A pilot study was performed to evaluate the efficacy and safety of gemcitabine and cisplatin combination in the treatment of patients with metastatic nasopharyngeal carcinoma (NPC). Eligible patients were those with metastatic NPC who had been treated with radiotherapy and cisplatin plus 5-fluorouracil chemotherapy. Cisplatin was given intravenously at the fixed dose of 30 mg/m2 on days 1-3. Gemcitabine was intravenously administered over 30 min infusion with the dose escalated from 800 to 1200 mg/m2 on days 1 and 8. The 3-week schedule defined a cycle of treatment. Fifteen patients were enrolled and assessed for the worst toxicities. For a total of 83 cycles, Grade 3-4 toxicity was 46.7 % for neutropenia, 40.0 % for thrombocytopenia, and 20.0% for anemia. Grade 3 nonhematologic toxicity was 13.3%. Fourteen patients were assessable for response. The overall response rate was 92.9%, with complete response in three patients (21.4%). Median survival was 10.2 months. Seven patients had lived more than one year, and two patients had lived more than 2 years. The recommended dose of gemcitabine was 1000 mg/m2 on days 1 and 8 in each cycle. In conclusion, the present combination is well tolerated and highly active in the treatment of advanced NPC patients.