Moffitt Cancer Center, Tampa, FL 33647, USA.
Expert Opin Investig Drugs. 2010 May;19(5):689-98. doi: 10.1517/13543781003801076.
Acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) incidence in the United States increases with age. Given the progressive ageing of the general population, incidence of these diseases is likely to continue to rise in the future. There is an acute need for therapeutic developments because of the poor prognosis of these diseases. Since the knowledge of molecular genetics in AML and MDS has expanded recently, targeted therapeutics should offer an exciting new frontier for advancement. Of all the targeted inhibitors developed, tipifarnib represents one of the few compounds with some activity as a single agent.
Described in this review are the molecular targets of tipifarnib, safety and tolerability of the drug, chemistry, and clinical efficacy in AML.
The reader will gain a thorough understanding of tipifarnib as it relates to the current and future use of the drug in AML.
The future of tipifarnib, along with other molecularly-targeted drugs, lies in achieving a better understanding of leukemia biology and harnessing the activity of this agent using predictive biomarkers for improved patient selection.
在美国,急性髓系白血病(AML)和骨髓增生异常综合征(MDS)的发病率随年龄增长而增加。鉴于总人口的老龄化进程不断推进,这些疾病的发病率在未来很可能继续上升。由于这些疾病的预后较差,因此迫切需要治疗方面的进展。由于 AML 和 MDS 中的分子遗传学知识最近有所扩展,靶向治疗应该为进一步发展提供一个令人兴奋的新领域。在所有开发的靶向抑制剂中,替匹法尼是具有一定单药活性的少数几种化合物之一。
本篇综述介绍了替匹法尼的分子靶点、该药的安全性和耐受性、化学特性以及在 AML 中的临床疗效。
读者将全面了解替匹法尼在 AML 中当前和未来的应用。
替匹法尼以及其他分子靶向药物的未来在于更好地了解白血病生物学,并利用预测生物标志物来利用该药物的活性,从而改善患者选择。