DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for fibromuscular dysplasia — 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 moduleFibromuscular dysplasia 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 fibromuscular dysplasia 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
phosphatase and actin regulator 1 (PHACTR1) — PHACTR1 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 16pdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6ZEE · 1.9 Å · ligand 3,6,9,12,15,18-HEXAOXAICOSANE (16P). Experimental structure, not a prediction.
What the evidence adds up to
The two consensus documents from 2019 describe fibromuscular dysplasia as a disease of small- and medium-sized arteries causing stenosis, dissections, and aneurysms, typically in the renal, carotid, and vertebral arteries, with possible involvement of the iliac, femoral, brachial, mesenteric, and coronary arteries. These are position statements and supplemental tables; they contain no trial data, no drug efficacy figures, and no survival or response-rate numbers. The editorial from the same year, from a Spanish-language journal, mentions the first national registry of fibromuscular dysplasia initiated five years prior, but provides no results from that registry. The same editorial discusses neurofibromatosis, depression, pembrolizumab for lung cancer, lingual thyroid, teleconsultations, and antiglaucoma drugs; none of these topics relate to fibromuscular dysplasia treatment, and no drug is proposed for the disease in any of the three abstracts.
No abstract reports a pharmacological intervention tested in fibromuscular dysplasia. There is no mention of any repurposed agent, no randomised controlled trial, no cohort with outcome data, and no biomarker validated for diagnosis or prognosis. The consensus material points to emerging biomarkers and future directions, but the abstracts do not specify which biomarkers or what directions, and they do not quantify any clinical benefit. The editorial's registry is described only as initiated, with no patient numbers, follow-up duration, or endpoint results provided.
The available evidence is therefore entirely descriptive and consensus-based. It establishes the disease's vascular distribution and the existence of a national registry, but it offers nothing on drug efficacy, safety, or patient stratification. What is missing is any prospective trial of a candidate therapy, any biomarker validation with sensitivity and specificity figures, and any registry data reporting outcomes such as stenosis progression, dissection rates, or aneurysm rupture. Funding for such a trial, a design that accounts for the disease's rarity and heterogeneous arterial involvement, and a strategy to stratify patients by genetic or imaging phenotype are all absent from these abstracts.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Sage Journals Data · 2019 · 0 citations · open access
Supplemental_Emerging_biomarkers_and_future_directions_final – Supplemental material for First International Consensus on the diagnosis and management of fibromuscular dysplasia
AbstractSupplemental material, Supplemental_Emerging_biomarkers_and_future_directions_final for First International Consensus on the diagnosis and management of fibromuscular dysplasia by Heather L Gornik (Co-Chair), Alexandre Persu (Co-Chair), David Adlam, Lucas S Aparicio, Michel Azizi, Marion Boulanger, Rosa Maria Bruno, Peter de Leeuw, Natalia Fendrikova-Mahlay, James Froehlich, Santhi K Ganesh, Bruce H Gray, Cathlin Jamison, Andrzej Januszewicz, Xavier Jeunemaitre, Daniella Kadian-Dodov, Esther SH Kim, Jason C Kovacic, Pamela Mace, Alberto Morganti, Aditya Sharma, Andrew M Southerland, Emmanuel Touzé, Patricia van der Niepen, Jiguang Wang, Ido Weinberg, Scott Wilson, Jeffrey W Olin and Pierre-Francois Plouin in Vascular Medicine
INDIGO (University of Illinois at Chicago) · 2019 · 0 citations · open access
Supplemental_Table_1_-_rev – Supplemental material for First International Consensus on the diagnosis and management of fibromuscular dysplasia
AbstractSupplemental material, Supplemental_Table_1_-_rev for First International Consensus on the diagnosis and management of fibromuscular dysplasia by Heather L Gornik (Co-Chair), Alexandre Persu (Co-Chair), David Adlam, Lucas S Aparicio, Michel Azizi, Marion Boulanger, Rosa Maria Bruno, Peter de Leeuw, Natalia Fendrikova-Mahlay, James Froehlich, Santhi K Ganesh, Bruce H Gray, Cathlin Jamison, Andrzej Januszewicz, Xavier Jeunemaitre, Daniella Kadian-Dodov, Esther SH Kim, Jason C Kovacic, Pamela Mace, Alberto Morganti, Aditya Sharma, Andrew M Southerland, Emmanuel Touzé, Patricia van der Niepen, Jiguang Wang, Ido Weinberg, Scott Wilson, Jeffrey W Olin and Pierre-Francois Plouin in Vascular Medicine
Revista del Hospital Italiano de Buenos Aires · 2019 · 0 citations · open access
Editorial de la revista número 4 del año 2019
AbstractFibromuscular dysplasia is a disease of small- and medium-sized arteries that can cause stenosis, dissections, and aneurysms. It typically affects the renal, carotid, and vertebral arteries, but it can also involve the iliac, femoral, brachial, mesenteric, and coronary arteries. Lucas Aparicio, from the Arterial Hypertension Section of the Department of Internal Medicine, presents the first national registry of fibromuscular dysplasia, initiated five years ago. Neurofibromatosis is a genetic disorder in which tumors form in any part of the nervous system, including the brain, spinal cord, and nerves. María Florencia Correa and Natalia Inés Pasik, from the Department of Internal Medicine, review the epidemiology, molecular aspects, clinical expression, and current management of neurofibromatosis types 1 and 2. Eduardo G. Giacomantone, from the Department of Internal Medicine, reviews the different clinical presentations of depression in general medicine and its treatment. In the Medical Education section, Yasmín Dana Moauro and collaborators from the University Institute of Hospital Italiano evaluated the training of surgical technologists at different universities. PD-1 is a negative regulator of T-cell activity. Its inhibition with monoclonal antibodies activates the antitumor response but may be associated with autoimmune complications. María Paz Micieli Galeazzi and collaborators from the Departments of Dermatology and Allergy and Immunology present the case of a patient treated with pembrolizumab for lung cancer who developed hypothyroidism. Lingual thyroid is an anomaly of thyroid development resulting from failure of the thyroid gland to descend to its prelaryngeal location. Carlos Santiago Ruggeri, Lautaro Acosta, and Ivo Bedini, from the Head and Neck Unit of the Department of Otolaryngology, report the case of a patient with thyroid tissue at the base of the tongue resected via a transoral approach. In the Hospital Italiano in Medline section, two publications are discussed. Santiago Andrés Frid and María Florencia Grande Ratti, from the Department of Health Informatics and the Internal Medicine Research Area, evaluated the implementation of the Virtual Care Program for unscheduled teleconsultations developed in 2018. Agustina Galmarini, from the Department of Ophthalmology, investigated whether antiglaucoma drugs are a factor contributing to lacrimal pathway obstruction.
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.
