Rare & Orphan Lab · DeCure for X

DeCure for Precocious puberty, central, 2

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for precocious puberty, central, 2 — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module1 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:0112309$DeCureRare

The disease map

Disease modulePrecocious puberty, central, 2 maps to a 1-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 precocious puberty, central, 2 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.

What the evidence adds up to

Treatment of central precocious puberty remains an active area of clinical investigation with unresolved questions. A 2009 review noted that many studies show children with onset of symptoms before age 6 benefit most from treatment, but one recent study found no difference in height benefit between girls first seen at age 7 or older. Greater delay from puberty onset to starting a gonadotropin-releasing hormone analogue negatively affects adult height. Monthly injections of these analogues have been used extensively, but a slower-release formulation given every 3 months is effective in most patients. A subcutaneous implant of the analogue histrelin provides gonadotropin suppression for 12 months.

A 2017 presentation stated that three drugs have been proven effective for precocious puberty, with the most recent, cyproterone acetate, producing clinical remissions with virtually no known side effects. A 2020 clinical guideline was based on three systematic reviews covering interventions for treatment (outcomes including final height, mental health, metabolic health, bone health, or blockade success), long-term outcomes in observational studies, and diagnostic test accuracy.

A 2023 article reported that central precocious puberty is defined by secondary sexual characteristics appearing before age 8 in girls and before 9 in boys. Incidence is generally 1 in 5000–10,000 children, but some countries have seen increases since 1990. Mutations in four genes (KISS1, KISS1R, MKRN3, DLK1) are confirmed causal variants for idiopathic cases. The 2009 review emphasised that there are still many areas of uncertainty, including which hormonal test results support the diagnosis, how best to predict adult height, and the effect of the child's age on height gained during treatment.

What is still missing are prospective trials that stratify patients by age at onset and genetic subtype, long-term data on metabolic and bone health outcomes beyond final height, and studies comparing the newer implant and quarterly formulations head-to-head with monthly injections in diverse populations.

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.

https://doi.org/10.1097/med.0b013e328320a650
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.

https://doi.org/10.1055/b-0042-186601
PubMed · 2020 · 0 citations

Synthesis of the evidence: Clinical guideline for the diagnosis and treatment of precocious puberty.

AbstractThree systematic reviews were conducted to formulate the recommendations on diagnosis, treatment and follow-up of patients with precocious puberty: interventions for the treatment of precocious puberty that included the outcomes of final or near-final height, mental health, metabolic health, health bone, or blockade success; comparative observational studies evaluating long-term outcomes in subjects with a history of precocious puberty; and diagnostic test accuracy studies for puberty.

https://doi.org/10.24875/bmhim.20000087
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.

https://doi.org/10.5281/zenodo.8264456

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.