DeCure's autonomous Cardio AI scientist is researching a drug-repurposing hypothesis for congenital heart disease — screening already-approved drugs against its 43-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCongenital heart disease maps to a 43-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 congenital heart disease 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
mitogen-activated protein kinase 1 (MAPK1) — MAPK1 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 2~{s}drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8AOJ · 1.12 Å · ligand 1-[(2~{S})-2-(5-methyl-3-pyridin-4-yl-1~{H}-pyrazol-4-yl)pyrrolidin-1-yl]propan-1-one (N8L). Experimental structure, not a prediction.
What the evidence adds up to
The number of adults with congenital heart disease is increasing because paediatric cardiology, surgery and intensive care have shifted mortality away from infancy toward adulthood. A 2014 review notes that as these patients age they acquire cardiovascular risk factors at rates similar to the general population, so acquired heart conditions now contribute substantially to outcome. The review calls for more attention to prevention, detection and therapy of those acquired conditions.
A 2023 review states that drug therapy in adult congenital heart disease remains largely empiric because clinical data are lacking and formal guidelines do not exist. Pharmacotherapy, with few exceptions, is supportive; significant structural abnormalities usually require interventional, surgical or percutaneous treatment. The review identifies knowledge gaps in the use of cardiac drugs and says further research is needed to determine the most effective treatment options.
Heart transplantation for congenital heart disease has been performed since 1967. A 2011 review notes that primary infant heart transplant is limited by donor organ shortages, and that a growing area is rescue therapy for older patients with end-stage heart failure after palliative procedures, particularly those with single-ventricle hearts, systemic right ventricles and associated arrhythmias. A 2015 retrospective cohort study of 110 Congolese children who had surgery in France reports a 5-year survival rate of 90% and a 20-year survival rate of 83.3%, concluding that heart surgery for congenital heart defects has improved survival. A 2004 review describes rapid developments in transcatheter treatment, including device closure of septal defects and patent ductus arteriosus, cutting balloons and covered stents for stenosis, and percutaneous aortic and pulmonary valve implantations, as well as hybrid surgical-interventional procedures for complex disease. A 2018 review states that an estimated 95% of patients with congenital heart disease in the Western world now reach adulthood, but that long-term morbidity and mortality remain decreased compared with healthy counterparts.
What is still missing are large-scale clinical trials to generate evidence for drug therapy in adults with congenital heart disease, adequate donor organs for transplantation, and better patient stratification to identify who will benefit from transcatheter versus surgical procedures.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Heart · 2014 · 71 citations
Acquired heart conditions in adults with congenital heart disease: a growing problem
AbstractThe number of adults with congenital heart disease is increasing due to the great achievements in the field of paediatric cardiology, congenital heart surgery and intensive care medicine over the last decades. Mortality has shifted away from the infant and childhood period towards adulthood. As congenital heart disease patients get older, a high prevalence of cardiovascular risk factors is encountered similar to the general population. Consequently, the contribution of acquired morbidities, especially acquired heart conditions to patient outcome, is becoming increasingly important. Therefore, to continue the success story of the last decades in the treatment of congenital heart disease and to further improve the outcome of these patients, more attention has to be given to the prevention, detection and adequate therapy of acquired heart conditions. The aim of this review is to give an overview about acquired heart conditions that may be encountered in adults with congenital heart disease.
Journal of Cardiovascular Development and Disease · 2023 · 6 citations · open access
Cardiac Drugs in ACHD Cardiovascular Medicine
AbstractAdult congenital heart disease (ACHD) is a growing population that requires life-long care due to advances in pediatric care and surgical or catheter procedures. Despite this, drug therapy in ACHD remains largely empiric due to the lack of clinical data, and formalized guidelines on drug therapy are currently lacking. The aging ACHD population has led to an increase in late cardiovascular complications such as heart failure, arrhythmias, and pulmonary hypertension. Pharmacotherapy, with few exceptions, in ACHD is largely supportive, whereas significant structural abnormalities usually require interventional, surgical, or percutaneous treatment. Recent advances in ACHD have prolonged survival for these patients, but further research is needed to determine the most effective treatment options for these patients. A better understanding of the use of cardiac drugs in ACHD patients could lead to improved treatment outcomes and a better quality of life for these patients. This review aims to provide an overview of the current status of cardiac drugs in ACHD cardiovascular medicine, including the rationale, limited current evidence, and knowledge gaps in this growing area.
