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Updated: Jun 9, 2026

An Orthotopic Sciatic Nerve Xenograft for Neurofibromatosis Type 1 Neurofibromas
Published on: October 10, 2025
Neurofibromatosis type 1-associated wild-type gastrointestinal stromal tumor treated with anti-IGF-1R monoclonal
F L Day1, J Desai, C L Corless
1Department of Medical Oncology, Peter MacCallum Cancer Centre, Locked Bag 1, A'Beckett St, Melbourne, VIC 8006, Australia. fday@med.usyd.edu.au.
Abstract:
Gastrointestinal stromal tumors lacking mutations in KIT or PDGFRα are known as wild type (WT) and are less responsive to imatinib. These WT tumors are hypothesized to be dependent on signaling through the insulin-like growth factor 1 receptor (IGF-1R). We report the case of a 29-year-old woman with neurofibromatosis type 1-associated WT GIST treated with an anti-IGF-1R monoclonal antibody. Treatment was ineffective, and the potential basis for lack of response is discussed in the context of IGF-1R expression levels measured within this patients' primary tumor. We suggest that future clinical trials of anti-IGF-1R therapies prospectively determine tumor IGF-1R expression levels for correlation with response to treatment.
Insights
Wild type gastrointestinal stromal tumors (GIST) resistant to imatinib were treated with an anti-insulin-like growth factor 1 receptor (IGF-1R) antibody. The treatment was ineffective, suggesting IGF-1R expression levels are crucial for therapy success.
Area of Science:
- Oncology
- Molecular Biology
- Gastroenterology
Background:
- Wild type (WT) gastrointestinal stromal tumors (GIST) lack KIT or PDGFRα mutations, rendering them less responsive to imatinib therapy.
- These WT GISTs are hypothesized to rely on signaling pathways involving the insulin-like growth factor 1 receptor (IGF-1R).
Observation:
- A case study of a 29-year-old woman with neurofibromatosis type 1-associated WT GIST is presented.
- The patient received treatment with a monoclonal antibody targeting IGF-1R.
Findings:
- The anti-IGF-1R antibody treatment proved ineffective in this patient.
- Tumor IGF-1R expression levels were measured and discussed as a potential factor influencing treatment response.
Implications:
- This case suggests that IGF-1R expression levels may be a critical determinant of treatment efficacy for WT GIST.
- Future clinical trials should prospectively assess tumor IGF-1R expression to correlate with outcomes in anti-IGF-1R therapies.
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