Rare & Orphan Lab · DeCure for X

DeCure for Fibrous dysplasia

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for fibrous dysplasia — 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 module3 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:0080031$DeCureRare

The disease map

Disease moduleFibrous dysplasia 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 fibrous 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

farnesyl diphosphate synthase (FDPS)FDPS 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 1-hydroxy-2-imidazo[1,2-a]pyridin-3-ylethane-1,1-diyldrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 2VF6 · 2.1 Å · ligand (1-HYDROXY-2-IMIDAZO[1,2-A]PYRIDIN-3-YLETHANE-1,1-DIYL)BIS(PHOSPHONIC ACID) (M0N). Experimental structure, not a prediction.

What the evidence adds up to

Fibrous dysplasia is a benign bone disorder of unknown cause in which normal bone is replaced by fibrous connective tissue containing abnormal bone. It occurs most commonly in the second decade of life, and in the craniofacial area nine out of ten patients present before the age of five. The condition has variable presentations that pose challenges in diagnosis and treatment; decisions are made case by case depending on symptoms, location, and possible complications. A 2025 case report of monostotic fibrous dysplasia of the mandible in a six-year-old child with severe bone pain and trismus describes prompt resolution of signs and symptoms after intravenous zoledronic acid.

Symptomatic lesions are treated with surgical resection, and cosmetic concerns are addressed by surgical contouring once lesions become dormant. A 2007 series from Bochum followed eight patients with fibrous dysplasia for six to twenty years and found that treatment can increase quality of life, but complete healing is rare and not the main target. The staged therapy concept includes conservative options, curative and palliative surgical methods, and combinations of both. For unresectable or recurrent lesions, bisphosphonate therapy is mentioned as a form of medical management.

No controlled trial data are reported in these abstracts. The 2007 series is small and uncontrolled, the 2025 case report is a single patient, and the other papers are reviews or case descriptions. There is no evidence of a drug that alters the natural history of the disease or produces durable remission in a defined population.

What is still missing is a prospective, controlled trial with sufficient sample size to test bisphosphonates or any other drug against placebo or standard surgical care, with predefined endpoints for pain, lesion size, and functional loss. Patient stratification by age, lesion site, and monostotic versus polyostotic disease has not been done in a systematic way. Funding for such a trial, and for longer follow-up beyond the few years reported, remains absent.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Journal of Clinical and Experimental Dentistry · 2011 · 4 citations · open access

Fibro-osseous lesion of maxilla. Report of two cases in a family with review of literature

AbstractFibrous dysplasia is a disturbance of bone metabolism that is classified as a benign fibro-osseous lesion. Fibrous connective tissue containing abnormal bone, replaces normal bone. The etiology of fibrous dysplasia is unknown. The radiographic appearance of the irregularly shaped trabeculae aids in the differential diagnosis. Occurring most commonly in the second decade of life, the lesions of fibrous dysplasia can be surgically recontoured for esthetic or functional purposes once they become dormant.

https://doi.org/10.4317/jced.3.e336
Laryngo-Rhino-Otologie · 2007 · 2 citations

Therapiekonzepte bei fibröser Dysplasie

AbstractBACKGROUND: The fibrous dysplasia is a rare disease of bone metabolism. Most common on the extremities, its appearance at the skull base is rare but of importance in clinical otorhinolaryngology. The patients suffer from problems such as cosmetical limitation, recurrent pain and in later phases from functional losses which result from bone dysplasia. METHODS: Based on the experiences of these cases and the study of the current literature we would like to suggest a stage dependent therapy concept that includes conservativ options as well as surgical methods which are divided into curative and palliative surgical treatments. PATIENTS: The Department of Otorhinolaryngology in Bochum has been involved in the therapy of eight patients suffering from fibrous dysplasia which have been treated and examined from six to twenty years. RESULTS: Treatment in fibrous dysplasia can be efficient in increasing quality of life. A complete healing is rare and not the main target of treatment. CONCLUSIONS: The staged therapy of fibrous dysplasia consists of conservative and surgical treatment and the combination of both. The decision for an individual therapy concept might be difficult and often requires close interdisciplinary cooperation.

https://doi.org/10.1055/s-2007-966513
Cureus · 2024 · 1 citations · open access

An Unusual Case of Fibrous Dysplasia, Temporomandibular Joint Ankylosis, and Eagle’s Syndrome

AbstractFibrous dysplasia is a benign bone disease in children and young adults. This is characterized by the replacement of normal bone with fibrous tissue along with immature woven bone. Fibrous dysplasia is a rare disorder and has variable presentations that pose challenges in diagnosis and treatment. Decisions are made on a case-by-case basis, depending on the symptoms, location, or possible complications. Symptomatic lesions are treated with surgical resection. cosmetic concerns of the patients are taken care of by surgical contouring. For any unresectable or recurrent lesion, bisphosphonate therapy can be used as a form of medical management.

https://doi.org/10.7759/cureus.68172
Journal of Craniofacial Surgery · 2025 · 0 citations

Bisphosphonate Therapy for Fibrous Dysplasia of The Jaw in Children

AbstractFibrous dysplasia (FD) is an uncommon genetic disorder in which bone is replaced by immature bony and fibrous tissue, manifesting as slow-growing lesions. The craniofacial area is the most common site of fibrous dysplasia, and 9 out of 10 patients with fibrous dysplasia affecting the craniofacial bones present before the age of 5. This case of monostotic fibrous dysplasia of the mandible in a 06 year old child presented with severe bone pain and trismus. This case report emphasises on medical management of fibrous dysplasia with intravenous zoledronic acid resulting in prompt resolution of sign and symptoms.

https://doi.org/10.1097/scs.0000000000012037

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.