封装的干细胞衍生的β细胞在1型糖尿病患者中施加葡萄糖控制
Bart Keymeulen1, Kaat De Groot2, Daniel Jacobs-Tulleneers-Thevissen2
1Diabetes Research Center, Vrije Universiteit Brussel and Universitair Ziekenhuis Brussel, Brussels, Belgium. bart.keymeulen@uzbrussel.be.
Nature biotechnology
|November 27, 2023
概括
较高剂量的干细胞衍生的胰腺细胞改善了一些1型糖尿病患者的葡萄糖控制. 这项试验优化了细胞输送,显示了未来糖尿病治疗的潜力.
科学领域:
- 再生医学是一种再生医学.
- 内分泌学 在内分泌学.
- 干细胞生物学 干细胞生物学
背景情况:
- 以前使用设备封装的人类胚胎干细胞衍生的胰腺前体细胞用于1型糖尿病的尝试显示胰岛素输出不足.
- 优化细胞移植对于临床疗效至关重要.
研究的目的:
- 报告一阶段1/2试验 (NCT03163511) 的临时1年结果,该试验研究了更高的细胞剂量和优化的设备膜穿孔,用于治疗1型糖尿病.
- 在干细胞衍生胰腺前体细胞移植后评估β细胞功能和血糖控制.
主要方法:
- 一个阶段1/2,开放标签,多中心试验,涉及1型糖尿病患者.
- 在具有优化膜穿孔的设备中,给予2-3倍更高的细胞剂量.
- 在12个月内监测饮食刺激的血C,持续血糖监测 (CGM) 和胰岛素剂量.
主要成果:
- 从第6个月起,10名没有检测到基线C的患者中,有3名患者达到≥0.1nmol/L的水平.
- 达到的C-水平与改善的CGM指标和减少胰岛素需求相关.
- 一名患者显示CGM时间范围显著增加 (12个月后55%至85%),可测量的β细胞质量恢复.
结论:
- 高剂量的干细胞衍生的胰腺前体细胞的优化输送可以恢复部分β细胞功能,并改善1型糖尿病的葡萄糖控制.
- 该研究指出了提高疗效的方向,例如进一步改善植入器件内的β细胞质量和功能.
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