Grzybowska-Izydorczyk Olga, Góra-Tybor Joanna, Robak Tadeusz
Katedra i Klinika Hematologii UM w Łodzi.
Postepy Hig Med Dosw (Online). 2006;60:490-7.
Chronic myeloid leukemia (CML) is a malignant clonal disorder of hematopoietic stem cells resulting in increased myeloid and erythroid cells and platelets, and marked hyperplasia in the bone marrow. The natural history of CML is progression from a benign chronic phase, often through an accelerated phase, to a rapidly fatal blast crisis within 3-5 years. The constitutively activated ABL tyrosine kinase domain of the chimeric BCR-ABL oncoprotein is responsible for the transformation of hematopoietic stem cells and the symptoms of CML. Imatinib mesylate (Glivec), a specific small-molecule inhibitor of BCR-ABL, has become the standard drug therapy in all phases of the disease. Imatinib has greatly improved the outcome for patients with CML. Unfortunately, mutations causing resistance to imatinib are leading to relapse in some patients. Considerable progress has recently been made in understanding the structural biology of ABL and the molecular basis for resistance, facilitating the discovery and development of second- generation drugs designed to combat mutant forms of BCR-ABL. The first of these compounds to enter clinical development were dasatinib (BMS-354825) and AMN107 and, from phase I results, both of these promise a breakthrough in the treatment of imatinib-resistant CML.
慢性髓性白血病(CML)是一种造血干细胞的恶性克隆性疾病,导致髓系细胞、红系细胞和血小板增多,并伴有骨髓明显增生。CML的自然病程是从良性慢性期开始,通常会经过加速期,在3至5年内迅速发展为致命的急变期。嵌合型BCR-ABL癌蛋白的组成型激活ABL酪氨酸激酶结构域是造血干细胞转化和CML症状的原因。甲磺酸伊马替尼(格列卫)是一种特异性的BCR-ABL小分子抑制剂,已成为该疾病各阶段的标准药物治疗方法。伊马替尼极大地改善了CML患者的预后。不幸的是,导致对伊马替尼耐药的突变正导致一些患者复发。最近在理解ABL的结构生物学和耐药的分子基础方面取得了相当大的进展,这有助于发现和开发旨在对抗BCR-ABL突变形式的第二代药物。进入临床开发的首批此类化合物是达沙替尼(BMS-354825)和AMN107,从I期结果来看,这两种药物都有望在治疗伊马替尼耐药的CML方面取得突破。