Tipifarnib effective oral therapy for myelodysplastic syndromes

Tipifarnib [Zarnestra, R 115777], an oral farnesyltransferase inhibitor, is an effective treatment for high-risk myelodysplastic syndromes (MDS), according to studies presented at the 46th Annual Meeting of the American Society of Hematology (ASH) [San Diego, California, US; December 2004]. A phase II study showed that tipifarnib produced durable clinical responses and haematological improvements in both treatment-naive and pre-treated patients. In a separate phase I study, alternate-week administration of tipifarnib produced clinical responses and major platelet responses in patients with MDS. Notably, a 50% response rate was observed in patients receiving the lowest dose of tipifarnib.