DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for pseudohypoparathyroidism — 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 modulePseudohypoparathyroidism 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 pseudohypoparathyroidism 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
GNAS complex locus (GNAS) — GNAS 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 oladrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8WW2 · 2.79 Å · ligand OLEIC ACID (OLA). Experimental structure, not a prediction.
What the evidence adds up to
Pseudohypoparathyroidism was first described in 1942 as a failure of end organ response in the kidney to parathyroid hormone. Clinically, patients present with short stature, round face, short metacarpals, low serum calcium, and increased serum phosphorus. Seizures are frequent, and abnormal calcifications appear on x-ray, particularly in the basal ganglia and ligamentous structures. By 1964, 150 cases had been described in the world literature, 13 of which were from a single author’s own series.
Pseudohypoparathyroidism type Ia is caused by heterozygous inactivating mutation of the GNAS1 gene, which encodes the signal transducer Gsalpha. This type is associated with Albright’s osteodystrophy. Patients who have the Albright phenotype without hormone resistance are classified as having pseudopseudohypoparathyroidism.
No treatment trials, drug interventions, or outcome data are reported in these abstracts. No concrete numbers on survival, response rates, or sample sizes beyond the 1964 case count are given. The abstracts contain no mention of any drug.
What is still missing: any controlled trial of a therapeutic agent, any measurement of treatment effect on calcium or phosphorus levels, any patient stratification by GNAS1 mutation type, and any funding for clinical studies in this rare disease.
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 Journal of Clinical Endocrinology & Metabolism · 1956 · 12 citations
CONCERNING THE TERM “PSEUDO-PSEUDOHYPOPARATHYROIDISM”
AbstractJournal Article CONCERNING THE TERM “PSEUDO-PSEUDOHYPOPARATHYROIDISM” Get access GÜNTHER A. FROMM, M.D. GÜNTHER A. FROMM, M.D. 1Endocrinological Department, Instituto de Círugia of the Province of Buenos Aires, Haedo (F.C.N.D.F.S.) Republica Argentina Search for other works by this author on: Oxford Academic Google Scholar The Journal of Clinical Endocrinology & Metabolism, Volume 16, Issue 2, 1 February 1956, Pages 293–295, https://doi.org/10.1210/jcem-16-2-293 Published: 01 February 1956
Guthrie Journal · 1971 · 1 citations · open access
Pseudohypoparathyroidism: A Case Report
AbstractPseudohypoparathyroidism was first described by Albright and his associates in 1942 1 . It is characterized by a failure of end organ response in the kidney to parathyroid hormone. Clinically, such patients are characterized by short stature, round face, and short metacarpals; they have low serum calciums and increased serum phosphorous. In addition they may be retarded, and seizures are a frequent occurrence. Abnormal calcifications are seen on x-ray, particularly on the basal ganglion and in ligamentous structures.
Experimentelle Medizin, Pathologie und Klinik Band 15 Pseudohypoparathyroidismus und Pseudo-Pseudohypoparathyroidismus vol 15.
AbstractThis monograph contains an excellent review of the world literature on pseudohypoparathyroidism and pseudo-pseudohypoparathyroidism. A total of 150 cases had been described by 1964, 13 of which are the author's own cases. The review deals with clinical aspects, genetics and contains a good discussion of pathophysiology. It is well tabulated, and the illustrations are of high quality. The bibliography is very complete. The reviewer is unaware of a similar presentation in the English literature.
[GNAS1 gene abnormality in pseudohypoparathyroidism I a].
AbstractPseudophypoparathyroidism (PHP) is characterized by hypocalcemia, hyperphosphatemia and elevated levels of parathyroid hormone (PTH) due to resistance to PTH. PHP type I a is caused by heterozygous inactivating mutation of the GNAS1 gene, which encodes signal transducer, Gsalpha. PHP type I a is associated with Albright's osteodystrophy (AHO). Those patients who have AHO phenotype without hormone resistance are affected by pseudopseudohypoparathyroidism.
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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