Phase 1 evaluation of CRISPR-CAR gene edited T cells in relapsed refractory B cell acute lymphoblastic leukaemia

白血病的新疗法利用基因疗法来重新编程白色细胞以对抗癌细胞，这显示出很大的希望。在一些试验中，每10名接受治疗的患者中有8名以上有良好的反应。到目前为止，大多数试验都依赖于收集患者自己的白色细胞，然后使用一种失能病毒将新基因装入一组称为T细胞的细胞中。这个过程然后武装细胞对抗白血病，所以当他们回到病人身上时，他们可以攻击疾病。我们最近发现，如果从健康捐赠者身上提取细胞，并在没有任何匹配的情况下移植到病人身上，不仅可以武装细胞对抗白血病，而且还可以解除它们的武装，以阻止副作用。一种名为基因编辑的技术被用来实现这一目标，我们目前正在进行一项临床试验，以在更多患者中测试这种方法。与此同时，我们修饰细胞的方式的改进导致了我们认为将被更广泛使用的新版本治疗的发展。该项目将测试使用CRISPR基因编辑制作的新版本，看看我们是否可以治疗所有其他治疗方法都失败的儿童白血病。我们的目标是在两年内治疗10名儿童，如果他们能够清除白血病，他们将接受骨髓移植，以提高治愈的机会。

New treatments for leukaemia using gene therapy to re-program white bloods cells against cancer cells are showing a great deal of promise. In some trials, over 8 out of every 10 patient treated had a good response. Most of the trials so far have relied in collecting a patients own white cells and then using a disabled virus to fit new genes into a group of cells called T cells. This process then arms the cells against leukaemia, so when they are given back to the patients they can attack the disease. We recently showed that its possible not only to arm cells against leukaemia but also disarm them to stop side effects if they are taken from a healthy donor and transplanted to a patient without any matching. A technique called gene editing was used to to achieve this, and we are currently running a clinical trial to test the approach in more patients. In the meantime, improvements in the way we modify cells have led to the development of new versions of the treatment that we think are going to be more widely used. This project will test the new versions, made using CRISPR gene editing, to see if we can treat leukaemia in children where all other treatments have failed. We aim to treat 10 children over two years, and if they can be cleared of leukaemia, they will receive a vote marrow transplant to improve their chances of cure.