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Imatinib mesylate in chordoma
Paolo G Casali1, Antonella Messina, Silvia Stacchiotti
1Adult Sarcoma Medical Oncology Unit, Department of Cancer Medicine, Istituto Nazionale per lo Studio e la Cura dei Tumori, Milan, Italy. paolo.casal@stitutotumori.mi.it
Cancer
|September 17, 2004
Summary
Imatinib mesylate shows antitumor activity in advanced chordoma patients by targeting platelet-derived growth factor receptor-beta (PDGFRB). Tumor response, including liquefaction, was observed, but long-term patient benefit requires further study.
Area of Science:
- Oncology
- Medical Therapy
- Molecular Targeted Therapy
Background:
- Advanced chordoma lacks effective medical therapies.
- Imatinib mesylate is a tyrosine kinase inhibitor targeting platelet-derived growth factor receptor-beta (PDGFRB), BCR-ABL, and KIT.
Purpose of the Study:
- To evaluate the efficacy of imatinib mesylate in patients with advanced chordoma.
- To investigate the potential role of PDGFRB in chordoma treatment response.
Main Methods:
- Six patients with advanced chordoma received imatinib mesylate (800 mg daily).
- Tumor PDGFRB expression and phosphorylation were assessed.
- Tumor response was monitored using CT, MRI, and PET scans.
Main Results:
- Tumor liquefaction observed on CT scans after treatment.
- Decreased contrast enhancement on MRI and reduced glucose uptake on PET scans noted.
- Subjective improvement reported in four of five symptomatic patients.
Conclusions:
- Imatinib mesylate demonstrates antitumor activity in chordoma, potentially via PDGFRB inactivation.
- Observed tumor response patterns resemble those in gastrointestinal stromal tumors but evolve more slowly.
- The clinical benefit of this response pattern in chordoma requires further elucidation, especially with significant local disease.