DeCure for Megacystis-microcolon-intestinal hypoperistalsis syndrome 1
DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for megacystis-microcolon-intestinal hypoperistalsis syndrome 1 — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMegacystis-microcolon-intestinal hypoperistalsis syndrome 1 maps to a 4-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 megacystis-microcolon-intestinal hypoperistalsis syndrome 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
leiomodin 1 (LMOD1) — LMOD1 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 atpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4Z94 · 2.4 Å · ligand ADENOSINE-5'-TRIPHOSPHATE (ATP). Experimental structure, not a prediction.
What the evidence adds up to
In a 1980 case, a male infant with megacystis-microcolon-intestinal hypoperistalsis syndrome showed no response to metoclopramide or caerulein. The authors noted that no other male cases had been reported in the literature at that time. A 1995 report described two neonates with microcolon-intestinal hypoperistalsis without megacystis; in both, drugs that stimulate bowel movement failed to induce peristalsis, and ileostomy did not function postoperatively. Biopsy showed normal ganglion cell number and appearance throughout the intestinal wall. A 2017 case of a 4-day-old male neonate with the syndrome presented with abdominal distension from intestinal obstruction and massive bladder enlargement, and was initially misdiagnosed as a mesenteric cyst on ultrasound.
Across these reports, no pharmacological agent tested — metoclopramide, caerulein, or unspecified drugs that stimulate bowel movement — produced any peristaltic response. The syndrome remains a functional obstruction without an identifiable organic lesion or ganglion cell abnormality. The 2017 report emphasises that the condition is rare and easily missed in emergency settings.
What is still missing is any evidence from a controlled trial, any drug that has shown even a partial effect on peristalsis in these patients, and any systematic attempt to stratify patients by genetic or histological subtype. The sample sizes are tiny, follow-up is absent, and no funding for a dedicated therapeutic trial has been reported.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Radiology · 1980 · 33 citations
Megacystis-microcolon-intestinal hypoperistalsis syndrome in a male infant.
AbstractMegacystis-microcolon-intestinal hypoperistalsis syndrome was observed in a male infant. Review of the literature failed to turn up any other cases involving males. The sonogram revealed megacystis, hydronephrosis, and a normal amount of amniotic fluid. The hypoperistalsis failed to respond to several pharmacological agents, including metoclopramide and caerulein.
European Journal of Pediatric Surgery · 1995 · 13 citations
Microcolon-Intestinal Hypoperistalsis Without Megacystis: Uncommon Form of Neonatal Intestinal Pseudoobstruction
AbstractTwo cases of microcolon-intestinal hypoperistalsis without megacystis are reported. They had dilated proximal small bowel and narrowed distal small bowel and malrotated microcolon. No organic obstructive intestinal lesion was found and double-barrel ileostomy was performed. The biopsy specimens showed ganglion cells to be normal in number and appearance in the entire intestinal wall. The ileostomy did not function postoperatively and drugs stimulating bowel movement failed to induce peristalsis. We have suggested that microcolon-intestinal hypoperistalsis without megacystis may be the cause of functional intestinal obstruction in neonates and it is a variant of megacystis-microcolon-hypoperistalsis syndrome.
Pediatric Urology Case Reports · 2017 · 1 citations · open access
Megacystis microcolon intestinal hypoperistalsis syndrome presenting as acute intestinal obstruction in 4-days male neonate: A rare case report
AbstractWe report a case of megacystis microcolon intestinal hypoperistalsis syndrome in a 4-days old male neonate. The patient presented with abdominal distension due to intestinal obstruction and massive enlargement of the urinary bladder and was misdiagnosed as the mesenteric cyst on ultrasonography abdomen. The aim of this report is to highlight the fact that due to the rarity of this syndrome, it can be missed in the emergency room and a high index of suspicion should be kept for timely intervention.
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.
