Rare & Orphan Lab · DeCure for X

DeCure for Cystic lymphangioma

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for cystic lymphangioma — screening already-approved drugs against its 5-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module5 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:3081$DeCureRare

The disease map

Disease moduleCystic lymphangioma maps to a 5-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 cystic lymphangioma 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

leucine zipper like post translational regulator 1 (LZTR1)LZTR1 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 gdpdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 9MEZ · 2.8 Å · ligand GUANOSINE-5'-DIPHOSPHATE (GDP). Experimental structure, not a prediction.

What the evidence adds up to

Two case reports describe adult cystic lymphangioma. A 27‑year‑old man and a 59‑year‑old man each presented with a neck mass; neither had pain, discolouration, discharge or sudden enlargement. The authors state that complete surgical removal is the proper management to prevent recurrence. A separate case describes a 45‑year‑old woman with a retroperitoneal cystic mass between the right kidney and liver, causing vague abdominal pain and intermittent vomiting for four months. The mass was excised laparoscopically, and histopathology confirmed cystic hygroma. The authors note that complete resection may be curative.

A 2022 paediatric case report describes the use of sirolimus for a macrocytic lymphangioma. Sirolimus is an immunosuppressive and antitumour agent that blocks the mTOR/PI3K pathway, inhibiting abnormal vascular proliferation. The authors report that it was effective and well‑tolerated in that single patient, with nausea, cytopenias and metabolic imbalances listed as the most significant potential adverse effects.

No controlled trials, no survival data, no response rates, and no sample sizes beyond single‑patient reports are provided. The adult cases rely entirely on surgery; the paediatric case is a single anecdote of drug treatment. What is missing is any prospective trial, any comparison of sirolimus against surgery or observation, any data on long‑term recurrence after drug therapy, and any stratification by patient age, tumour site or genetic markers. Funding for a randomised trial in this rare disease would be needed to move beyond case reports.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Turkish Journal of Pathology · 2011 · 12 citations · open access

Adult lymphangioma - a rare entity: a report of two cases

AbstractOBJECTIVE: Lymphangiomas are rare congenital malformations commonly seen in children. Its occurrence in adults is uncommon with very scarce reports in the literature. It mainly occurs in infants or children younger than two years of age. We report these cases to emphasize the need to consider cystic lymphangiomas in differential diagnosis of neck masses in adults. CASE REPORTS: We describe two cases in adults, one a 27-year-old man and the other a 59-year-old male. The former presented with recurrent neck mass for 5 years, and the latter presented with asymptomatic neck mass of a short duration. Both were not associated with any pain, discoloration, discharge or sudden increase in size. CONCLUSION: The awareness of occurrence of cystic lymphangioma in adults is important for its proper management which includes complete surgical removal, to prevent recurrence.

https://doi.org/10.5146/tjpath.2012.01103
International Journal of Surgery Case Reports · 2022 · 5 citations · open access

A rare incidence of retroperitoneal Cystic Lymphangioma in a 45-year-old female - A Case Report

AbstractINTRODUCTION AND IMPORTANCE: Cystic lymphangioma is an extremely rare benign vascular neoplasm of mesodermal origin, arising from lymphatic vessels and occurring principally in male children. The retroperitoneum is the rarest site, accounting for <1 % of all types of lymphangioma. The incidence of cystic lymphangioma is unknown due to the scarcity of published data. CASE PRESENTATION: A 45-year-old female presented to our hospital complaining of vague abdominal pain and intermittent episodes of vomiting over four months. Preoperative imaging via ultrasound and Computed Tomography revealed a well-demarcated retroperitoneal cystic mass between the right kidney and the liver. CLINICAL DISCUSSION: The mass was excised laparoscopically. Histopathological examination confirmed a cystic hygroma (a subtype of cystic lymphangioma). CONCLUSION: Cystic lymphangioma is rare, often misdiagnosed and may present with vague symptoms. Complete resection may be feasible and can be curative.

https://doi.org/10.1016/j.ijscr.2022.107606
Portuguese Journal of Dermatology and Venereology · 2022 · 0 citations · open access

Sirolimus in the treatment of cystic lymphangioma in a pediatric patient

AbstractCystic lymphangioma (CL) is a rare benign tumor, which occurs typically during childhood, with craniofacial, cervical or axillary being the most common locations. Lymphangiomas management can be challenging due to their permeative growth throughout tissue layers. Sirolimus is an immunosuppressive and antitumor agent that can inhibit abnormal vascular proliferation by blocking the mTOR/PI3K pathway. It is typically well-tolerated, with nausea, cytopenias, and metabolic imbalances as the most significant adverse effects. We present the case of a pediatric patient in which sirolimus was used to treat a macrocytic lymphangioma, highlighting its effectiveness and safety.

https://doi.org/10.24875/pjdv.m22000035

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.