DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for lymphatic malformation 1 — 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 1 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 1 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
fms related receptor tyrosine kinase 4 (FLT4) — FLT4 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 4BSJ · 2.5 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
A 2015 review of management options for lymphatic malformations in children lists nonoperative management, surgery, sclerotherapy, radiofrequency ablation, and laser therapy as current treatments, and notes that sildenafil, propranolol, sirolimus, and vascularised lymph node transfer are emerging therapies. The review states that the primary focus of management centres on the patient’s quality of life, and that multimodal treatment continues to expand as new information about the biology and genetics of these lesions is discovered. A 2016 update on orbital lymphatic malformations emphasises the difficulty in treating these lesions, noting that most patients have long complicated histories with multiple recurrences. That update states that sclerosing agents show great promise for treating macrocysts, but that more knowledge is needed in the development of the disease process.
A 2024 review describes lymphatic malformations as localised lesions that manifest as fluid-filled cysts or extensive infiltrative lymphatic vessel overgrowth, often with debilitating or life-threatening consequences. It reports that genetic causes of LMs have been uncovered and that several promising drug-based therapies are currently under investigation. No concrete numbers for survival, response rates, or sample sizes are provided in any of these three abstracts, and no specific drug is named as having demonstrated efficacy in a controlled trial.
What is still missing are completed prospective trials that report objective response rates and durable outcomes for any of the emerging drug therapies, as well as validated biomarkers to stratify patients by genetic subtype or lesion morphology. Funding for such trials and for the development of antilymphangiogenic agents remains limited.
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 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.
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.
Lymphatische Malformationen – neues System zur Beurteilung des Therapieerfolgs
AbstractLymphatische und gemischt venös-lymphatische Malformationen (LM, VLM) finden sich bei Kindern in zwei Drittel der Fälle in der Kopf-Hals-Region. Eine chirurgische Entfernung ist deshalb häufig schwierig, denn aufgrund der Nähe zu lebenswichtigen Strukturen können die Malformationen häufig nicht vollständig entfernt werden. Eine Alternative zur Operation ist die perkutane Sklerotherapie.
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.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
