Aljedai Abdullah, Buckle Anne-Marie, Hiwarkar Prashant, Syed Farhatullah
Faculty of Life Sciences, Manchester Institute of Biotechnology (MIB), University of Manchester, Manchester, United Kingdom.
Department of Paediatric Haematology, Great Ormond Street Hospital (GOSH), London, United Kingdom.
PLoS One. 2015 Apr 7;10(4):e0123016. doi: 10.1371/journal.pone.0123016. eCollection 2015.
Notch signalling is critical for haemopoietic stem cell (HSC) self-renewal and survival. The role of Notch signalling has been reported recently in chronic myeloid leukaemia (CML) - a stem cell disease characterized by BCR-ABL tyrosine kinase activation. Therefore, we studied the relationship between BCR-ABL and Notch signalling and assessed the expression patterns of Notch and its downstream target Hes1 in CD34+ stem and progenitor cells from chronic-phase CML patients and bone marrow (BM) from normal subjects (NBM). We found significant upregulation (p<0.05) of Notch1, Notch2 and Hes1 on the most primitive CD34+Thy+ subset of CML CD34+ cells suggesting that active Notch signalling in CML primitive progenitors. In addition, Notch1 was also expressed in distinct lymphoid and myeloid progenitors within the CD34+ population of primary CML cells. To further delineate the possible role and interactions of Notch with BCR-ABL in CD34+ primary cells from chronic-phase CML, we used P-crkl detection as a surrogate assay of BCR-ABL tyrosine kinase activity. Our data revealed that Imatinib (IM) induced BCR-ABL inhibition results in significant (p<0.05) upregulation of Notch activity, assessed by Hes1 expression. Similarly, inhibition of Notch leads to hyperactivation of BCR-ABL. This antagonistic relationship between Notch and BCR-ABL signalling was confirmed in K562 and ALL-SIL cell lines. In K562, we further validated this antagonistic relationship by inhibiting histone deacetylase (HDAC) - an effector pathway of Hes1, using valproic acid (VPA) - a HDAC inhibitor. Finally, we also confirmed the potential antagonism between Notch and BCR/ABL in In Vivo, using publically available GSE-database, by analysing gene expression profile of paired samples from chronic-phase CML patients pre- and post-Imatinib therapy. Thus, we have demonstrated an antagonistic relationship between Notch and BCR-ABL in CML. A combined inhibition of Notch and BCR-ABL may therefore provide superior clinical response over tyrosine-kinase inhibitor monotherapy by targeting both quiescent leukaemic stem cells and differentiated leukaemic cells and hence must be explored.
Notch信号通路对造血干细胞(HSC)的自我更新和存活至关重要。最近有报道称Notch信号通路在慢性髓性白血病(CML)中发挥作用,CML是一种以BCR-ABL酪氨酸激酶激活为特征的干细胞疾病。因此,我们研究了BCR-ABL与Notch信号通路之间的关系,并评估了慢性期CML患者CD34+干细胞和祖细胞以及正常受试者骨髓(NBM)中Notch及其下游靶点Hes1的表达模式。我们发现,在CML CD34+细胞中最原始的CD34+Thy+亚群上,Notch1、Notch2和Hes1显著上调(p<0.05),这表明CML原始祖细胞中存在活跃的Notch信号通路。此外,Notch1也在原发性CML细胞CD34+群体内不同的淋巴样和髓样祖细胞中表达。为了进一步阐明Notch与BCR-ABL在慢性期CML的CD34+原代细胞中的可能作用及相互作用,我们使用P-crkl检测作为BCR-ABL酪氨酸激酶活性的替代检测方法。我们的数据显示,伊马替尼(IM)诱导的BCR-ABL抑制导致Notch活性显著上调(p<0.05),通过Hes1表达进行评估。同样,抑制Notch会导致BCR-ABL的过度激活。Notch与BCR-ABL信号通路之间的这种拮抗关系在K562和ALL-SIL细胞系中得到了证实。在K562中,我们通过使用丙戊酸(VPA)——一种HDAC抑制剂,抑制组蛋白去乙酰化酶(HDAC)——Hes1的效应通路,进一步验证了这种拮抗关系。最后,我们还通过分析慢性期CML患者伊马替尼治疗前后配对样本的基因表达谱,利用公开可用的GSE数据库,在体内证实了Notch与BCR/ABL之间的潜在拮抗作用。因此,我们证明了CML中Notch与BCR-ABL之间存在拮抗关系。因此,联合抑制Notch和BCR-ABL可能通过靶向静止的白血病干细胞和分化的白血病细胞,比酪氨酸激酶抑制剂单一疗法提供更好的临床反应,因此必须进行探索。