DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for lymphatic malformation 5 — 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 moduleLymphatic malformation 5 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 lymphatic malformation 5 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
forkhead box C2 (FOXC2) — FOXC2 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6AKP · 2.323 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
A 2004 review of lymphatic malformation management noted that the sclerosing agent OK-432 was effective for macrocystic lesions but showed less promise for microcystic, mixed, or lesions outside the head and neck. Surgical excision, staged when necessary, remained integral. A 2019 case report described a neonate with an 8.0 cm × 8.0 cm × 3.60 cm cervical lymphatic malformation treated with sclerotherapy using pingyangmycin combined with triamcinolone acetonide or lauromacrogol foam; the child was followed to 17 months with good growth and development, and the authors concluded that large neck lesions do not necessarily require surgical removal.
A 2015 review listed emerging therapies including sildenafil, propranolol, sirolimus, and vascularised lymph node transfer, and stated that multimodal treatment continues to expand as new biological and genetic information is discovered. A 2024 review noted that genetic causes of lymphatic malformations have been uncovered and several drug-based therapies are under investigation. A 2025 systematic review of medical therapies for paediatric soft-tissue lymphatic malformations examined 77 studies. Reported success rates (reduction in lesion size over 10%) were: alpelisib (oral) 9/9, sirolimus (oral) 257/287, sirolimus (topical) 13/15, acetylsalicylic acid (oral) 18/23, propranolol (oral) 19/29, and sildenafil (oral) 33/71. Complete response (over 90% reduction) was reported with isotretinoin (1/1), cyclophosphamide (intravenous, 1/2), acetylsalicylic acid (oral, 4/23), sirolimus (topical, 2/15), and sirolimus (oral, 17/287). The review concluded that alpelisib and sirolimus showed promising results but that additional long-term data are needed to validate efficacy and safety, and noted heterogeneity and potential risk of bias as limitations.
The evidence base remains limited by small sample sizes, lack of randomised controlled trials, and heterogeneous outcome definitions. The 2025 systematic review explicitly noted these limitations. What is still missing are adequately powered prospective trials, standardised response criteria, long-term safety data, and patient stratification by lesion type (macrocystic, microcystic, mixed) and genetic mutation status.
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.
Journal of Clinical Investigation · 2024 · 29 citations · open access
Lymphatic malformations: mechanistic insights and evolving therapeutic frontiers
AbstractThe lymphatic vascular system is gaining recognition for its multifaceted role and broad pathological significance. Once perceived as a mere conduit for interstitial fluid and immune cell transport, recent research has unveiled its active involvement in critical physiological processes and common diseases, including inflammation, autoimmune diseases, and atherosclerosis. Consequently, abnormal development or functionality of lymphatic vessels can result in serious health complications. Here, we discuss lymphatic malformations (LMs), which are localized lesions that manifest as fluid-filled cysts or extensive infiltrative lymphatic vessel overgrowth, often associated with debilitating, even life-threatening, consequences. Genetic causes of LMs have been uncovered, and several promising drug-based therapies are currently under investigation and will be discussed.
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.
Frontiers of Oral and Maxillofacial Medicine · 2019 · 1 citations · open access
Sclerotherapy for a giant lymphatic malformation in the neck: a case report
AbstractBackground: Lymphatic malformation is an abnormal development in the local area of the lymphatic system, often occurring in the head and neck of children within 2 years of age. Although this is a benign lesion, it rarely resolve spontaneously, enlarges and suppresses the vital organs around it, endangering life. Case Description: We herein reported a case of a neonate with a large lymphatic malformation in the neck. The lesion was about 8.0 cm × 8.0 cm × 3.60 cm at birth. After multidisciplinary jointed diagnosis and treatment, sclerotherapy with pingyangmycin combined with triamcinolone acetonide or lauromacrogol foam remained very effective. The child was followed up until 1 year and 5 months, and the growth and development of the child were good. Conclusions: This case may demonstrate that large lymphatic malformations in the neck do not necessarily require surgical removal, and sclerotherapy can be effective.
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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