DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for pseudohypoparathyroidism type 1B — screening already-approved drugs against its 3-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease modulePseudohypoparathyroidism type 1B maps to a 3-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 type 1b 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 is a rare heterogeneous group of disorders defined by hypocalcemia, hyperphosphatemia, elevated serum parathyroid hormone, and resistance to its biologic activity in the kidney. The condition was first described in 1942. Clinically, patients may present with short stature, round face, short metacarpals, intellectual disability, and seizures. Abnormal calcifications are seen on x-ray, particularly in the basal ganglia and ligamentous structures. By 1964, a total of 150 cases had been described in the world literature, 13 of which were from a single author's series.
A 2024 report on pseudohypoparathyroidism type IB describes a patient with subclinical hypothyroidism and presents a pedigree investigation. The abstract does not provide any quantitative data on response rates, survival, or sample sizes beyond the single case. A 2022 report describes a Tunisian family with pseudohypoparathyroidism type 1a and a novel GNAS mutation, but again gives no numerical outcomes. A 1971 case report reiterates the classic clinical features but provides no treatment results or patient numbers.
No drug treatment is mentioned in any of these abstracts. There is no evidence of any therapeutic intervention being tested, no response rates, and no survival data. The abstracts are limited to case descriptions, genetic findings, and literature reviews. What is missing is any clinical trial, any tested drug, any measure of treatment efficacy, and any systematic patient stratification beyond single families or case reports.
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 Case Reports · 2022 · 3 citations · open access
Fahr syndrome discovered in adulthood revealing a rare <i>GNAS</i> mutation in pseudohypoparathyroidism type 1a in a Tunisian family
AbstractAbstract Pseudohypoparathyroidism (PHP) indicates a rare heterogeneous group of disorders characterized by hypocalcemia, hyperphosphatemia, increased serum concentration of parathyroid hormone (PTH), and insensitivity to the biologic activity of PTH. One of its most common types is PHP‐1a. In this report, we present a familial PHP‐1a and a novel mutation of the GNAS gene.
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.
Diabetes Metabolic Syndrome and Obesity · 2024 · 0 citations · open access
Pseudohypoparathyroidism Type IB with Subclinical Hypothyroidism: a Pedigree Investigation and Literature Review
AbstractPseudohypoparathyroidism (PHP) is a rare genetic disease characterized by hypocalcemia, hyperphosphatemia, and elevated parathyroid hormone (PTH) in serum. Here, we report a case of a patient with pseudohypoparathyroidism type IB (PHPIB) and subclinical hypothyroidism, analyze the clinical and genetic data of his family members, review the relevant literature, and classify and discuss the pathogenesis and clinical characteristics of each subtype. Finally, we discuss the treatment approach to improve clinicians' understanding of the disease.
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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