DeCure for Malignant peripheral nerve sheath tumor
DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for malignant peripheral nerve sheath tumor — screening already-approved drugs against its 42-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMalignant peripheral nerve sheath tumor maps to a 42-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 malignant peripheral nerve sheath tumor 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
galectin 1 (LGALS1) — LGALS1 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 acetylaminodrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 4Q27 · 1.2 Å · ligand prop-2-en-1-yl 2-(acetylamino)-2-deoxy-beta-D-glucopyranoside (TVS). Experimental structure, not a prediction.
What the evidence adds up to
Malignant peripheral nerve sheath tumours (MPNST) are rare and often occur in patients with neurofibromatosis 1. Surgical resection is the mainstay of treatment. Complete tumour resection is advocated, but because major nerves may need to be sacrificed to achieve a tumour-free margin, and because patients are often reluctant to undergo amputation, subtotal resection with adjuvant radiotherapy is described as the best alternative for treating this aggressive disease. In one reported case of a 30-year-old African male with neurofibromatosis type I and an MPNST involving the sciatic nerve, subtotal resection with preservation of the sciatic nerve resulted in a good neurological outcome. Radiation and chemotherapy have a role in selected patients.
Accurate pathologic diagnosis remains a challenge in many cases of MPNST. The tumours represent a spectrum from benign schwannoma and neurofibroma to high-grade malignancy, and the different benign variants have a propensity for malignant transformation. Recent advances in understanding the molecular pathogenesis of MPNST are described as the best opportunities to develop new management strategies. These molecular biology advances have provided new insights into the nature of peripheral nerve sheath tumours and suggest potential novel targeted therapeutic strategies.
No clinical trial data, response rates, or survival numbers are reported in these abstracts. The evidence is limited to case reports and narrative reviews. The abstracts do not report any drug showing efficacy in MPNST. What is still missing are prospective clinical trials, validated biomarkers for early diagnosis and patient stratification, and funding to translate molecular insights into therapies that have been tested in patients.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
The Oncologist · 2014 · 367 citations · open access
Malignant Peripheral Nerve Sheath Tumors
AbstractMalignant peripheral nerve sheath tumors (MPNST) are uncommon, biologically aggressive soft tissue sarcomas of neural origin that pose tremendous challenges to effective therapy. In 50% of cases, they occur in the context of neurofibromatosis type I, characterized by loss of function mutations to the tumor suppressor neurofibromin; the remainder arise sporadically or following radiation therapy. Prognosis is generally poor, with high rates of relapse following multimodality therapy in early disease, low response rates to cytotoxic chemotherapy in advanced disease, and propensity for rapid disease progression and high mortality. The last few years have seen an explosion in data surrounding the potential molecular drivers and targets for therapy above and beyond neurofibromin loss. These data span multiple nodes at various levels of cellular control, including major signal transduction pathways, angiogenesis, apoptosis, mitosis, and epigenetics. These include classical cancer-driving genetic aberrations such as TP53 and phosphatase and tensin homolog (PTEN) loss of function, and upregulation of mitogen-activated protein kinase (MAPK) and (mechanistic) target of rapamycin (TOR) pathways, as well as less ubiquitous molecular abnormalities involving inhibitors of apoptosis proteins, aurora kinases, and the Wingless/int (Wnt) signaling pathway. We review the current understanding of MPNST biology, current best practices of management, and recent research developments in this disease, with a view to informing future advancements in patient care.
Journal of Surgical Oncology · 2008 · 218 citations
Malignant peripheral nerve sheath tumor: Molecular pathogenesis and current management considerations
AbstractMalignant Peripheral Nerve Sheath Tumors (MPNSTs) are rare tumors that often occur in patients with neurofibromatosis 1. Surgical resection represents the mainstay of treatment. Radiation and chemotherapy have a role in selected patients with MPNST. Accurate pathologic diagnosis remains a challenge in many cases of MPNST. There are many recent advances in the understanding of the molecular pathogenesis of MPNST which represent the best opportunities to develop new strategies for management of patients with MPNST.
