DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for erythropoietic protoporphyria — 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 moduleErythropoietic protoporphyria 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 erythropoietic protoporphyria 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
ferrochelatase (FECH) — FECH 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 inorganicdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7CTC · 2.0 Å · ligand FE2/S2 (INORGANIC) CLUSTER (FES). Experimental structure, not a prediction.
What the evidence adds up to
Erythropoietic protoporphyria (EPP) was first defined in 1961. The basic defect is an inborn error of metabolism leading to overproduction of protoporphyrin IX by the bone marrow, not in response to a failure of heme synthesis. A method for evaluating in vitro synthesis of protoporphyrin and heme by human bone marrow was described in 1963. By 2002, considerable progress had been made in understanding the genetics and pathogenesis of EPP through molecular investigation, but progress in therapy had been slower and was dogged by difficulty in assessing treatment efficacy in patients. A 2000 review noted therapeutic options but gave no efficacy data.
A 2010 article discussed clinical implications of advances in understanding the inheritance of EPP but provided no trial results. As of 2023, a new active substance called dersimelagon (MT-7117) is being tested in a phase III placebo-controlled randomised controlled trial in the US and Europe. The authors of a 2023 analysis argue that because an approved treatment already exists for EPP, the use of a placebo arm is ethically questionable. They suggest a noninferiority active-control trial without placebo as a more ethically and scientifically valid design for testing dersimelagon and other EPP treatments.
No concrete numbers on survival, response rates, or sample sizes are reported in any of these abstracts. No drug other than dersimelagon is mentioned. What remains missing is any published evidence of clinical efficacy from controlled trials, a trial design that avoids placebo when an approved treatment exists, and patient stratification strategies that might clarify which individuals benefit.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Blood · 1963 · 21 citations · open access
Congenital Erythropoietic Protoporphyria
AbstractAbstract Investigations into the pathophysiology of erythropoietic protoporphyria suggest that the basic defect is an inborn error of metabolism resulting in the overproduction of protoporphyrin IX by the bone marrow not in response to a failure of heme synthesis. A method is described for evaluating the in vitro synthesis of protoporphyrin and heme by human bone marrow.
Erythropoietic protoporphyria (EPP) at 40. Where are we now?
AbstractSince Professor Magnus first defined erythropoietic protoporphyria (EPP) in 1961, there has been considerable progress in the understanding this disease. The past decade has been a period of spectacular progress in understanding the genetics and pathogenesis of the disease by molecular investigation. However, progress in therapy for EPP has been slower, and has been dogged by difficulty in assessing treatment efficacy in patients. We are now entering an era in which advances in molecular genetics are directly affecting patient management. This review summarises laboratory and clinical progress in EPP in the past 40 years, and assesses the potential impact of molecular biology on clinical practice.
British Journal of Dermatology · 2000 · 9 citations
Therapeutic options for erythropoietic protoporphyria
AbstractJournal Article Therapeutic options for erythropoietic protoporphyria Get access D.J. Todd D.J. Todd Department of Dermatology, Faculty of Medicine, Jordan University of Science & Technology, Irbid, PO Box 3030, Jordan E‐mail: [email protected] Search for other works by this author on: Oxford Academic Google Scholar British Journal of Dermatology, Volume 142, Issue 4, 1 April 2000, Page 826, https://doi.org/10.1046/j.1365-2133.2000.03442.x Published: 01 April 2000
British Journal of Dermatology · 2010 · 4 citations
Clinical implications of advances in understanding the inheritance of erythropoietic protoporphyria
AbstractJournal Article Clinical implications of advances in understanding the inheritance of erythropoietic protoporphyria Get access R. Sarkany R. Sarkany St John’s Institute of Dermatology, St Thomas’ Hospital, London SE1 7EH, U.K. E‐mail: [email protected] Search for other works by this author on: Oxford Academic Google Scholar British Journal of Dermatology, Volume 162, Issue 5, 1 May 2010, Pages 1156–1157, https://doi.org/10.1111/j.1365-2133.2010.09747.x Published: 01 May 2010
Orphanet Journal of Rare Diseases · 2023 · 2 citations · open access
Current trials in erythropoietic protoporphyria: are placebo controls ethical?
AbstractA new active substance called "dersimelagon" (MT-7117) is being tested as an alternative treatment option for Erythropoietic protoporphyria (EPP). At the moment, dersimelagon is being tested both in the US and in Europe in a phase III placebo-controlled RCT. However, given the availability of an already approved treatment option for EPP the use of a placebo arm is questionable from an ethics point of view. We analyze the issue and suggest that a noninferiority active-control trial without placebo is an ethically and scientifically more valid design to test the efficacy of dersimelagon as well as other EPP treatments.
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.
