DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for ptosis — screening already-approved drugs against its 29-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePtosis maps to a 29-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 ptosis 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
tryptophanyl tRNA synthetase 2, mitochondrial (WARS2) — WARS2 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 5sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5EKD · 1.82 Å · ligand (5S)-5-[(1R)-1-(1H-indol-3-yl)ethyl]-2-(methylamino)-1,3-oxazol-4(5H)-one (5BX). Experimental structure, not a prediction.
What the evidence adds up to
The 2002 case review of 340 paediatric ptosis patients under 16 in a single tertiary centre found that 79% had myogenic ptosis. Surgery was performed for visual indications in 43% and cosmetic indications in 57%. A good outcome was achieved in 77% (260/340), a suboptimal outcome in 10% (35/340), and a poor outcome requiring re-operation in 13% (45/340). Recorded patient satisfaction was 90% (206/229). There was no statistically significant difference in outcome between patients with mild, moderate, or severe ptosis or with good, moderate, or poor levator function. The 2008 review states that treatment options for ptosis are strictly surgical, that no ideal technique exists for every patient, and that a surgeon must be familiar with an algorithm for choosing the appropriate method.
A 2020 pooled analysis of two phase 3 randomised, double-masked, placebo-controlled trials examined oxymetazoline hydrochloride 0.1% ophthalmic solution in 304 participants aged 9 years and older with acquired ptosis and superior visual field deficit. Participants were randomised 2:1 to oxymetazoline or vehicle, self-administered once daily for 42 days. On day 1 (6 hours after instillation), the mean increase in points seen on the Leicester Peripheral Field Test was 5.9 (SD 6.4) for oxymetazoline versus 1.8 (SD 4.1) for vehicle (mean difference 4.07, 95% CI 2.74 to 5.39, p<0.001). On day 14 (2 hours after instillation), the increase was 7.1 (SD 5.9) versus 2.4 (SD 5.5) (mean difference 4.74, 95% CI 3.43 to 6.04, p<0.001). Marginal reflex distance 1 also increased significantly: day 1 mean increase 0.96 mm (SD 0.89) versus 0.50 mm (SD 0.81) (mean difference 0.47 mm, 95% CI 0.27 to 0.67, p<0.001); day 14 mean increase 1.16 mm (SD 0.87) versus 0.50 mm (SD 0.80) (mean difference 0.67 mm, 95% CI 0.46 to 0.88, p<0.001). Treatment-emergent adverse events occurred in 31.0% of the oxymetazoline group and 35.6% of the vehicle group; 81% of those in the oxymetazoline group with an adverse event had a maximum intensity of mild, and 62% had no event suspected as treatment-related. The authors note that further study is needed to elucidate the clinical relevance of these findings beyond six weeks.
What is still missing is long-term safety and efficacy data beyond six weeks for oxymetazoline, and any direct comparison of this pharmacological approach against surgical outcomes. The paediatric data are from a single centre and are purely surgical; no medical treatment for childhood ptosis is described. No trial has yet stratified patients by ptosis aetiology or severity to determine which subgroups, if any, derive meaningful functional or cosmetic benefit from oxymetazoline beyond the short-term visual field improvement reported.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
British Journal of Ophthalmology · 2002 · 79 citations · open access
Aetiology and surgical treatment of childhood blepharoptosis
AbstractAIMS: To describe the aetiology, demography, surgical management, and outcome of a cohort of paediatric ptosis patients in a large tertiary referral oculoplastic centre. METHODS: A case note review of all patients undergoing ptosis surgery below the age of 16 years in a tertiary referral oculoplastic unit documenting the laterality, aetiology, severity of ptosis, indications for and type of surgery undertaken, the proportion of good, suboptimal, and poor surgical outcomes, re-operations, and level of patient satisfaction. RESULTS: 340 patients (82% (280/340) unilateral, 18% (60/340) bilateral ptosis) with myogenic (79%, 269/340), aponeurotic (5%, 16/340), neurogenic (11%, 37/340), mechanical (2%, 6/340), apparent (1%, 2/340), and syndrome related (3%, 10/340) ptosis underwent anterior (41%, 141/340) and posterior (26%, 90/340) levator resection, frontalis suspension with mersilene (9%, 29/340) and autogenous fascia lata (17%, 59/340), levator transposition (5%, 15/340) and other surgery (1%, 6/340) for visual (43%, 141/333) and cosmetic (57%, 189/333) indications. 77% (260/340) of patients achieved a good outcome, 10% (35/340) a suboptimal outcome, and 13% (45/340) a poor outcome requiring re-operation. There was no statistically significant difference in surgical outcome between patients with mild, moderate, or severe ptosis and with good, moderate, or poor levator function. The level of recorded patient satisfaction with the surgical outcome was 90% (206/229). CONCLUSIONS: Results suggest that most groups of paediatric ptosis patients, including those with poor levator function and severe ptosis, achieve satisfactory results with the appropriate ptosis surgery.
