DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for lymphatic malformation — screening already-approved drugs against its 6-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 maps to a 6-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 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
FKBP prolyl isomerase 1A (FKBP1A) — FKBP1A 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 4~{s},5~{r},6~{z},9~{s},10~{s},12~{e}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6I1S · 1.52 Å · ligand (4~{S},5~{R},6~{Z},9~{S},10~{S},12~{E})-16-(ethylamino)-4,5-dimethyl-9,10,18-tris(oxidanyl)-3-oxabicyclo[12.4.0]octadeca-1(14),6,12,15,17-pentaene-2,8-dione (E26). Experimental structure, not a prediction.
What the evidence adds up to
A 2025 systematic review of medical therapies for paediatric lymphatic malformations of the soft tissues analysed 77 studies from 4937 screened abstracts. Treatment success was defined as a reduction in lesion size greater than 10%, and complete response as a reduction greater than 90%. Success rates varied widely: oral alpelisib succeeded in 9 of 9 patients, oral sirolimus in 257 of 287, topical sirolimus in 13 of 15, oral acetylsalicylic acid in 18 of 23, oral propranolol in 19 of 29, and oral sildenafil in 33 of 71. Complete responses were reported 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 heterogeneity and a potential risk of bias, and concluded that additional long-term data are needed to validate efficacy and safety.
A 2019 case report described a neonate with a large neck lymphatic malformation measuring 8.0 cm × 8.0 cm × 3.60 cm at birth. After multidisciplinary assessment, sclerotherapy with pingyangmycin combined with triamcinolone acetonide or lauromacrogol foam was reported as very effective. The child was followed to 1 year and 5 months with good growth and development. The authors stated that large neck lymphatic malformations do not necessarily require surgical removal and that sclerotherapy can be effective.
A 2022 case report described a 10-year-old boy with an orbital venolymphatic malformation causing proptosis and palpebral oedema. The lesion was initially treated with local sclerotherapy but relapsed, and was then successfully treated with oral sirolimus. The authors noted that prospective studies are warranted to determine the appropriate dose and extend the indications of sirolimus in these patients.
What is still missing are prospective, controlled trials with standardised outcome measures, long-term safety and efficacy data, and clear patient stratification by malformation subtype, location, and genetic profile. Funding for such trials and for comparative studies of the various 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.
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.
INDIGO (University of Illinois at Chicago) · 2022 · 0 citations · open access
Treatment of orbital lymphatic malformation with oral sirolimus: a case report
AbstractABSTRACT It is estimated that lymphatic malformations in children account for 6% of all benign vascular malformations. New medical therapies have been developed for the management of lymphatic orbital disease. The purpose of this article was to describe a clinical case of orbital venolymphatic malformation in a 10-year-old boy, causing proptosis and palpebral edema. The lesion was initially treated with local sclerotherapy. However, the lesion relapsed, and was successfully treated with oral sirolimus. Prospective studies are warranted to determine the appropriate dose and extend the indications of sirolimus in these patients.
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.
