Cell Therapy for T1D Beyond BLA: Gearing Up Toward Clinical Practice.

IF 3.8 3区医学 Q2 Medicine

Diabetes Therapy Pub Date : 2025-06-01 Epub Date: 2025-04-11 DOI:10.1007/s13300-025-01732-9

Yong Wang, YingYing Chen, James McGarrigle, Jenny Cook, Peter D Rios, Giovanna La Monica, Wei Wei, Jose Oberholzer

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Abstract

Type 1 diabetes (T1D) remains a significant global health challenge and patients with T1D need lifelong insulin therapy. Islet transplantation holds transformative potential by replacing autoimmune-mediated destruction of insulin-producing beta cells. This review examines the trajectory of islet transplantation for T1D, focusing on the process and benefits of obtaining biologics license application (BLA) approval for cell-based therapies. Following US Food and Drug Administration (FDA) approval, the authors identify key steps urgently needed to foster islet transplantation as a viable treatment for a broader population of patients with T1D. Furthermore, the authors highlight recent advances in encapsulation technologies, stem cell-derived islets, xenogeneic islets, and gene editing as strategies to overcome challenges such as immune rejection and limited islet sources. These innovations are pivotal in enhancing the safety and efficacy of islet transplantation. Ultimately, this review emphasizes that while BLA approval represents a critical milestone, realizing the full potential of cell therapy for T1D requires addressing the scientific, clinical, and logistical challenges of its real-world implementation. By fostering innovation, collaboration, and strategic partnerships, the field can transform T1D care, offering patients a durable, life-changing alternative to traditional insulin therapy.

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超越BLA的T1D细胞治疗：加速临床实践。

1型糖尿病（T1D）仍然是一个重大的全球健康挑战，T1D患者需要终身胰岛素治疗。胰岛移植通过取代自身免疫介导的胰岛素生成β细胞的破坏而具有转化潜力。本文综述了胰岛移植治疗T1D的发展轨迹，重点介绍了细胞疗法获得生物制品许可申请（BLA）批准的过程和益处。在美国食品和药物管理局（FDA）批准后，作者确定了迫切需要的关键步骤，以促进胰岛移植作为更广泛的T1D患者的可行治疗方法。此外，作者强调了封装技术、干细胞衍生的胰岛、异种胰岛和基因编辑的最新进展，作为克服免疫排斥和有限胰岛来源等挑战的策略。这些创新对于提高胰岛移植的安全性和有效性至关重要。最后，本综述强调，虽然BLA的批准是一个关键的里程碑，但要实现T1D细胞治疗的全部潜力，需要解决其在现实世界中实施的科学、临床和后勤挑战。通过促进创新、合作和战略伙伴关系，该领域可以改变T1D护理，为患者提供传统胰岛素治疗之外的持久、改变生活的替代方案。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Diabetes Therapy Medicine-Endocrinology, Diabetes and Metabolism

CiteScore

6.90

自引率

7.90%

发文量

130

审稿时长

6 weeks

期刊介绍： Diabetes Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all areas of diabetes. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Diabetes Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.