DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for caudal regression sequence — screening already-approved drugs against its 2-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCaudal regression sequence maps to a 2-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 caudal regression sequence 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
VANGL planar cell polarity protein 1 (VANGL1) — VANGL1 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 9JK9 · 2.2 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
Caudal regression syndrome is a congenital malformation of the lower spine, often accompanied by defects in the hindgut, genitourinary system, and lower limbs. A 1999 case report describes a patient with total absence of the lumbar vertebral column but an intact spinal cord and bony sacrum, an unusual presentation. A 2013 case used computed tomography to document megacolon in a patient with caudal regression syndrome who had been constipated for about 25 days. No treatment or intervention is described in any of these reports.
The strongest known association is with maternal pregestational diabetes mellitus and poor metabolic control. A 2003 paper reports a case diagnosed by ultrasound at 17 weeks of pregnancy in a diabetic woman, noting abrupt interruption of the lumbar spine and a Buddha-like posture of the lower limbs. A 2016 case describes a stillborn fetus delivered at 22 weeks of gestation to a mother with uncontrolled pregestational diabetes and polyhydramnios. The authors call the syndrome a “unique complication of maternal diabetes mellitus.”
No drug, no therapy, and no animal model are mentioned in any of these abstracts. There is no evidence of any pharmacological intervention being tested or proposed for caudal regression syndrome. What is missing is any clinical trial, any attempt at drug repurposing, any funding for preclinical work, and any systematic effort to stratify patients by the severity of spinal or visceral involvement.
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 Neuroimaging · 1999 · 4 citations
Lumbar Agenesis with Sacral Sparing: a Case Report
AbstractCaudal agenesis or caudal regression syndrome refers to a spectrum of malformations involving the lower spine, which can result in significant progressive or permanent neurologic defects. Caudal agenesis is frequently associated with other congenital anomalies of the axillary skeleton, hindgut and genitourinary systems. Described in this paper is a patient who presented with multiple clinical and radiographic findings consistent with caudal agenesis. This case is unique in that there is total absence of the lumbar vertebral column with an intact spinal cord and bony sacrum.
Progresos de Obstetricia y Ginecología · 2003 · 2 citations
Diagnóstico prenatal temprano de síndrome de regresión caudal asociado con diabetes mellitus
AbstractEl síndrome de regresión caudal (SRC) es un cuadro malformativo que se observa con mayor frecuencia en los embarazos de madres con diabetes mellitus pregestacional, con mal control metabólico. En este artículo describimos los hallazgos ecográficos, que incluyen la brusca interrupción de la columna lumbar y la posición en postura de Buda de los miembros inferiores, encontrados en un caso de SRC diagnosticado a las 17 semanas de embarazo en una gestante con diabetes mellitus pregestacional. The caudal regression syndrome (CRS) is a congenital anomaly which is most often found in offspring of mother with pregestational diabetes mellitus with poor metabolic control. In this article we describe the ultrasonographic findings, which include abrupt interruption of the lumbar spine and Buda position of the lower limbs, found in a CRS case diagnosed at 17 weeks of pregnancy in a woman with pregestational diabetes mellitus.
International Journal of Science and Research (IJSR) · 2016 · 0 citations · open access
Caudal Regression Syndrome: A Journey from Sweet to Sour: A Unique Complication of Maternal Diabetes Mellitus: A Case Report of a Still Born Fetus
AbstractCaudal regression syndrome is a rare congenital disorder that encompasses a spectrum of anomalies. A case of caudal regression syndrome and polyhydraminios that was associated with uncontrolled pre-gestational diabetes mellitus is presented. The outcome was a still born fetus delivered at the gestational age of 22 weeks.
Computed Tomography Findings of a Case of Megacolon Associated with Caudal Regression Syndrome
AbstractCaudal regression syndrome is a congenital disease that affects the development of distal spinal segments and is reflected by abnormalities in the pelvis and genitourinary and gastrointestinal systems along with severe deformities in the extremities and spinal problems. Our goal was to report the computed tomography (CT) findings of a case of megacolon associated with caudal regression syndrome (CRS) that presented with constipation that had lasted for approximately 25 days. doi: http://dx.doi.org/10.4021/jmc1321w
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.
