Abstract
The largest phase 2 study done to date in patients with platelet-derived growth factor β (PDGFβ)-positive advanced chordoma treated with imatinib (800 mg daily), failed to elicit an overall tumour response defined by RECIST. However, at 6 months, 70% of patients remained stable during treatment, 64% showed a clinical benefit, and 18% showed some reduction in tumour size. “Although the responses were not dramatic, nearly two-thirds of chordoma patients benefited from imatinib treatment. For a disease with few other treatment options and no approved drugs, these results are significant”, comments Josh Sommer, from the Chordoma Foundation (Durham, NC, USA).
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