Background: Treatment results in advanced colorectal cancer (CC) remain unsatis-factory and palliative, with 5-fluorouracil with or without calcium folinate being the only drug able to induce clinically acceptable response rates.Patients and Methods: A clinical phase II study is presented with an oral formulationof the phospholipid derivative miltefosine (MIL) in patients with advanced colorec-tal cancer. Patients were stratified according to pretreatment. Only non-pretreatedor pretreated patients who had received 5-fluorouracil with or without calcium folinate were accepted. MIL was given as capsule twice daily at a single dose of 50 mg for the 1st week with dose escalation to 150 mg (50 mg × 3) in the 2nd week and subsequently in case of good tolerability. Nine weeks were considered the minimal duration of treatment.Results: 54 patients were evaluable for toxicity and 44 were evaluable for response. A short-lived partial response was observed in one pretreated female patient with multiple lung lesions, a ‘no change’ status in 14 patients, including 8 nonpretreated patients (42%) and 6 pretreated patients (24%). Side effects were distinct, with loss of appetite, nausea and vomiting up to grade 4 WHO and weight loss of more than 5 kg in 3 months in a considerable number of patients. Furthermore, an increase of leukocyte and platelet counts was observed during the first 2 months of treatment. Conclusion: Oral MIL is considerably toxic and has only marginal therapeutic activity in patients with colorectal cancer.
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