DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hypercalcemia, infantile, 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 moduleHypercalcemia, infantile, 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 hypercalcemia, infantile, 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
Three case reports from Japan in 1960 describe idiopathic hypercalcemia of infancy with anorexia, constipation, vomiting, growth failure and mental impairment; serum calcium was elevated in all three, and nonprotein nitrogen was elevated in two. A 2017 retrospective review of 20 children with sustained hypercalcemia (median age 6.25 years, range 0.03–17.88) found that 30% were asymptomatic at presentation, and physical examination was normal in 20%. The median time to diagnosis was 45 days (range 2–720 days). Among the 20 patients, the most common causes were primary hyperparathyroidism (n=5), idiopathic infantile hypercalcemia (IIH) (n=5), and malignancy (n=4). Nephrocalcinosis occurred in 45% of patients (n=9). Serum calcium and creatinine showed a moderate positive correlation (r=0.53, p=0.02). Two patients with mild hypercalcemia received no treatment; others received medical treatment with or without surgery. Treatment-resistant cases were those with malignancy or neonatal severe hyperparathyroidism. On long-term follow-up, three infants diagnosed with IIH had persistent hypercalciuria.
A 2011 case report describes one infant with recurrent idiopathic infantile hypercalcemia, poor weight gain, constipation, and a renal stone. The infant was treated with a low-calcium diet and intravenous pamidronate, and the hypercalcemia resolved. No other patients treated with pamidronate are described in these abstracts. The 1960 case series predates modern bisphosphonate therapy and reports no specific treatment. The 2017 review does not specify which medical treatments were used for the IIH patients, nor does it report pamidronate use in that cohort.
What remains missing is a prospective trial of pamidronate or any other bisphosphonate specifically in children with genetically confirmed idiopathic infantile hypercalcemia, with standardised calcium monitoring and long-term renal outcomes. The 2017 review notes that mild cases may go unrecognised and that symptoms can be missed when other illnesses are present, so diagnostic delay is a persistent problem. No abstract provides data on the genetic basis of the condition or on the use of calcimimetics or other targeted therapies.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The Tohoku Journal of Experimental Medicine · 1960 · 10 citations · open access
Idiopathic Hypercalcemia of Infancy
AbstractThree cases of idiopathic hypercalcemia of infancy were presented. The present paper is the first report of this syndrome in Japan. In all the cases the typical clinical features such as anorexia, constipation, vomiting, growth failure and mental impairment were noticed. Serum calcium levels were elevated in all the cases. Nonprotein nitrogen level was elevated except in one case.
Journal of Clinical Research in Pediatric Endocrinology · 2017 · 9 citations · open access
Childhood Sustained Hypercalcemia: A Diagnostic Challenge
AbstractOBJECTIVE: This study aimed to call attention to hypercalcemia, a rare finding in children which carries the potential of leading to serious complications without proper intervention. METHODS: Diagnosis, treatment, and clinical course of children with sustained hypercalcemia admitted between the years 2006-2016 were reviewed. Group 1 [parathyroid hormone (PTH)-dependent] consisted of patients with high/unsuppressed PTH levels and group 2 (PTH-independent) included cases with normal/suppressed PTH levels. RESULTS: Twenty patients (11 male, 9 female) with a median age of 6.25 (0.03-17.88) years were evaluated. Symptoms were mostly related with the gastrointestinal system, while six patients (30%) were asymptomatic. Physical examination findings were diverse, non-specific, and normal in four patients (20%). Median time of diagnosis was 45 (2-720) days. Patients were divided into group 1 (n=7) and group 2 (n=13). Most frequent etiologies were primary hyperparathyroidism (n=5), idiopathic infantile hypercalcemia (IIH) (n=5), and malignancy (n=4). A moderate positive correlation was noted between serum calcium and creatinine levels (r=0.53, p=0.02). Nephrocalcinosis was the most common complication (n=9) (45%). Treatment was not implemented in 2 patients with mild hypercalcemia, while other patients received medical treatment ± surgery. Treatment-resistant patients were cases of malignancies and neonatal severe hyperparathyroidism. Long-term follow-up displayed resistant hypercalciuria in three infants diagnosed as IIH. CONCLUSION: Many patients with childhood hypercalcemia are asymptomatic or exhibit a non-specific and heterogeneous clinical presentation, resulting in delayed diagnosis. Mild cases may not be recognized, while symptoms may be missed in the presence of accompanying illnesses. Nevertheless, serious complications may only be avoided with prompt diagnosis and intervention.
Journal of Korean Society of Pediatric Endocrinology · 2011 · 0 citations · open access
A Case of Idiopathic Infantile Hypercalcemia Treated with Intravenous Pamidronate Infusion
AbstractHypercalcemia is not common, and occurs more frequently in children than in adults. Left untreated, hypercalcemia could result in a profound impact on growth and development. We report a case of recurrent idiopathic infantile hypercalcemia with poor weight gain, constipation, and a renal stone. We successfully treated the infantile hypercalcemia with a low-calcium diet and intravenous pamidronate. (
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.
