A cell therapy developed by Vertex Pharma has allowed 10 of 12 patients with type 1 diabetes to control their blood sugar without insulin, according to new data from a phase 1/2 trial.

根据1/2期试验的新数据，Vertex制药公司开发的一种细胞疗法已使12名1型糖尿病患者中的10人能够在无需胰岛素的情况下控制血糖。

One-year results from the company's

公司一年以来的成果

FORWARD-101

向前101

trial of islet cell therapy zimislecel (also known as

胰岛细胞疗法zimislecel（也称为

VX-880

VX-880

) showed that all 12 patients with type 1 diabetes and impaired hypoglycaemia awareness – meaning they find it hard to spot the telltale signs of declining blood sugar levels – showed sustained glycaemic control after the one-shot treatment.

）结果显示，所有12名患有1型糖尿病且低血糖意识受损（意味着他们难以察觉血糖水平下降的明显症状）的患者在接受一次性治疗后，都表现出持续的血糖控制。

Participants received a full dose of the off-the-shelf islet cells, transplanted into the liver via a portal vein infusion under a standard immunosuppression drug regimen.

参与者接受了全剂量的现成胰岛细胞，通过门静脉输注移植到肝脏，并采用标准的免疫抑制药物方案。

Vertex was also developing a version of the therapy called VX-264 in which the cells were encapsulated in a protective device designed to protect them from rejection by the immune system, but that failed to hit the mark in early clinical testing and has been dropped.

Vertex 还在开发一种名为 VX-264 的疗法版本，其中细胞被封装在一种旨在保护它们免受免疫系统排斥的保护装置中，但在早期临床试验中未能达到预期效果，因此已被放弃。

All of the patients in the trial started producing endogenous insulin after the procedure, and after 90 days, none of them had a severe hypoglycaemic event throughout the remainder of the year.

试验中的所有患者在手术后开始产生内源性胰岛素，并且在 90 天后，他们在这一年的剩余时间里都没有出现严重的低血糖事件。

They all hit glycaemic control targets – measured by haemoglobin A1C levels reaching the target of less than 7% with a time in range of over 70% – and reduced their need for insulin injections by 92%, according to Michael Rickels of the University of Pennsylvania's Perelman School of Medicine, who presented the results at the ADA congress in Chicago..

他们都达到了血糖控制目标——通过血红蛋白A1C水平测量，达到小于7%的目标，并且血糖在目标范围内的时间超过70%——而且他们对胰岛素注射的需求减少了92%，据宾夕法尼亚大学佩雷尔曼医学院的迈克尔·里克尔斯称，他在芝加哥举行的ADA大会上公布了这一结果。

Hypoglycaemia remains a dangerous risk for individuals with type 1 diabetes, particularly in those with impaired awareness, as episodes can lead to confusion, coma, seizures, cardiovascular events, and even death, Rickels told delegates.

瑞克斯告诉与会者，低血糖对1型糖尿病患者来说仍然是一个危险的风险，特别是对那些意识受损的患者，因为低血糖发作可能导致混乱、昏迷、癫痫发作、心血管事件甚至死亡。

'As I think about my patients and the unmet need in the type 1 diabetes community, the results we’ve seen so far for restoring endogenous insulin secretion with a stem cell-derived islet therapy bring me hope and confidence for a transformative treatment option for individuals with type 1 diabetes in the not-so-distant future,' he added..

“当我考虑到我的患者以及1型糖尿病社区未满足的需求时，我们迄今为止看到的通过干细胞衍生的胰岛疗法恢复内源性胰岛素分泌的结果，让我对未来不远的1型糖尿病患者的变革性治疗选择充满希望和信心，”他补充道。

Vertex has expanded the FORWARD-101 trial into a phase 3 stage that has a target of enrolling and dosing 50 subjects before the end of the year, and has also started recruiting patients in Europe and Canada as well as the US.

Vertex已经将FORWARD-101试验扩展到第3阶段，目标是在年底前招募并给药50名受试者，同时已开始在美国、欧洲和加拿大招募患者。

Meanwhile, the company is preparing a second study of zimislecel in around 10 adults with type 1 diabetes who are already receiving immunosuppressive therapy following a prior kidney transplant.

同时，该公司正准备对大约 10 名已在接受先前肾移植后免疫抑制治疗的 1 型糖尿病成人进行第二次 zimislecel 研究。

'These data on the first fully differentiated, stem cell-derived, off-the-shelf islet cell therapy continue to be unprecedented,' said Vertex's chief medical officer Carmen Bozic. 'We are excited to complete enrolment and dosing in the phase 1/2/3 programme and look forward to potential regulatory submissions next year.'.

“这些关于首个完全分化、干细胞衍生、现成可用的胰岛细胞疗法的数据仍然是前所未有的，”Vertex首席医疗官卡门·博齐克表示。“我们很高兴完成了1/2/3期项目的入组和给药，并期待明年可能的监管提交。”