Phase I Safety and Clinical Activity Study of Thymoquinone in Patients with Advanced Refractory Malignant Disease.

Purpose: This phase I study was conducted to determine the general toxicities of thymoquinone in humans, as well as any anti-cancer effects that the drug may have in patients with advanced cancer for which there were no standard curative or palliative measures.

Patients and methods: Adult patients with solid tumors or hematological malignancies who had failed or relapsed from standard therapy were included in this study. Patients who were at least 18 years of age with an Eastern cooperative oncology group performance status (ECOG) of ? 2 received thymoquinone orally at a starting dose level of 3, 7, or 10mg/kg/day. Dose escalation proceeded according to a modified Fibonacci design.

Results: All 21 patients received at least one week of treatment, with a median of 3.71 weeks (range 1 week to 20 weeks). No side effects were reported and the maximum tolerated dose (MDT) was not identified. No anti-cancer effects were observed.

Conclusion: On the basis of this study, thymoquinone was well tolerated at a dose ranging from 75mg/day to 2600mg/day. Neither toxicities nor therapeutic responses were reported.