Identification, characterisation and therapeutic targeting of leukaemic stem/propagating cells in Acute Myeloid Leukaemia

在英国，每年约有3000例急性髓系白血病（AML）新发病例。这是最常见的侵袭性白血病。尽管在治疗60岁以下患者方面取得了进展，但这些患者中仍有50%死于疾病。此外，80%的患者年龄在60岁以上，在这个年龄组中，只有5%的患者治愈。因此，我们需要改进治疗。AML主要通过在调节血细胞产生的复杂过程的多个基因中获得DNA的遗传变化而零星发生。在一些患者中，存在称为骨髓增生异常（MDS）的白血病前期病症。进一步了解AML的一种方法是定义AML和MDS中所有DNA的遗传变化纲要，并评估这些变化如何解除正常造血干细胞和祖细胞的调节，并将其转化为癌细胞群。这些必要的信息为未来的发展提供了一个合理的平台，直接使患者护理受益。此外，MDS和AML的研究提供了发现癌症生物学和治疗的一般原则的模型。

There are ~3000 new cases a year in the UK of Acute Myeloid Leukaemia (AML). This is the most common aggressive leukaemia. Despite advances in treating patients under the age of 60 years, 50% of these patients still die of their disease. Furthermore, 80% of patients are over the age of 60 and in this age group only 5% of patients are cured. Thus, we need to improve therapy. AML mainly arises sporadically through acquisition of genetic changes in DNA in multiple genes that regulate the complex process of blood cell production. In some patients there is a preleukaemia condition called Myelodysplasia (MDS). One way to further our understanding AML is to define the compendium of genetic changes throughout all our DNA in both AML and MDS and assess how changes deregulate normal blood stem and progenitor cells and transform them into cancerous populations. This necessary information provides a rational platform for future developments to directly benefit patient care. Furthermore study of MDS and AML provides a model to discover general principles in cancer biology and therapy.