干细胞衍生的胰岛疗法被证明可以减少对注射胰岛素的需求-动脉网

Stem Cell-Derived Islet Therapies Shown to Reduce the Need for Injectable Insulin

CISION 等信源发布 2025-06-21 07:30

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Breakthrough Studies Demonstrate Stem Cell-Based Technologies as a Potential Alternative Treatment for Type 1 Diabetes

突破性研究表明，基于干细胞的技术可作为1型糖尿病的潜在替代疗法。

CHICAGO

芝加哥

，

June 20, 2025

2025年6月20日

/PRNewswire/ -- Findings from two studies reveal advancements in stem cell-derived treatment options to treat type 1 diabetes. Results from the FORWARD study, also simultaneously published in the

/PRNewswire/ -- 两项研究的发现揭示了干细胞衍生治疗方案在治疗1型糖尿病方面的进展。FORWARD研究的结果也同时发表在

New England Journal of Medicine

新英格兰医学杂志

(

NEJM

新英格兰医学杂志

), evaluating the first and only allogeneic, stem-cell derived, islet cell therapy and a study of genetically modified stem cells were unveiled as an oral presentation and late-breaking poster, respectively, at the 85

），评估首个也是唯一的同种异体、干细胞衍生的胰岛细胞疗法以及一项关于基因改造干细胞的研究，分别在第85届会议上作为口头报告和最新突破性海报展示。

Scientific Sessions of the American Diabetes Association

美国糖尿病协会科学会议

(ADA) in

（ADA）在

Chicago

芝加哥

。

Since the 1920s, the standard of care for type 1 diabetes was insulin replacement therapy—requiring millions of Americans to administer insulin via a pump or injection multiple times a day. There is a growing body of research for alternative treatment solutions including allogenic therapies, where stem cells are used to regenerate damaged or impaired tissues as well as insulin production..

自 20 世纪 20 年代以来，1 型糖尿病的护理标准是胰岛素替代疗法——这需要数百万美国人通过泵或注射每天多次注入胰岛素。目前，对于替代治疗方案的研究正在增多，其中包括使用同种异体疗法，利用干细胞再生受损或功能障碍的组织以及胰岛素生产。

'Stem cell therapy is showing tremendous promise in transforming type 1 diabetes care, offering real hope for insulin independence,' said

“干细胞疗法在转变1型糖尿病治疗方面展现了巨大的希望，为实现胰岛素独立提供了真正的希望，”

Marlon Pragnell

马龙·普拉涅尔

, the ADA's vice president of research and science. 'With advances in genetic engineering, these therapies may one day also evade immune attack—eliminating the need for immunosuppressive drugs.'

，ADA研究与科学副总裁。“随着基因工程的进步，这些疗法有朝一日可能还会逃避免疫攻击——消除对免疫抑制药物的需求。”

First-of-Its-Kind Allogenic, Stem Cell Treatment Reduces Injectable Insulin Use in All Participants

首次同种异体干细胞治疗减少所有参与者的注射胰岛素使用量

Findings from the Phase 1/2 FORWARD clinical study—evaluating the safety and efficacy of the stem cell-derived islet product VX-880 for improving glycemic control and reducing the need for exogenous insulin in adults with type 1 diabetes—were presented as an oral presentation at this year's Scientific Sessions and simultaneously published in the .

评估干细胞衍生胰岛产品VX-880在改善1型糖尿病成人患者的血糖控制和减少外源性胰岛素需求方面的安全性和有效性的一期/二期FORWARD临床研究结果，在今年的科学会议上作为口头报告展示，并同时发表在《新英格兰医学杂志》上。

NEJM

新英格兰医学杂志

。

The Phase 1/2, open-label three-part study enrolled 12 adult participants with type 1 diabetes and impaired awareness of hypoglycemia complications to assess the implementation of VX-880, the first and only allogeneic, stem-cell derived, fully differentiated insulin-producing islet cell therapy on glycemic control in pivotal development.

这项1/2期、开放标签、分为三部分的研究招募了12名患有1型糖尿病且对低血糖并发症意识受损的成年参与者，以评估VX-880（首个也是唯一的同种异体、干细胞衍生、完全分化的胰岛素生成细胞疗法）在关键开发阶段对血糖控制的实施效果。

Participants received a full dose of VX-880 islets, transplanted into the liver via a portal vein infusion under a standard immunosuppression drug regime..

参与者接受了一整剂VX-880胰岛，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。

All 12 participants demonstrated restoration of endogenous insulin secretion (measured as C-peptide), elimination of severe hypoglycemia events, and achievement of recommended glycemic control targets (A1C <7% and time in range >70%). The treatment reduced exogenous insulin use in all patients (mean reduction of 92%) and eliminated exogenous insulin use in 10 patients.

所有 12 名参与者均表现出内源性胰岛素分泌（以 C 肽衡量）的恢复、严重低血糖事件的消除，并达到了推荐的血糖控制目标（A1C <7% 且范围内时间 >70%）。该治疗减少了所有患者的外源性胰岛素使用量（平均减少 92%），并在 10 名患者中完全消除了外源性胰岛素的使用。

Adverse events related to the use of VX-880 were consistent with typical islet infusion procedures and existing immunosuppressive drug regimens, and no additional adverse symptoms were observed in any of the participants..

