DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for diffuse lymphatic malformation — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleDiffuse lymphatic malformation maps to a 1-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 diffuse lymphatic malformation 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
piezo type mechanosensitive ion channel component 1 (Er blood group) (PIEZO1) — PIEZO1 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 d12drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8ZU3 · 3.1 Å · ligand DODECANE (D12). Experimental structure, not a prediction.
What the evidence adds up to
A 2025 systematic review of medical therapies for paediatric soft-tissue lymphatic malformations examined 77 studies. Success rates, defined as lesion size reduction greater than 10%, were reported for oral alpelisib (9 of 9 patients), oral sirolimus (257 of 287), topical sirolimus (13 of 15), oral acetylsalicylic acid (18 of 23), oral propranolol (19 of 29), and oral sildenafil (33 of 71). Complete response, defined as size reduction over 90%, was seen with isotretinoin (1 of 1), intravenous cyclophosphamide (1 of 2), oral acetylsalicylic acid (4 of 23), topical sirolimus (2 of 15), and oral sirolimus (17 of 287). The review noted variable results, heterogeneity across studies, and a potential risk of bias, and concluded that additional long-term data are needed to validate efficacy and safety.
Earlier reviews describe a broader range of interventions. A 2004 review reported that the sclerosing agent OK-432 was effective for macrocystic lymphatic malformations but showed less promise for microcystic lesions, mixed lesions, and lesions outside the head and neck region. Somnoplasty showed promise for reduction of tongue lymphatic malformations, and surgical excision, staged when necessary, remained integral to management. A 2015 review listed emerging therapies including sildenafil, propranolol, and sirolimus, and emphasised that multimodal treatment continues to expand as new information about biology and genetics is discovered, with a patient-centred approach guiding timing and modality.
A 2012 case report described a female with progressive lymphatic malformation of the orbito-temporal region since childhood. Despite many surgical interventions including radical excision and shunt drainage, recurrence followed every intervention. The condition regressed after she began taking a contraceptive and virtually disappeared after pregnancy. A 2024 retrospective analysis of head and neck lymphatic malformations over 10 years presented data on age distribution, localisation, therapy, and recurrence rates, but the abstract does not give specific numbers for these outcomes.
What is still missing are prospective, controlled trials with standardised outcome measures, long-term safety and efficacy data for the drugs that show initial promise, and a clear understanding of which patient subgroups — defined by lesion type, location, or genetic markers — are most likely to benefit from each agent. The systematic review’s heterogeneity and risk of bias mean that the reported success rates cannot be taken as reliable estimates of treatment effect.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Current Opinion in Otolaryngology & Head & Neck Surgery · 2004 · 172 citations
Management of lymphatic malformations
AbstractPURPOSE OF REVIEW: Innovative otolaryngologists, plastic surgeons, craniofacial surgeons, pediatric surgeons, radiologists, anesthesiologists, neonatologists, obstetricians, and scientists have continued to advance our understanding of the etiology, diagnosis, and treatment of lymphatic malformations. This article reviews the publications over the past 2 years with respect to these advances. RECENT FINDINGS: Fast-sequence MRI limits motion artifacts and allows prenatal MR to be used as a complementary study to ultrasound in the evaluation of large congenital neck masses. Three-dimensional ultrasonography may also be helpful in evaluating prenatal lymphatic malformations. Fluorescence in situ hybridization techniques can be used to evaluate lymphatic malformations for prenatal chromosomal analysis with emphasis on chromosomes 13, 18, 21, X, and Y. The sclerosing agent OK-432 is effective for macrocystic lymphatic malformations but showed less promise for microcystic lesions, mixed lesions, and lesions outside the head and neck region. Somnoplasty shows promise for reduction of tongue lymphatic malformations. Surgical excision, staged when necessary, continues to be integral to management in many cases. SUMMARY: Basic science research has furthered understanding of lymphatic malformations. Clinical research has expanded and refined our diagnostic and therapeutic options for patients with these lesions. Further identification of genes selectively expressed by lymphatic endothelium should facilitate identification of usable vascular markers that can enable analysis of the underlying biology, physiology, pathology, and treatment of the lymphatic system and its malformations.
Current Opinion in Pediatrics · 2015 · 118 citations
Management of lymphatic malformations in children
AbstractPURPOSE OF REVIEW: To review the literature on lymphatic malformations and to provide current opinion about the management of these lesions. RECENT FINDINGS: Current treatment options include nonoperative management, surgery, sclerotherapy, radiofrequency ablation, and laser therapy. New therapies are emerging, including sildenafil, propranolol, sirolimus, and vascularized lymph node transfer. The primary focus of management centers on the patient's quality of life. SUMMARY: Multimodal treatment of lymphatic malformations continues to expand as new information about the biology and genetics of these lesions is discovered, in addition to knowledge gained from clinical practice. A patient-centered approach should guide timing and modality of treatment. Continued study of lymphatic malformations will increase and solidify a treatment algorithm for these complicated lesions.
Unusual regression of severe recurrent lymphatic malformation of a face after contraception and pregnancy
AbstractWe report the case of a female who had suffered from progressive lymphatic malformation in the orbito-temporal region since childhood. Many surgical interventions were performed, including radical excision and shunt drainage. Despite aggressive surgical treatment, recurrence was observed after every intervention. Eventually, the condition regressed after the patient began taking a contraceptive. Moreover, it virtually disappeared after pregnancy.
Journal of Cutaneous Medicine and Surgery · 2025 · 2 citations · open access
Medical Therapies for Pediatric Lymphatic Malformations: A Systematic Review
AbstractINTRODUCTION: Lymphatic malformations (LM) are vascular anomalies that can be challenging to manage with new medical therapies emerging. This systematic review examines current medical therapies for pediatric patients with LMs that involve the soft tissues. MATERIALS AND METHODS: MEDLINE, Embase, Cochrane Library, and SCOPUS were searched on April 12, 2024, using variations of the keywords "lymphatic malformation" AND "drug therapy" AND "pediatric." Language was limited to English, and no date restriction was applied. Treatment success was defined as a reduction in lesion size of over 10%, with complete response (CR) defined as a reduction in size of over 90%. RESULTS: Our review encompassed 4937 title/abstracts, 436 full-texts and ultimately included 77 studies. Reported success rates were variable, with notable results for alpelisib (oral) (n = 9/9), sirolimus (oral) (n = 257/287), sirolimus (topical) (n = 13/15), acetylsalicylic acid (oral) (n = 18/23), propranolol (oral) (n = 19/29), and sildenafil (oral) (n = 33/71). CR was reported with isotretinoin (n/a) (n = 1/1), cyclophosphamide (iv) (n = 1/2), acetylsalicylic acid (oral) (n = 4/23), sirolimus (topical) (n = 2/15), and sirolimus (oral) (n = 17/287). CONCLUSION: Overall, therapies such as alpelisib and sirolimus showed promising results in the reduction of pediatric LM size; however, additional long-term data are needed to validate their efficacy and safety profile. Limitations of our study include heterogeneity and a potential risk of bias.
Lymphatic malformations of the head and neck region – a retrospective analysis over 10 years
AbstractIntroduction Lymphatic malformations (LM) are rare vascular anomalies of the lymphatic vessels that can also occur in the head and neck region. This condition can lead to external cosmetic changes and functional impairments. Treatment options vary depending on the severity and symptoms. This study presents a retrospective analysis focusing on age distribution, localization, therapy, and recurrence rates.
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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