A BMI1/CHD7 signature in medulloblastoma with poor prognosis.

脑肿瘤在儿童肿瘤中占很大比例。成神经管细胞瘤是幼童中最常见的脑癌。它们是由发育不良的细胞在生命的早期阶段形成的恶性肿瘤。它们在大脑小脑区域的后脑窝区域发育，但也可能扩散到大脑的其他部位。髓母细胞瘤很少会扩散到身体的其他部位。目前治疗成神经管细胞瘤的主要方法是手术、放疗或化疗。这些治疗方法可以有效地杀死一部分患者的肿瘤细胞；然而，它们几乎总是会产生严重的副作用，对大脑仍在发育的幼儿尤其有害。这些副作用主要影响儿童的身体和智力发展，包括听力和视觉障碍、生长和荷尔蒙变化、生育能力下降、行为改变、学习问题、协调困难和继发性癌症。新慈善资本（New Philanthropy Capital）最近编制的一份报告指出，脑肿瘤是资金投入最严重的癌症之一。2002年至2011年间，脑肿瘤的累计研究支出不到NCRI所有研究支出的1%。我们最近采用了强大的新遗传方法来鉴定特定的分子变化，这些变化表征了一种特别具有侵袭性的髓母细胞瘤亚型，这种亚型没有特定的治疗方法。我们已经从这些肿瘤中培养出了可以在实验室中生长的细胞，我们也有能力培养出患有相同类型肿瘤的老鼠。我们将使用最先进的方法来研究这些肿瘤形成和生长的机制。这些研究将导致识别新的基因和途径，可以阻止或减少肿瘤的形成和生长。我们将以这些基因和途径为目标，确定最有希望作为治疗靶点的基因和途径，这将是朝着开发有效的新一代治疗方法迈出的重要的第一步，治疗这种毁灭性疾病中最具侵略性的亚型之一。

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.