prediction.
What the evidence adds up to
Lisch epithelial corneal dystrophy is a rare corneal condition. A 2022 case report described a 65-year-old woman with a recurrent left-sided corneal lesion consistent with LECD. Slit-lamp examination showed an opalescent, whorl-shaped corneal lesion; high-resolution optical coherence tomography revealed a trapezoidal area of normal thickness epithelial hyperreflectivity. Histopathology demonstrated a mucosal epithelium with foamy cytoplasm and increased cell size. Treatment with two 1-week cycles of topical 5-fluorouracil resulted in marked clearance of the lesion on slit-lamp examination and HR-OCT. The authors concluded that 5-FU may be considered as a treatment option for LECD. This is a single case report, not a controlled trial.
A 2024 case report described a 46-year-old woman with suspected LECD who underwent an unintentionally staged alcohol keratectomy and intentionally targeted minor limbal excision with cautery. After alcohol keratectomy there was complete resolution but visually significant recurrence at 2 years. The abnormal epithelial recurrence originated in a linear tapered pattern from a focal superior limbal region. After minor limbal excision with cautery and alcohol keratectomy, the patient's vision returned to baseline with a clear cornea and no recurrence in over 5 years of follow-up. Histopathological analysis demonstrated cytoplasmic vacuolation and glycogen granules; the limbal tissue showed normal histological features. The authors concluded this two-staged approach provides definitive treatment and highlights the focal limbal origin of abnormal cells. Again, this is a single case.
A 2007 study of 18 homozygous patients with granular corneal dystrophy type II (Avellino corneal dystrophy), a different TGFBI-associated condition, found that all patients who underwent surgeries (phototherapeutic keratectomy, penetrating keratoplasty, lamellar keratoplasty, or deep lamellar keratoplasty) acquired better vision immediately after surgery, but corneal deposits reappeared soon after treatments and recurrences became progressively more rapid and severe with treatments. A 2014 case report of a 49-year-old man with granular corneal dystrophy noted that after penetrating keratoplasty in 1989 and phototherapeutic keratectomy with mitomycin C for graft recurrence in 2002, his vision and comfort were stable with minimal recurrence of granular opacities in the left surgical eye at last examination. A 2018 review of corneal dystrophies stated that no effective etiopathogenetically targeted treatment is currently known, and that conservative treatment is symptomatic (lubricants, epithelizing agents, soft contact lenses).
What is still missing for LECD are prospective studies with more than one patient, randomised controlled trials of topical 5-fluorouracil, and replication of the staged keratectomy and limbal excision approach in a larger series. The natural history of LECD and the proportion of patients who experience recurrence after any treatment remain poorly quantified. No drug has been tested in a controlled fashion, and no trial funding is evident.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Cornea · 2007 · 41 citations · open access
Homozygous Granular Corneal Dystrophy Type II (Avellino Corneal Dystrophy)
AbstractPURPOSE: To describe the clinical features of homozygous granular corneal dystrophy type II (GCDII) with age and with several kinds of treatment in 18 homozygous patients in several different conditions. METHODS: Eighteen homozygous GCDII patients, confirmed with DNA analysis, of 13 families were enrolled. Their clinical features that include age at detection by parents, visual acuity, and disease progression were evaluated. We also studied the recurrence patterns for the 13 patients who underwent phototherapeutic keratectomy, penetrating keratoplasty, lamellar keratoplasty, or deep lamellar keratoplasty. RESULTS: The age at detection by the parents ranged from 3 to 5 years; visual loss begins in childhood with progression into the 20s. All of the patients who had undergone surgeries acquired better vision immediately after surgery. Corneal deposits reappeared soon after treatments. Recurrences became progressively more rapid and severe with treatments. CONCLUSIONS: The clinical features of homozygous GCDII are characterized by a severe granular type of corneal dystrophy with an early onset and rapid progression. After surgical treatment, recurrence is rapid and severe.
Russian Annals of Ophthalmology · 2018 · 7 citations
Corneal dystrophies
AbstractThe article reviews modern clinical, morphological, and genetic aspects of corneal dystrophies based on the most recent international classification updated in 2015. Corneal dystrophies is a group of slow progressing, non-inflammatory corneal pathologies, most of which are characterized by variability of the associated traits. The existence of such pathologies makes important their differential diagnosis from acute inflammatory processes of various etiology, which require urgent therapy. Conservative treatment suitable for dystrophies is usually associated with disorders of the anterior corneal surface and employ symptomatic tactic (lubricants, epithelizing agents, soft contact lenses). No effective etiopathogenetically targeted treatment is currently known. In cases with significant visual acuity decrease, it is possible to perform phototherapeutic keratectomy, abrasive polishing of Bowman's membrane with diamond bur and various types of keratoplasty depending on the depth of involvement.
