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.

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.