O'Hare Thomas, Eide Christopher A, Tyner Jeffrey W, Corbin Amie S, Wong Matthew J, Buchanan Sean, Holme Kevin, Jessen Katayoun A, Tang Crystal, Lewis Hal A, Romero Richard D, Burley Stephen K, Deininger Michael W
Division of Hematology and Medical Oncology, Oregon Health & Science University Cancer Institute, 3181 SW Sam Jackson Park Road, Portland, OR 97239, USA.
Proc Natl Acad Sci U S A. 2008 Apr 8;105(14):5507-12. doi: 10.1073/pnas.0800587105. Epub 2008 Mar 26.
Imatinib inhibits Bcr-Abl, the oncogenic tyrosine kinase that causes chronic myeloid leukemia. The second-line inhibitors nilotinib and dasatinib are effective in patients with imatinib resistance resulting from Bcr-Abl kinase domain mutations. Bcr-Abl(T315I), however, is resistant to all Abl kinase inhibitors in clinical use and is emerging as the most frequent cause of salvage therapy failure. SGX393 is a potent inhibitor of native and T315I-mutant Bcr-Abl kinase that blocks the growth of leukemia cell lines and primary hematopoietic cells expressing Bcr-Abl(T315I), with minimal toxicity against Bcr-Abl-negative cell lines or normal bone marrow. A screen for Bcr-Abl mutants emerging in the presence of SGX393 revealed concentration-dependent reduction in the number and range of mutations. Combining SGX393 with nilotinib or dasatinib preempted emergence of resistant subclones, including Bcr-Abl(T315I). These findings suggest that combination of a T315I inhibitor with the current clinically used inhibitors may be useful for reduction of Bcr-Abl mutants in Philadelphia chromosome-positive leukemia.
伊马替尼可抑制导致慢性粒细胞白血病的致癌酪氨酸激酶Bcr-Abl。二线抑制剂尼罗替尼和达沙替尼对因Bcr-Abl激酶结构域突变而产生伊马替尼耐药的患者有效。然而,Bcr-Abl(T315I)对临床使用的所有Abl激酶抑制剂均耐药,并且正成为挽救治疗失败的最常见原因。SGX393是天然型和T315I突变型Bcr-Abl激酶的强效抑制剂,可阻断表达Bcr-Abl(T315I)的白血病细胞系和原代造血细胞的生长,对Bcr-Abl阴性细胞系或正常骨髓的毒性极小。对在SGX393存在的情况下出现的Bcr-Abl突变体进行的筛选显示,突变的数量和范围呈浓度依赖性减少。将SGX393与尼罗替尼或达沙替尼联合使用可预防耐药亚克隆的出现,包括Bcr-Abl(T315I)。这些发现表明,将T315I抑制剂与目前临床使用的抑制剂联合使用可能有助于减少费城染色体阳性白血病中的Bcr-Abl突变体。