Molecular basis for treatment of gastrointestinal stromal tumours

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9 Scopus citations


Gastrointestinal stromal tumour (GIST) is a rare turnour of the gastrointestinal tract that arises from activating mutations in KIT or platelet-derived growth factor receptor α (PDGFRα). Imatinib, a molecularly targeted therapy that inhibits the kinase activity of KIT, PDGFRs, ABL, and BCR-ABL, has been shown to be highly efficacious in patients with advanced GIST Most patients with advanced GIST treated with imatinib achieve either a partial response or experience stable disease, and median survival is longer than 3 years in either case. There is a strong correlation between the type of KIT or PDGFRA mutation and the clinical response to imatinib in patients with advanced GIST. In a phase II trial, significantly higher partial response rates were achieved in patients with GISTs harbouring KIT exon 11 mutations than in patients with GISTs harbouring KIT exon 9 mutations or in patients with GISTs exhibiting no detectable KIT or PDGFRA mutations. Resistance to imatinib treatment is a clinical challenge in the management of patients with advanced GIST Many imatinib-resistant GISTs have an associated secondary kinase mutation of KIT or PDGFRA. Continuing research efforts are directed at optimising the use of imatinib in patients with advanced GIST as well as the development of novel treatment approaches to prevent and/or treat imatinib-resistant clones of GIST.

Original languageEnglish (US)
Pages (from-to)10-18
Number of pages9
JournalEuropean Journal of Cancer, Supplement
Issue number3
StatePublished - Mar 2006


  • Gastrointestinal stromal tumour
  • KIT
  • PDGFRα.
  • activating mutations
  • clinical response resistance
  • imatinib
  • management strategies
  • molecular mechanisms

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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