DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for Central precocious puberty — 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 moduleCentral precocious puberty 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 central precocious puberty 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
delta like non-canonical Notch ligand 1 (DLK1) — DLK1 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 bgcdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 9D20 · 2.673 Å · ligand beta-D-glucopyranose (BGC). Experimental structure, not a prediction.
What the evidence adds up to
Treatment of central precocious puberty remains an area with many unresolved questions, including which hormonal test results best support the diagnosis, how best to predict adult height, and how the child's age affects height gained during treatment. Many studies show that children with onset of symptoms before age 6 benefit the most from therapy, but one recent study found no difference in height benefit between girls first seen at age 7 or older. A greater delay from the onset of puberty to the start of gonadotropin-releasing hormone analogue therapy has a negative effect on adult height according to two reports. Monthly injections of these analogues have been used extensively, but a slower-release formulation given every 3 months is also effective in the majority of patients, and a subcutaneous implant of the analogue histrelin provides gonadotropin suppression for 12 months.
Three drugs have been proven effective for precocious puberty, with cyproterone acetate described as producing exciting clinical remissions with virtually no known side effects. The condition is defined as the appearance of secondary sexual characteristics before age 8 in girls and before 9 in boys. Prevalence varies greatly by study, with the incidence generally thought to be 1 in 5,000 to 10,000 children, though some countries including the United States, Spain, France, Denmark, Korea and China have seen an increase since 1990. Complex interactions between genetic, nutritional and environmental factors determine the timing of puberty.
Mutations in four genes (KISS1, KISS1R, MKRN3, DLK1) have been confirmed as causal variants leading to central idiopathic precocious puberty. The condition can be confusing and frightening to affected children and their families. Radioimmunoassays for gonadotropins and several key hormones are now available to evaluate and follow patients.
What is still missing are large, prospective studies that resolve the ongoing uncertainties about which children benefit most from treatment, particularly regarding age thresholds and height outcomes. The evidence base lacks randomised trials comparing the different long-acting formulations head-to-head for long-term safety and efficacy. Better patient stratification by genetic cause, age at onset, and predicted height loss could refine treatment decisions, but such stratified trial data do not yet exist.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Current Opinion in Endocrinology Diabetes and Obesity · 2009 · 24 citations
Treatment of central precocious puberty
AbstractPURPOSE OF REVIEW: In consideration of the large number of the rapid advances in this area, it seems appropriate to highlight some of the recent studies that address factors involved in the decision to treat a child with central precocious puberty, and two recent advances in drug therapy. RECENT FINDINGS: There are still many areas of uncertainty regarding treatment of central precocious puberty, including the hormonal test results that support the diagnosis, the best way to predict adult height, and the effect of the age of the child on the amount of height gained during treatment (adult height minus predicted height). Many studies show that children with onset of symptoms before age 6 benefit the most, but a recent study showed no difference in height benefit between girls initially seen at at least 7 or more than 7 years. Two reports indicate that greater delay from the onset of puberty to the start of therapy with gonadotropin-releasing hormone analogue has a negative effect on adult height. Although there has been considerable experience with monthly injections of gonadotropin-releasing hormone analogues to suppress pubertal development, recent studies show that a slower released formulation given every 3 months is also effective in the majority of patients. The newest form of therapy involves a subcutaneous implant of the gonadotropin-releasing hormone analogue histrelin, which gives excellent gonadotropin suppression for 12 months. SUMMARY: Treatment of central precocious puberty continues to be a very active area of clinical investigation, but there are still unresolved questions that future studies will need to address.
Thieme Medical and Scientific Publishers Private Limited eBooks · 2017 · 2 citations · open access
29 A Child with Precocious Puberty
AbstractPrecocious puberty can be very confusing and frightening to an affected child and his or her family. The following presentation includes the pertinent physiology, psychology, etiology, diagnosis, and treatment of precocious pubertal development. Radioimmunoassays for gonadotropins and several key hormones are now available which enable the clinician to evaluate and follow the patient's condition with confidence and expertise. Three drugs have been proven effective and the most recent one, cyproterone acetate, has produced exciting clinical remissions with virtually no known side effects.
Zenodo (CERN European Organization for Nuclear Research) · 2023 · 0 citations · open access
PRECOCIOUS PUBERTY: MOLECULAR GENETICS, MODERN APPROACHES TO THE DIAGNOSIS AND TREATMENT
AbstractThe article is devoted to the basic molecular-genetic mechanisms and novel approaches to the diagnosis and management of the development of central precocious puberty. Precocious puberty is the appearance of the secondary sexual characteristics before the age years in girls and before 9 years in boys. Prevalence varies greatly by study. The incidence is generally thought to be 1 in 5000-10000 children, but some countries around the world have seen an increase in the incidence, including the United States, Spain, France, Denmark, Korea and China since 1990. Complex interactions with genetic, nutritional and environmental factors play a decisive role in determining the timing of puberty. To date, mutations in four genes (KISS1, KISS1R, MKRN3, DLK1) have been confirmed as causal variants leading to central idiopathic Precocious Puberty.
Zenodo (CERN European Organization for Nuclear Research) · 2023 · 0 citations · open access
PRECOCIOUS PUBERTY: MOLECULAR GENETICS, MODERN APPROACHES TO THE DIAGNOSIS AND TREATMENT
AbstractThe article is devoted to the basic molecular-genetic mechanisms and novel approaches to the diagnosis and management of the development of central precocious puberty. Precocious puberty is the appearance of the secondary sexual characteristics before the age years in girls and before 9 years in boys. Prevalence varies greatly by study. The incidence is generally thought to be 1 in 5000-10000 children, but some countries around the world have seen an increase in the incidence, including the United States, Spain, France, Denmark, Korea and China since 1990. Complex interactions with genetic, nutritional and environmental factors play a decisive role in determining the timing of puberty. To date, mutations in four genes (KISS1, KISS1R, MKRN3, DLK1) have been confirmed as causal variants leading to central idiopathic Precocious Puberty.
AbstractSince recent years, the incidence of precocious puberty has been rising. However, questions regarding its treatment and other conditions remain. This article reviews recent published papers with long-term outcome data and guidelines, and systemical introduction to identification of precocious puberty, different treatment options, therapeutic goals, and indications for different types of diseases, as well as adverse effects of drugs.
Key words:
Puberty, precocious; Gonadotropin-releasing hormone analogues; Growth hormone
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.
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