DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for fibrodysplasia ossificans progressiva — screening already-approved drugs against its 6-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleFibrodysplasia ossificans progressiva maps to a 6-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 fibrodysplasia ossificans progressiva 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
matrix metallopeptidase 9 (MMP9) — MMP9 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 3-azanyl-4-fluoranyl-5,7,8,9-tetrahydropyrido[4,3-c]azepin-6-yldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 8K5Y · 1.52 Å · ligand (3-azanyl-4-fluoranyl-5,7,8,9-tetrahydropyrido[4,3-c]azepin-6-yl)-[6-(2-oxidanylpropan-2-yl)-1H-indol-2-yl]methanone (VP6). Experimental structure, not a prediction.
What the evidence adds up to
Fibrodysplasia ossificans progressiva is an ultra-rare progressive genetic disease affecting about one in a million individuals. A mutation in the ACVR1 gene was identified as the causative mutation in 2006. The disease is characterised by heterotopic ossifications in muscles, fascia, and tendons, along with congenital and skeletal deformities that form during life. Patients progressively develop bone in soft tissues, resulting in increasing immobility and early death. Around 200 patients with the condition had been reported in world literature as of 2007. A 2024 clinical observation described a patient followed from age 1 year and 3 months to 29 years; care and symptomatic treatment during that period could not prevent regular appearance of new heterotopic ossifications, which led to severe functional disorders and loss of ability to self-care.
Radiologic findings from seven Iranian patients admitted to paediatric wards between 1983 and 2002 were analysed in a 2007 report. The 2024 case report emphasises that unjustified surgical interventions carry risks of increasing the severity of the disease course and functional disorders. A 2022 editorial notes that much effort is being devoted to searching for a cure, and a 2021 workshop update states that after the 2006 identification of the ACVR1 mutation, the pathophysiology has been further elucidated by research groups worldwide.
The 2024 review states that scientific studies in recent years have enabled development of effective pharmacotherapy, which provides hope for preventing progression and improving quality of life and social adaptation. However, the same report notes unresolved challenges in monitoring the disease, predicting its course and complications, and a lack of generally accepted effective treatment. The 2021 workshop update does not report any specific trial results, response rates, or survival data from any drug intervention.
What is still missing are completed, publicly reported randomised controlled trials with survival or functional endpoints, a standardised monitoring protocol that can predict disease flares, and any validated method for stratifying patients by disease activity or genetic modifier status. No drug is mentioned by name in any of 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.
Frontiers in Endocrinology · 2021 · 26 citations · open access
Fibrodysplasia Ossificans Progressiva: What Have We Achieved and Where Are We Now? Follow-up to the 2015 Lorentz Workshop
AbstractFibrodysplasia ossificans progressiva (FOP) is an ultra-rare progressive genetic disease effecting one in a million individuals. During their life, patients with FOP progressively develop bone in the soft tissues resulting in increasing immobility and early death. A mutation in the ACVR1 gene was identified as the causative mutation of FOP in 2006. After this, the pathophysiology of FOP has been further elucidated through the efforts of research groups worldwide. In 2015, a workshop was held to gather these groups and discuss the new challenges in FOP research. Here we present an overview and update on these topics.
Radiologic findings in seven patients with fibrodysplasia ossificans progressiva.
AbstractFibrodysplasia ossificans progressiva is a rare and disabling syndrome, which is characterized by heterotopic ossifications and skeletal deformities. So far, around 200 patients with fibrodysplasia ossificans progressiva have been reported in the world literature. Herein, we analyze the clinical records of 7 known cases of fibrodysplasia ossificans progressiva from Iran who were admitted to the pediatrics wards of our centers between 1983 and 2002, and present the radiologic findings.
Editorial of Special Issue “Fibrodysplasia Ossificans Progressiva: Studies on Disease Mechanism towards Novel Therapeutic Approaches”
AbstractThe Special Issue on “Fibrodysplasia Ossificans Progressiva: Studies on Disease Mechanism towards Novel Therapeutic Approaches” has published interesting and useful review articles and original experimental articles on fibrodysplasia ossificans progressiva (FOP), a very rare genetic disorder for which much effort is being devoted to search for a cure. In this editorial, I briefly cite the essential content of all the published articles.
N N Priorov Journal of Traumatology and Orthopedics · 2024 · 1 citations · open access
Fibrodysplasia ossificans progressiva (clinical observation with a brief review of the literature)
AbstractBACKGROUND: Fibrodysplasia ossificans progressiva is a rare genetically determined disease of the musculoskeletal system and characterized by heterotopic ossifications in the muscles, fascia, and tendons and congenital and skeletal deformities that form during life. Owing to the lack of awareness of doctors, unresolved challenges in monitoring the disease and predicting the course and development of its complications, and the lack of generally accepted effective treatment, fibrodysplasia ossificans progressiva leads to severe disability and social disadaptation, limiting the life expectancy of patients. CLINICAL CASE DESCRIPTION: The characteristic anamnestic data of a patient with fibrodysplasia ossificans progressiva are presented. The course of the disease from the moment of detection at age 1 year and 3 months to 29 years was determined. Notably, the care and symptomatic treatment performed during this period could not prevent the regular appearance of new heterotopic ossifications, which led to severe functional disorders and loss of the patient’s ability to self-care. In a brief review, the current possibilities of pathogenetic therapy for this disease and prevention of progression and complications were considered. The risks of unjustified surgical interventions leading to increased severity of the course and functional disorders are emphasized. CONCLUSION: The scientific studies conducted in recent years to examine the etiopathogenesis of fibrodysplasia ossificans progressiva enabled the development of effective pharmacotherapy, which provides hope for the possibility of preventing the progression of the disease and improving the quality of life and social adaptation of patients with fibrodysplasia ossificans progressiva.
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.
