DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for neurofibroma — screening already-approved drugs against its 19-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleNeurofibroma maps to a 19-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 neurofibroma 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
neurotrophic receptor tyrosine kinase 3 (NTRK3) — NTRK3 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 4-aminophenyldrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6KZD · 1.708 Å · ligand 3-[2-[6-(4-aminophenyl)imidazo[1,2-a]pyrazin-3-yl]ethynyl]-2-methyl-~{N}-[3-(4-methylpiperazin-1-yl)-5-propan-2-yl-phenyl]benzamide (DZ6). Experimental structure, not a prediction.
What the evidence adds up to
A 2025 comprehensive review of published literature describes selumetinib as a targeted therapy for inoperable plexiform neurofibroma in neurofibromatosis type 1 patients, a condition that arises in about 50% of NF1 patients and carries a risk of malignant transformation. The review does not provide response rates, survival data, or sample sizes from the studies it covers, and it does not compare selumetinib to placebo or any other treatment. No other drug is mentioned in any of the abstracts.
Solitary neurofibromas not associated with neurofibromatosis are rare. A 2010 case report describes a woman with a solitary neurofibroma of the temporal and infratemporal regions who underwent complete surgical excision and had no recurrence at two-year follow-up. A 2020 case report of a solitary intraosseous neurofibroma of the mandible in a 32-year-old woman notes the histopathologic feature as unique but provides no follow-up data. A 2020 case of a neurofibroma in the ethmoid and sphenoid sinus causing unilateral nasal obstruction is presented as a rare differential diagnosis, but again no treatment outcome or recurrence data are given.
A 2015 case report describes a 46-year-old man with NF1 who had a large neurofibroma of the upper back that had been partially removed at ages 20 and 36 but regrew continuously, reaching 258 × 43 × 159 mm by MRI. The lesion became pruritic and painful, and the overlying skin was raised, hairy, and hyperpigmented. The report does not state whether the subsequent surgical resection was complete or whether recurrence followed.
What is still missing: for selumetinib, the review does not provide quantitative efficacy data such as tumour shrinkage rates, progression-free survival, or quality-of-life measures, and no randomised controlled trial is cited. For all neurofibromas, there are no large prospective studies comparing surgical excision, observation, or drug therapy, and no validated biomarkers to predict which solitary lesions will recur or which plexiform tumours will undergo malignant transformation. Funding for such trials and for patient stratification by NF1 mutation type or tumour location 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.
Indian Journal of Pathology and Microbiology · 2020 · 17 citations · open access
Solitary intraosseous neurofibroma of the mandible: Report of an extremely rare histopathologic feature
AbstractNeurofibroma (NF) is a benign tumor derived from the peripheral nerve sheath. Neurofibromas may present either as solitary lesions or as part of the generalized syndrome of neurofibromatosis or von Recklinghausen's disease of the skin. The intraosseous variant of NF is very rare. We report a case of a 32-year-old female who was diagnosed with a solitary intraosseous neurofibroma of the mandible. The present case is rare with respect to its unique histopathologic feature.
Journal of Craniofacial Surgery · 2010 · 7 citations
Solitary Neurofibroma of the Temporal Bone
AbstractNeurofibroma is a benign peripheral nerve sheath tumor that can be occasionally found in the head and neck region as multiple lesions associated with neurofibromatosis type 1 (NF-1) or as a solitary tumor. The real frequency of isolated neurofibromas not associated with NF is uncertain, and lesions in the temporal region are extremely rare. The aim of the current article was to report an unusual case of solitary neurofibroma localized in the temporal and infratemporal regions with 10 years of evolution in a female patient without any other manifestation or familiar history of NF-1. The patient underwent surgical treatment for complete excision of the lesion, and the 2-year follow-up revealed no signs of recurrence.
Selumetinib use as targeted therapy for plexiform neurofibroma: a comprehensive review of the literature
AbstractNeurofibromatosis type 1 is inherited in an autosomal dominant fashion. The approximate prevalence of neurofibromatosis type 1 is 1:2500 to 1:3500 regardless of ethnic group. Around 50% of neurofibromatosis type 1 patients develop plexiform neurofibroma, a lesion that has a tendency for malignant transformation. Understanding the pathophysiology of plexiform neurofibromatosis and the implication of neurofibromin signal transduction has led to the development and use of targeted therapy on this pathway. In this article, we comprehensively reviewed the recently published literature on the use of selumetinib to treat inoperable plexiform neurofibroma in neurofibromatosis type 1 patients.
European Journal of Dermatology · 2015 · 1 citations
Do juxtaposed compound nevus and neurofibroma with melanocytic differentiation share an identical cell of origin in NF1?
AbstractWe report a 46-year-old man fulfilling the diagnostic criteria of Neurofibromatosis type 1 (NF1) who was admitted for surgical resection of a large neurofibroma at the upper back. At ages 20 and 36 years, a neurofibroma was partially removed from the same region but the lesion grew again continuously and became pruritic and painful. The tumor measured 258 × 43 × 159 mm by MRI. Macroscopically, the overlying skin was raised, hairy and hyperpigmented (figure 1). The tumor showed a pale-grey [...]
Laryngo-Rhino-Otologie · 2020 · 0 citations · open access
Case Report: Neurofibroma of the ethmoid and sphenoid sinus - a rare differential diagnosis of unilateral nasal obstruction
AbstractIntroduction Neurofibroma is a benign tumor, which arises from the connective tissue of peripheral nerves. The most frequent locations are the extremities and the head and neck area. In only about 4 % of the cases the tumor presents in the nasal cavity and paranasal sinuses. The tumor is often seen in patients with neurofibromatosis (NF1).
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.