AbstractPURPOSE: The purpose of this case report is to review granular corneal dystrophy (GCD) and examine the new paradigm in its classification and treatment. CASE REPORT: A 49-year-old white male patient reported yearly for monitoring of GCD. He had an ocular surgical history in the left eye for penetrating keratoplasty in 1989 and phototherapeutic keratectomy with mitomycin C for graft recurrence of stromal bread-crumb opacities 17+ years later in 2002. At his last examination, the patient's vision and comfort was stable in each eye, with minimal recurrence of granular opacities in the left surgical eye, stable granular opacities in the right eye, no recurrent corneal erosion symptoms in either eye, and best spectacle-corrected vision of 20/40 OD and 20/30 OS. CONCLUSIONS: GCD is a Category 1, Stromal, TGFBI-associated corneal dystrophy. Although it is classified as a stromal dystrophy, research suggests the possibility that the granular opacities have an origination to the corneal epithelium with a migratory effect to the corneal stroma. Patients with Groenouw I, like the one in this report, usually do not have severely compromised vision. When vision is significantly affected or recurrent corneal erosion occurs, despite first- and second-line treatments, viable management options thereafter include photokeratectomy and other new surgical treatments such as femtosecond deep anterior lamellar keratoplasty and femtosecond laser-assisted keratoplasty. Future advancements in diagnostic technology, immunohistologic and genetic testing, medications, and surgery will allow for advancements in treating and managing patients with GCD.
Recurrent Lisch Epithelial Corneal Dystrophy Treated With 5-Fluorouracil: A Case Report and Review of the Literature
AbstractPURPOSE: The aim of the study was to describe a case of Lisch epithelial corneal dystrophy (LECD), review its clinical and histopathological features and diagnostic imaging, and introduce a novel treatment approach using topical 5-fluorouracil (5-FU). METHODS: A 65-year-old woman presented with a recurrent left-sided corneal lesion consistent with LECD. The lesion was evaluated clinically, with high-resolution optical coherence tomography (HR-OCT), and histologically. The lesion was successfully treated with two 1-week cycles of topical 5-FU. RESULTS: Slit-lamp examination showed an opalescent, whorl-shaped corneal lesion. HR-OCT revealed a trapezoidal area of normal thickness epithelial hyperreflectivity. Histopathology demonstrated a mucosal epithelium with foamy cytoplasm and increased cell size consistent with LECD. Treatment with topical 5-FU resulted in marked clearance of the corneal lesion on slit-lamp examination and HR-OCT. CONCLUSIONS: 5-FU may be considered as a treatment option for LECD.
Definitive Treatment of Lisch Epithelial Corneal Dystrophy via Staged Keratectomy and Targeted Minor Limbal Excision With Cautery
AbstractPURPOSE: We demonstrate a novel approach for the definitive treatment of Lisch epithelial corneal dystrophy via an unintentionally staged alcohol keratectomy and intentionally targeted minor limbal excision with cautery. METHODS: A 46-year-old woman presented with visually significant corneal changes, suspected to be Lisch epithelial corneal dystrophy after clinical examination, anterior segment optical coherence tomography, and confocal microscopy. Alcohol keratectomy was performed with complete resolution, but there was visually significant recurrence at 2 years. As the abnormal epithelial recurrence originated in a linear tapered pattern from a focal superior limbal region, minor limbal excision with cautery and alcohol keratectomy was performed. RESULTS: Histopathological analysis demonstrated cytoplasmic vacuolation and glycogen granules. The limbal tissue demonstrated normal histological features. The patient's vision returned to baseline on reepithelialization with a clear cornea, with no recurrence in over 5-year follow-up. CONCLUSIONS: Our targeted two-staged approach provides definitive treatment for Lisch epithelial corneal dystrophy and highlights the focal limbal origin of abnormal cells.
DOAJ (DOAJ: Directory of Open Access Journals) · 2020 · 0 citations · open access
Research progress of descemetorhexis without endothelial keratoplasty promoting corneal endothelial regeneration
AbstractEndothelial dysfunctionis traditionally considered irreversible, and endothelial keratoplasty(EK)is almost the only treatment available. Recently, however, a surgery called descemetorhexis without endothelial keratoplasty(DWEK)can regenerate the central corneal endothelial cells in patients with Fuchs endothelial corneal dystrophy(FECD), and local Rho-associated kinase inhibitor can enhance its efficacy.
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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