Current Opinion in Ophthalmology · 2008 · 43 citations
Ptosis repair options and algorithm
AbstractPURPOSE OF REVIEW: The treatment options for ptosis are strictly surgical. Each patient needs to be individually assessed and treated. A surgeon needs to be familiar with all types of surgical repair to choose the best possible option for his/her particular patient. RECENT FINDINGS: There are certain established surgeries in ptosis repair; however, there is no ideal surgical technique to treat every patient without any drawbacks. Several modifications of known techniques have been described to address the weaknesses of original applications. Also, new techniques such as orbicularis plication, levator readaptation, and new approaches in frontalis suspension surgery were presented. An algorithm showing the appropriate/most preferred ptosis surgery techniques is prepared based on the traditional knowledge and the latest publications. SUMMARY: There have been many modifications and new proposals in the management of ptosis. The surgeon needs to be familiar with an algorithm for the appropriate surgical method.
JAMA Ophthalmology · 2020 · 36 citations · open access
Association of Oxymetazoline Hydrochloride, 0.1%, Solution Administration With Visual Field in Acquired Ptosis
AbstractImportance: Treatment of acquired blepharoptosis (ptosis) is currently limited to surgical intervention. Objective: To examine the efficacy and safety of oxymetazoline hydrochloride, 0.1%, ophthalmic solution (oxymetazoline, 0.1%) in participants with acquired ptosis. Design, Setting, and Participants: This pooled analysis of 2 randomized, double-masked, placebo-controlled, multicenter phase 3 clinical trials included participants 9 years and older with acquired ptosis and superior visual field deficit. The 2 studies were conducted across 16 and 27 sites in the United States. Patients were enrolled from May 2015 to April 2019. Analyses for the individual trials were initiated after database lock and completed on September 6, 2017, and May 16, 2019. Pooled analysis was completed on August 25, 2019. Interventions: Participants (randomized 2:1) received oxymetazoline, 0.1%, or vehicle, self-administered as a single drop per eye, once daily, for 42 days. Main Outcomes and Measures: The primary efficacy end point was change from baseline in the number of points seen on the Leicester Peripheral Field Test, a test to detect superior visual field deficits due to ptosis, on days 1 (6 hours after instillation) and 14 (2 hours after instillation). The secondary end point, change from baseline in marginal reflex distance 1, was assessed at the same time points. Results: In total, 304 participants were enrolled (mean [SD] age, 63.8 [13.8] years; 222 women [73%]). Overall, 97.5% (198 of 203) of participants receiving oxymetazoline, 0.1%, and 97.0% (98 of 101) of participants receiving vehicle completed the studies. Oxymetazoline, 0.1%, was associated with a significant increase in the mean (SD) number of points seen on the Leicester Peripheral Field Test vs vehicle (day 1: 5.9 [6.4] vs 1.8 [4.1]; mean difference, 4.07 [95% CI, 2.74-5.39]; P < .001; day 14: 7.1 [5.9] vs 2.4 [5.5]; mean difference, 4.74 [95% CI, 3.43-6.04]; P < .001). Oxymetazoline, 0.1%, also was associated with a significant increase in marginal reflex distance 1 from baseline (mean [SD]: day 1: 0.96 [0.89] mm vs 0.50 [0.81] mm; mean difference, 0.47 mm [95% CI, 0.27-0.67]; P < .001; day 14: 1.16 [0.87] mm vs 0.50 [0.80] mm; mean difference, 0.67 mm [95% CI, 0.46-0.88]; P < .001). Treatment-emergent adverse events (TEAEs) occurred in 31.0% (63 of 203) of participants receiving oxymetazoline, 0.1%, and 35.6% (36 of 101) of participants receiving vehicle. Among participants receiving oxymetazoline, 0.1%, with a TEAE, 81% (51 of 63) had a maximum TEAE intensity of mild, and 62% (39 of 63) had no TEAE suspected of being treatment related. Conclusions and Relevance: Oxymetazoline, 0.1%, was associated with positive outcomes and was well tolerated in phase 3 trials after instillation at days 1 and 14, demonstrating its potential promise for the treatment of acquired ptosis, although further study is needed to elucidate the clinical relevance of these findings beyond 6 weeks.
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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