Phase II trial of edatrexate in small cell lung cancer

Background. Long-term survival with extensive stage small cell lung cancer is rare. There have been no major advances in the treatment of this stage of disease in the last 15–20 years. New agents with activity against this malignancy are needed. This study was designed to evaluate the efficacy of edatrexate against small cell lung cancer in a Phase II trial. Methods. This was a multicenter cooperative oncology group trial. Patients were either previously untreated or had failed only one prior chemotherapy regimen. All previously untreated patients had extensive stage disease. Patients in whom prior therapy had been unsuccessful had either limited or extensive stage disease. All cases had histologic documentation. Patients received edatrexate (80 mg/m2) intravenously over 20–30 minutes every 7 days. Previously untreated patients with disease progression at any time or stable disease after 6 weeks of treatment were crossed over to treatment with cisplatin and etoposide. The primary end points of the study were clinical response and toxicity to edatrexate. All patients were observed for survival. Results. Eleven previously untreated and 22 previously treated patients were enrolled. A median of five doses of chemotherapy was given to each group. No major clinical response was observed in either group. The median survival time for the 11 previously untreated patients was 9.8 months versus 3.7 months for individuals who had received prior therapy. Myelosuppression and stomatitis were the primary toxicities, and both were infrequent. Conclusions. Edatrexate is inactive against small cell lung cancer. Cancer 1994;73:1189–93.