DeCure for Isolated congenital growth hormone deficiency
DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for isolated congenital growth hormone deficiency — 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 moduleIsolated congenital growth hormone deficiency 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 isolated congenital growth hormone deficiency 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
growth hormone releasing hormone receptor (GHRHR) — GHRHR 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 plmdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7CZ5 · 2.6 Å · ligand PALMITIC ACID (PLM). Experimental structure, not a prediction.
What the evidence adds up to
A 1981 report describes a family with isolated growth hormone deficiency affecting two children, their mother, and presumably two maternal ununts and their maternal grandmother, with autosomal dominant inheritance proposed. The paper notes that isolated growth hormone deficiency is a heterogeneous condition that includes autosomal dominant, autosomal recessive, and non-genetic forms. No other drugs or interventions are discussed in that abstract.
A 2006 review discusses the rationale for using growth hormone therapy in paediatric conditions not strictly related to growth hormone deficiency, stating that linear growth is only one of many expected effects. The review does not present original data, survival figures, or response rates for any specific condition, and it does not address isolated congenital growth hormone deficiency directly.
A 2011 article presents diagnostic criteria and treatment results for children with growth hormone deficiency. It reports that treatment effectiveness depends on the patient's status at the time of treatment and on the dose and schedule of recombinant growth hormone. A significant increase in IGF-1 and IGF-binding protein-3 during treatment, compared to baseline, is described as a criterion for both diagnosis and treatment effectiveness. No concrete numbers for survival, response rates, or sample sizes are given in the abstract.
What is still missing: randomised controlled trials comparing different dosing schedules in isolated congenital growth hormone deficiency, long-term safety data beyond growth outcomes, and studies that stratify patients by genetic subtype (dominant, recessive, sporadic) to see whether response differs. No drug other than recombinant growth hormone is mentioned in these abstracts.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Clinical Genetics · 1981 · 9 citations
Familial isolated growth hormone deficiency
AbstractA family is reported with isolated growth hormone deficiency in two children, their mother and, presumably, also in two maternal uncles and their maternal grandmother. Autosomal dominant inheritance is the best explanation. Isolated growth hormone deficiency is apparently a heterogeneous condition, including autosomal dominant, autosomal recessive as well as non-genetic diseases.
AbstractUNLABELLED: Human growth hormone therapy is allowed in certain clinical conditions according to national healthcare criteria. Growth hormone, however, produces a wide spectrum of effects. Linear growth is only one of the many expected results, and there are interesting possibilities to explore which could provide additional means of improving the quality of life for the ever-increasing numbers of chronic paediatric patients. CONCLUSION: In this review, we discuss the rationale for and possibility of using growth hormone therapy in some conditions not strictly related to growth hormone deficiency.
Клінічна ендокринологія та ендокринна хірургія · 2011 · 0 citations
Діагностика та лікування соматотропної недостатності у дітей
AbstractThe article presents criteria of the diagnostics and results of treatment of children with growth hormone deficiency. Effectiveness of treatment of children with above mentioned deficiency depends on the status of the patient at the time of treatment and on the dose and schedule of administration of recombinant growth hormone. Significant increase in IGF -1 and IGF – binding protein – 3 is seen in the process of treatment with growth hormone in comparison with their background values allows us to use them not only as diagnostic criteria of growth hormone deficiency but also as criteria of effectiveness of treatment with growth hormone as well.
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.
