A BMI1/CHD7 signature in medulloblastoma with poor prognosis.

脑瘤在儿童肿瘤中占很大比例。髓母细胞瘤是儿童中最常见的脑癌。它们是在生命的早期阶段由发育不良的细胞形成的恶性肿瘤。它们在大脑后部称为小脑区域的后窝区域发育，但也可能扩散到大脑的其他部分。极少数情况下，髓母细胞瘤可能会扩散到身体其他部位。目前，儿童髓母细胞瘤的主要治疗方法是手术、放射治疗或化疗。这些治疗方法可以有效地杀死一定比例的患者的肿瘤细胞；然而，它们几乎总是会导致严重的副作用，尤其是在幼儿中，因为大脑仍在发育。这些副作用主要影响儿童的身体和智力发育，包括听力和视觉障碍、生长和荷尔蒙变化、生育力下降、行为变化、学习问题、协调困难和继发性癌症。新慈善资本最近汇编的一份报告指出，脑瘤是最糟糕的资助癌症之一，2002至2011年间，脑瘤的累计研究支出不到NCRI研究支出的1%。我们最近采用了强大的新的遗传学方法来识别特定的分子变化，这些变化表征了一种特别侵袭性的亚型髓母细胞瘤，目前还没有针对这种亚型的特殊治疗方法。我们已经从这些肿瘤中建立了细胞，可以在实验室中生长，我们有能力生产出患有相同类型肿瘤的小鼠。我们将使用最先进的方法来研究这些肿瘤的形成和生长的机制。这些研究将导致识别新的基因和途径，可以有针对性地阻止或减少肿瘤的形成和生长。我们将针对这些基因和途径，确定那些最有希望作为治疗靶点的基因和途径，这将是为这种毁灭性疾病最具侵袭性的亚型之一开发有效的新一代治疗方法的第一个重要步骤。

Brain tumours account for a large proportion of childhood tumours. Medulloblastomas are the most common brain cancers seen in young children. They are malignant tumours formed from poorly developed cells at a very early stage of their life. They develop in a region at the back of the head called the posterior fossa in the region of the brain called the cerebellum, but may spread to other parts of the brain. Very rarely, medulloblastomas may spread to other parts of the body. The main treatments available to children with medulloblastomas today are surgery, radiotherapy or chemotherapy. These treatments can be effective and kill the tumour cells in a proportion of patients; however they almost invariably also result in severe side effects which are particularly damaging in young children as the brain is still growing. These side effects mainly affect a child's physical and intellectual development with hearing and visual disturbances, growth and hormonal changes, reduced fertility, behavioural changes, learning problems, difficulties with coordination and secondary cancers. A report recently compiled by the New Philanthropy Capital, has identified brain tumours as one of the worst funded cancer, the cumulative research spend on brain tumours between 2002 and 2011 was less than 1% of all NCRI research spend. We have recently employed powerful new genetic approaches to identify specific molecular changes that characterize a particularly aggressive subtype of medulloblastomas for which no specific treatment exists. We have established cells from these tumours that can be grown in the laboratory and we have the ability to produce mice that develop the same types of tumours. We will use state-of-the-art methods to study the mechanisms responsible for the formation and growth of these tumours. These studies will lead to the identification of new genes and pathways that can be targeted to stop or reduce tumour formation and growth. We will target these genes and pathways to identify the ones that show most promise as therapeutic targets, which will represent the first important steps towards developing effective, new generation treatments for one of the most aggressive subtypes of this devastating disease.