Phase II study of daily oral miltefosine (hexadecylphosphocholine) in advanced colorectal cancer.

34 patients with metastatic colorectal cancer were treated with the ether lipid miltefosine (hexadecylphosphocholine). Most patients received 3 x 50 mg daily, while in 11 patients the dose could be escalated to 4 x 50 mg daily. Nausea and vomiting were the most frequent side-effects occurring in all but 3 patients, nephrotoxicity was observed in 11 patients. Leucocytosis was observed in 24 and thrombocytosis in 17 patients. 28 patients are evaluable for response. 1 patient obtained a partial response of liver metastases for a duration of 8 months. 3 patients had stable disease while 24 progressed during treatment. We conclude that miltefosine in this dose and schedule has limited activity in colorectal cancer.