Objective: This was a phase II study of perillyl alcohol in the treatment of advanced ovarian cancer. The primary endpoint was to evaluate the 6-month progression-free rate of perillyl alcohol as compared with historic controls. Secondary objectives were to evaluate the objective response rate, time to progression and survival, dropout rate, and number of cycles administered; define the qualitative nature of acute and chronic toxicities; and evaluate the effect of perillyl alcohol on triglycerides and total, HDL, and LDL cholesterol levels. Methods. Women who had received prior platinum-based therapy and had residual or recurrent disease were eligible. Perillyl alcohol was administered orally, four times daily, at a dose of 1200 mg/m(2). This was repeated until disease progression or unacceptable toxicity was experienced.
Results: The 6-month progression-free rate was 17%. None of the patients achieved a complete or partial response. The median progression-free survival was 1.7 months. The median overall survival was 9.1 months. Compliance was greater than 90% but gastrointestinal toxicity (grade 1-2 nausea, satiety, eructation in 70%) and fatigue (grade 1-2 in 40%) were common and limited the ability to escalate the dose from 1200 to 1500 mg/m(2).
Conclusion: Perillyl alcohol administered at this dose and formulation did not exhibit signs of extending the time-to-progression in patients with advanced ovarian carcinoma.