World Journal for Pediatric and Congenital Heart Surgery · 2011 · 5 citations
Heart Transplantation for Congenital Heart Disease
AbstractCongenital heart disease affects 0.8% of all live-born infants. Some of the malformed hearts can at best be palliated by conventional surgical or catheter interventions from the start. Others fail slowly from chronic overloading. Patients with congenital heart disease have been among the first transplant recipients since 1967. Primary therapy with infant heart transplant is a convincing concept from an immunological perspective but large-scale implementation is limited by donor organ shortages. Another growing area is rescue therapy for older patients with end-stage heart failure after palliative procedures, particularly those with single-ventricle hearts, systemic right ventricles, and associated arrhythmias.
Epidemiology and outcome of Congolese children who had surgery for heart defects
AbstractAIMS: to determine the principal heart defects for which children underwent surgery and to determine the survival rate. PATIENT AND METHODS: this retrospective cohort study involves Congolese babies treated surgically from September 1989 to September 2010 in France for congenital heart defects (through "Mécénat chirurgie cardiaque" and "Chaîne de l'espoir"). It includes only 110 of the 182 recorded patients during the study period. RESULTS: The sex ratio for the 110 subjects included in the analysis was 1. Their mean age at surgery was 77.4 ± 57.6 months old (range: 8 to 204 months). The main congenital heart defects for which surgery was performed were ventricular septal defect (21.9%), tetralogy of Fallot either isolated (22.8%) or associated with patent foramen ovale (1.8%) or coronary anomalies (1.8%), atrial septal defect associated with other malformations (8.2%), pulmonary atresia with ventricular septal defect (5.5%), aortic stenosis (3.7%), atrioventricular septal defect (0.9%), and Laubry-Pezzi syndrome (0.9%). The median length of follow-up was 42.4 ± 35.6 months (range, 3-240 months). Patients' mean age at the study's end was 121.1 ± 86.3 months (range 20-372 months). The 5-year survival rate was 90% and the 20-year survival, 83.3%. CONCLUSION: Heart surgery for congenital heart defects has improved survival.
Current Opinion in Anaesthesiology · 2004 · 2 citations
New developments in the transcatheter treatment of congenital heart disease
AbstractPURPOSE OF REVIEW: This review summarizes recent developments in the field of interventional paediatric cardiology. This is a rapidly evolving field with many new techniques that have been optimized or introduced during the past few years. RECENT FINDINGS: Important new data have emerged on the device closure of atrial septal defects, ventricular septal defects and patent ductus arteriosus. The technology has been improved further and more results on complications and problems have become available. New technical developments in the treatment of stenosis of the pulmonary arteries and the aorta, such as the use of cutting balloons and (covered) stents, have resulted in new treatment options for congenital disorders that were not considered suitable for percutaneous treatment. Fascinating emerging technologies such as percutaneous aortic and pulmonary valve implantations have been introduced and the first results have been reported. Changes in imaging technology such as intracardiac echocardiography and magnetic resonance imaging affect interventional cardiology. These changes result in a new approach to congenital heart disease in which hybrid surgical and interventional procedures are used to treat complex disease. SUMMARY: Interventional congenital cardiology is a rapidly growing field that offers new treatment options for patients with congenital heart disease.
AbstractCongenital heart disease (CHD) is the most common type of congenital malformation, representing a major public health problem affecting the health of Chinese children.Great progress has been made in many aspects in the treatment of CHD in China.The vast majorities of children with CHD have been treated and they have survived for a long time, but cardiac dysfunction and pulmonary hypertension are still common complications of it before or after surgery.In this paper, the advances in drug treatment of CHD in children in recent years were mainly discussed, focused on the evaluation, treatment and management of CHD with heart failure and pulmonary hypertension in order to further improve the pediatrician′s therapeutic level of managing common complications of CHD.
Key words:
Congenital heart disease; Child; Treatment
Oxford University Press eBooks · 2018 · 0 citations
Long-term outcomes
AbstractAbstract Due to tremendous improvements in corrective surgery, and medical therapy, survival of patients with congenital heart disease has improved dramatically over the past decades, with an estimated 95% of such patients in the Western world currently reaching adulthood. Nonetheless, patients with congenital heart disease have decreased long-term outcomes in terms of morbidity and mortality compared to their healthy counterparts.
Zenodo (CERN European Organization for Nuclear Research) · 2024 · 0 citations · open access
TETRALOGY OF FALLOT-COMPLEX GENETIC NETWORK RELATED CONGENITAL HEART DEFECT
AbstractAccording to many cases, congenital heart disease (CHD) is represented by a complex phenotype and an array of several functional and morphological cardiac disorders. One widespread defect that thousand suffer from is Tetralogy of Fallot in which a baby is born with four abnormalities in the period of their heart developed. These issues make it hard for the baby’s heart to send enough oxygen to their entire body. In this article, I illustrate most widespread causes of this disease and experienced methods to avoid and number of ways of treatment. Despite this defect is rare in these days, it is still observed in many countries even in medicine properly developed one.
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.
