DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for porphyria cutanea tarda — 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 modulePorphyria cutanea tarda 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 porphyria cutanea tarda 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
uroporphyrinogen decarboxylase (UROD) — UROD 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 1cpdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 1R3S · 1.65 Å · ligand COPROPORPHYRINOGEN I (1CP). Experimental structure, not a prediction.
What the evidence adds up to
Twenty-one patients (twenty men, one woman) with porphyria cutanea tarda were treated with oral chloroquine 125 mg twice weekly. Nineteen took the drug for an average of 8 to 5 months before complete clinical and biochemical remission occurred. Treatment initially caused a considerable increase in urinary uroporphyrin and a mild increase in serum transaminases, but no adverse clinical reactions were observed. The mechanism of chloroquine action in porphyria cutanea tarda had not been clarified, and the authors cautioned that special care must be taken when using it therapeutically.
Seven patients received ten courses of low-dose oral chloroquine (125 mg chloroquine phosphate twice weekly) for a mean of 14.9 months. All went into clinical and biochemical remission. Relapse occurred in four patients on six occasions after a mean 17 months. In four patients there was no relapse after a mean 47.3 months. There were no adverse side-effects. The authors concluded that low-dose oral chloroquine appears safe, effective and convenient, though relapses may occur requiring further therapy.
Four children with porphyria cutanea tarda symptomatica, two with a history of pesticide exposure, received daily treatment of 250 mg chloroquine for seven days. Liver function test results were altered immediately after therapy and porphyrin levels were very high. Light and electron microscopy of eight control liver biopsies showed fatty changes before treatment, extensive liver damage during treatment, and regeneration of the liver parenchyma after treatment. The authors stated that because of the transient but severe liver damage, this therapeutic modality is not recommended for children with porphyria cutanea tarda.
Serum gamma-glutamyl transferase was elevated fourfold in all thirty-four new cases of porphyria cutanea tarda. Phlebotomy treatment decreased it. Twelve patients in relapse showed a considerable increase, while those in remission tended to a gradual elevation over years. Gamma-glutamyl transferase was described as a useful auxiliary parameter, particularly in untreated cases. Two cases of porphyria cutanea tarda symptomatica associated with hepatic granulomata were reported, one with the melanodermic type. What remains missing is a modern controlled trial comparing chloroquine with phlebotomy or other treatments, systematic data on long-term relapse rates and liver safety in adults, and any evidence on patient stratification by liver histology or iron status.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
British Journal of Dermatology · 2006 · 81 citations
Treatment of porphyria cutanea tarda with chloroquine
AbstractTwenty men and one woman with porphyria cutanea tarda were treated with oral chloroquine, 125 mg twice weekly. In nineteen of the patients chloroquine was taken for 8–5 months on average before complete clinical and biochemical remission occurred. Initially, treatment led to a considerable increase in urinary uroporphyrin and a mild increase in serum transaminases, but no adverse clinical reactions were observed. As the mechanism of chloroquine action in porphyria cutanea tarda has not yet been clarified, special caution must be observed when it is used therapeutically.
British Journal of Dermatology · 1984 · 70 citations
Low-dose oral chloroquine in the treatment of porphyria cutanea tarda
AbstractSeven patients with porphyria cutanea tarda received a total of ten courses of low-dose oral chloroquine therapy (125 mg chloroquine phosphate twice weekly). Patients were treated for a mean 14.9 months during which time all went into clinical and biochemical remission. Relapse occurred in four patients on a total of six occasions after a mean 17 months. In four patients there was no relapse after a mean 47.3 months. There were no adverse side-effects from the treatment. Low-dose oral chloroquine therapy appears to be a safe, effective and convenient treatment for porphyria cutanea tarda, although relapses may occur requiring further therapy.
British Journal of Dermatology · 1980 · 10 citations
Clinical value of serum γ-glutamyl transferase estimation in porphyria cutanea tarda
AbstractSerum gamma-glutamyl transferase was elevated fourfold in all thirty-four new cases of porphyria cutanea tarda. Phlebotomy treatment decreased it. The twelve patients in relapse showed a considerable increase, while those in remission tended to a gradual elevation in the enzyme over the course of years. gamma-Glutamyl transferase is a useful auxiliary parameter in porphyria cutanea tarda particularly in untreated cases.
PORPHYRIA CUTANEA TARDA SYMPTOMATICA IN CHILDREN TREATED WITH CHLOROQUINE: REPORT OF FOUR CASES WITH CONTROL LIVER BIOPSIES
AbstractABSTRACT Four children with porphyria cutanea tarda symptomatica were reported. Two of them had a previous history of exposure to pesticides. All four received a daily treatment of 250 mg of chloroquine for seven days. Liver function test results were altered immediately after conclusion of the therapy and porphyrin levels were very high. A light and electron microscopic examination of eight control liver biopsies revealed fatty changes before treatment, extensive liver damage during treatment, and regeneration of the liver parenchyma after the treatment. In view of the transient, but severe, liver damage, this therapeutic modality is not recommended for the treatment of children with porphyria cutanea tarda.
Metabolic alkalinization in porphyria cutanea tarda
AbstractAt the Mayo Clinic, four patients with porphyria cutanea tarda were treated with metabolic alkalinization by orally administered sodium bicarbonate for ten weeks to nine months. Clinically, blistering and ulceration that had occurred after sun exposure and trauma were reduced or completely alleviated. Under the experimental conditions outlined, the skin in the light-exposed areas in two of the patients became more resistant to controlled blistering by a suction apparatus. These observations of clinical activity and porphyrin excretion indicate that systemic alkalinization may be beneficial in the treatment of porphyria cutanea tarda.
Australasian Annals of Medicine · 1963 · 1 citations
PORPHYRIA CUTANEA TARDA SYMPTOMATICA ASSOCIATED WITH HEPATIC GRANULOMATA
AbstractSUMMARY Two cases are described of the previously unreported association of porphyria cutanea tarda (P.C.T.) symptomatica and hepatic granulomata. One of the patients also had the melanodermic type of porphyria cutanea tarda (P.C.T.) symptomatica. Abnormalities of bromsulphalein and iron metabolism in porphyria cutanea tarda (P.C.T.) symptomatica are discussed.
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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