World Journal of Surgical Oncology · 2023 · 7 citations · open access
Surgical management of craniospinal axis malignant peripheral nerve sheath tumors: a single-institution experience and literature review
AbstractBACKGROUND: Malignant peripheral nerve sheath tumor (MPNST) is an exceedingly rare and aggressive tumor, with limited literature on its management. Herein, we present our series of surgically managed craniospinal MPNSTs, analyze their outcomes, and review the literature. METHODS: We retrospectively reviewed surgically managed primary craniospinal MPNSTs treated at our institution between January 2005 and May 2023. Patient demographics, tumor features, and treatment outcomes were assessed. Neurological function was quantified using the Frankel grade and Karnofsky performance scores. Descriptive statistics, rank-sum tests, and Kaplan-Meier survival analyses were performed. RESULTS: Eight patients satisfied the inclusion criteria (4 male, 4 female). The median age at presentation was 38 years (range 15-67). Most tumors were localized to the spine (75%), and 3 patients had neurofibromatosis type 1. The most common presenting symptoms were paresthesia (50%) and visual changes (13%). The median tumor size was 3 cm, and most tumors were oval-shaped (50%) with well-defined borders (75%). Six tumors were high grade (75%), and gross total resection was achieved in 5 patients, with subtotal resection in the remaining 3 patients. Postoperative radiotherapy and chemotherapy were performed in 6 (75%) and 4 (50%) cases, respectively. Local recurrence occurred in 5 (63%) cases, and distant metastases occurred in 2 (25%). The median overall survival was 26.7 months. Five (63%) patients died due to recurrence. CONCLUSIONS: Primary craniospinal MPNSTs are rare and have an aggressive clinical course. Early diagnosis and treatment are essential for managing these tumors. In this single-center study with a small cohort, maximal resection, low-grade pathology, young age (< 30), and adjuvant radiotherapy were associated with improved survival.
BMJ Case Reports · 2015 · 2 citations · open access
Late and multifocal presentations of malignant peripheral nerve sheath tumours following radiotherapy
AbstractPeripheral nerve sheath tumours are a well-recognised complication of radiotherapy. Single tumours presenting up to 10 years after initial treatment have been previously described. We report an individual with two anatomically separate nerve sheath tumours developing at different time intervals following initial treatment, after 38 and 42 years, respectively. This case illustrates the importance of maintaining vigilance for the development of complications even at an advanced stage following index radiotherapy treatment.
Plastic & Reconstructive Surgery Global Open · 2018 · 2 citations · open access
Novel Treatment of a Malignant Peripheral Nerve Sheath Tumor of the Median Nerve
AbstractMalignant peripheral nerve sheath tumors are rare, associated with a poor prognosis and uncertainty regarding the appropriate management. We report a novel oncologic and reconstructive treatment of a young patient with a malignant peripheral nerve sheath tumor of the median nerve of the left hand. The patient underwent a wide local excision, an opponensplasty, a nerve reconstruction by nerve allografts followed by brachytherapy treatment. Two years later, the patient remains disease free with preserved function of her hand.
The Internet Journal of Surgery · 2012 · 0 citations
Malignant Peripheral Nerve Sheath Tumour In Neurofibromatosis Type 1
AbstractMalignant peripheral nerve sheath tumours (MPNST) are rare. We present a case of a 30-year-old African male patient with neurofibromatosis type I with a MPNST involving the sciatic nerve. A subtotal resection of the tumour was done with preservation of the sciatic nerve with good neurological outcome. Complete tumour resection is advocated. However, due to sacrifice of major nerves to achieve a tumour-free margin and the reluctance of patients to undergo amputation, subtotal resection and adjuvant radiotherapy provides the best alternative in treating this aggressive disease.
AbstractPeripheral nerve sheath tumors represent a spectrum of entities ranging from benign tumors, such as schwannoma, to high-grade malignant tumors termed malignant peripheral nerve sheath tumors. In this chapter, we discuss current concepts and problematic areas in the diagnosis, treatment, and management of peripheral nerve sheath tumors. We discuss the major categories of nerve sheath tumors including schwannomas, neurofibromas, and malignant peripheral nerve sheath tumors. The different benign variants and their propensity for malignant transformation are highlighted. We suggest useful guidelines for the surgical management of these challenging cases. Finally, we discuss some of the advances in molecular biology that have provided new insights into the nature of the various peripheral nerve sheath tumors and suggest potential novel targeted therapeutic strategies for these tumors. This review contains 11 figures, 5 tables and 66 references. Key Words: malignant peripheral nerve sheath tumors, nerve sheath tumor, neurofibroma, neurofibromatosis, schwannoma, schwannomatosis, NF1, NF2
AbstractPeripheral nerve sheath tumors represent a spectrum of entities ranging from benign tumors, such as schwannoma, to high-grade malignant tumors termed malignant peripheral nerve sheath tumors. In this chapter, we discuss current concepts and problematic areas in the diagnosis, treatment, and management of peripheral nerve sheath tumors. We discuss the major categories of nerve sheath tumors including schwannomas, neurofibromas, and malignant peripheral nerve sheath tumors. The different benign variants and their propensity for malignant transformation are highlighted. We suggest useful guidelines for the surgical management of these challenging cases. Finally, we discuss some of the advances in molecular biology that have provided new insights into the nature of the various peripheral nerve sheath tumors and suggest potential novel targeted therapeutic strategies for these tumors. This review contains 11 figures, 5 tables and 66 references. Key Words: malignant peripheral nerve sheath tumors, nerve sheath tumor, neurofibroma, neurofibromatosis, schwannoma, schwannomatosis, NF1, NF2
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.
