DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for lymphatic malformation 3 — 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 3 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 3 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.
What the evidence adds up to
The 2004 review notes that 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. Surgical excision, staged when necessary, remains integral to management in many cases. The 2016 update on orbital lymphatic malformations states that all articles discussed emphasise the difficulty in treating lymphatic malformations, with most patients having long complicated histories with multiple recurrences. That review also says the use of sclerosing agents shows great promise for treating macrocysts.
A 2019 case report describes a neonate with a large neck lymphatic malformation (8.0 cm × 8.0 cm × 3.60 cm at birth). After multidisciplinary 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, with good growth and development. The authors conclude that large neck lymphatic malformations do not necessarily require surgical removal and that sclerotherapy can be effective. A 2025 case report presents a 3-year-old boy with a giant cervical lymphatic malformation that fully responded to a single doxycycline sclerotherapy injection session. That report states that doxycycline is a safe and successful agent with a response rate reaching 86%, and that different sclerosing agents have been used including ethanol, doxycycline, bleomycin, and OK432.
The 2024 review notes that genetic causes of lymphatic malformations have been uncovered and that several promising drug-based therapies are currently under investigation. The 2004 review calls for further identification of genes selectively expressed by lymphatic endothelium to facilitate analysis of the underlying biology, physiology, pathology, and treatment. The 2016 update says more knowledge is needed in the development of the disease process and expresses hope that antilymphangiogenic agents can provide targeted treatment. What remains missing are completed controlled trials comparing sclerosing agents head-to-head, validated biomarkers to predict which lesions will respond to which agent, and prospective studies with long-term follow-up that include patient stratification by cyst type and anatomical location.
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.
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.
Current Opinion in Ophthalmology · 2016 · 21 citations
Update on orbital lymphatic malformations
AbstractPURPOSE OF REVIEW: The purpose of this article is to review the recent published literature in 2015 on lymphatic malformation and provide updated information on the disease. Specifically, this article will describe recent advances in identifying and managing lymphatic malformations. RECENT FINDINGS: All articles discussed in this article emphasize the difficulty in treating lymphatic malformations. Most patients have long complicated histories with multiple recurrences. Studies are trying to identify which lesions will be successful treated by certain therapeutic modality. Treatment includes nonsurgical and surgical therapies. SUMMARY: Lymphatic malformations are difficult lesions to treat because they do not respect tissue planes. The use of sclerosing agents shows great promise for treating macrocysts. More knowledge is needed in the development of the disease process. Hopefully, antilymphangiogenic agent can provide targeted treatment.
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.
Saudi Journal of Otorhinolaryngology Head and Neck Surgery · 2025 · 0 citations
Doxycycline Sclerotherapy: The Occasional Dramatic Response in Cervical Lymphatic Malformations
AbstractCervical lymphatic malformations are congenital lesions diagnosed during infancy or later in life. The first line of treatment is surgical excision, which puts the patient at risk for high morbidity and mortality. Over the past 30 years, sclerotherapy has been arising as a plausible first-line option to treat lymphatic malformation. Different sclerosing agents have been used including ethanol, doxycycline, bleomycin, and OK432. Doxycycline is a safe and successful agent with a response rate reaching 86%. We, hereby, present the case of a 3-year-old boy with a giant lymphatic malformation that fully responded to a single doxycycline sclerotherapy injection session.
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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