与使用VX-880相关的不良事件与典型的胰岛输注程序和现有的免疫抑制药物方案一致，所有参与者均未观察到额外的不良症状。

'Hypoglycemia remains a dangerous risk for individuals with type 1 diabetes who are dependent on exogenous insulin administration,' shared

“对于依赖外源性胰岛素注射的1型糖尿病患者来说，低血糖仍然是一个危险的风险，”分享道。

Michael Rickels

迈克尔·里克尔斯

, MD, MS,

医学博士，硕士，

Willard and Rhoda Ware

威尔拉德和罗达·威尔

professor in diabetes and metabolic diseases, Division of Endocrinology, Diabetes & Metabolism,

糖尿病和代谢疾病教授，内分泌、糖尿病与代谢科，

University of Pennsylvania

宾夕法尼亚大学

Perelman School of Medicine and presenting investigator. 'These findings indicate the potential for a novel cellular therapy that restores endogenous insulin secretion to improve outcomes for type 1 diabetes patients who have been struggling to achieve glycemic control.'

佩雷尔曼医学院及报告研究员。“这些发现表明了一种新型细胞疗法的潜力，这种疗法可以恢复内源性胰岛素分泌，从而改善那些一直在努力实现血糖控制的1型糖尿病患者的治疗效果。”

The FORWARD study is now in phase 3 which aims to complete enrollment and dosing of approximately 50 participants throughout 2025. Additionally, they plan to launch a VX-880 islet-after-kidney study involving around 10 adults with type 1 diabetes who are already receiving immunosuppressive therapy following a prior kidney transplant..

FORWARD研究现已进入第3阶段，目标是在2025年全年完成大约50名参与者的招募和给药。此外，他们计划启动一项VX-880胰岛细胞移植研究，涉及约10名已经因之前肾移植而接受免疫抑制治疗的1型糖尿病成人患者。

Stem Cell-Based Treatment with Safety Switch Shows Potential for Patients with Type 1 Diabetes

基于干细胞的治疗与安全开关显示出对1型糖尿病患者的潜力

Findings demonstrating the early-stage success of genetically modified stem cell-derived islet (SC-islet) replacement therapies for the treatment of type 1 diabetes, leveraging a novel safety feature, were presented as a late-breaking poster.

展示了一项研究结果的最新海报，该结果证明了利用新型安全特性进行基因改造干细胞衍生胰岛（SC-胰岛）替代疗法在治疗1型糖尿病早期阶段的成功。

Researchers used genetically engineered human embryonic stem cell (hESC) lines to develop specialized cells designed to evade immune system attacks with the integration of eight added protective genes to the SC-islets. The treatment included an inducible kill switch as a safety feature designed to eliminate unwanted or fast-growing cells when activated, using a common, Food and Drug Administration (FDA)-approved antiviral drug (Ganciclovir)..

研究人员使用了经过基因工程改造的人类胚胎干细胞（hESC）系来开发专门的细胞，这些细胞通过在SC胰岛中加入八个保护性基因，旨在逃避免疫系统的攻击。该治疗方案还包含一个诱导性“杀死开关”作为安全特性，当被激活时，可以利用一种常见的、经美国食品药品监督管理局（FDA）批准的抗病毒药物（更昔洛韦）来消除不需要的或快速增长的细胞。

Results showed the specialized cells effectively produced insulin and showed strong and consistent immune evasion while in the cell culture phase of testing. The genetically engineered hESC lines efficiently differentiated into insulin-secreting SC-islets in vitro. When co-cultured with various immune cell types, the SC-islets suppressed immune cell activation and were resistant to immune cell-mediated killing, meaning they successfully evaded immune system attacks.

结果显示，这些特化细胞在培养阶段有效产生胰岛素，并表现出强大而一致的免疫逃逸能力。经过基因工程改造的人类胚胎干细胞系在体外高效分化为分泌胰岛素的SC胰岛。当与多种免疫细胞类型共培养时，SC胰岛抑制了免疫细胞的活化，并对免疫细胞介导的杀伤具有抵抗力，这意味着它们成功逃避免疫系统攻击。

The added safety switch effectively destroyed unwanted cell growth..

附加的安全开关有效地消除了不必要的细胞生长。

'Lab-grown insulin-producing cells show great potential for treatment of type 1 diabetes, but immune system attacks to implanted cells pose an ongoing challenge,' said

“实验室培育的胰岛素生成细胞在1型糖尿病治疗方面展现了巨大的潜力，但免疫系统对植入细胞的攻击仍然是一个持续的挑战，”研究人员表示。

Jia Zhao

贾钊

, PhD, postdoctoral researcher,

，博士，博士后研究员，

University of British Columbia

不列颠哥伦比亚大学

and presenting investigator of the study. 'These early results show potential for safer, longer term cell therapy for diabetes as we look to provide patients with solutions without the need for harmful immune-suppressing drugs.'

以及该研究的负责研究员。'这些早期结果展示了更安全、更长期的糖尿病细胞疗法的潜力，因为我们希望为患者提供无需使用有害免疫抑制药物的解决方案。'

Researchers are now testing these cells in animals to see if they can avoid immune attacks and if the safety switch works as expected in the body.

研究人员现在正在动物身上测试这些细胞，以观察它们是否能够避免免疫攻击，以及安全开关是否在体内按预期工作。

Research presentation details:

研究陈述细节：

Dr. Rickels will present the findings as an oral presentation during the regular abstract session:

里克尔斯博士将在常规摘要会议期间以口头报告的形式展示这些发现：

Innovation and Progress in Stem Cell-Derived Islet-Cell Replacement Therapy

干细胞衍生胰岛细胞替代疗法的创新与进展

Durable Glycemic Control and Elimination of Exogenous Insulin Use with VX-880 in Patients with Type 1 Diabetes (T1D)—VX-880-101 (FORWARD)

VX-880在1型糖尿病（T1D）患者中实现持久的血糖控制并消除外源性胰岛素使用——VX-880-101（FORWARD）

Presented on

呈现于

Friday, June 20

6月20日，星期五

在

6:15 p.m. CT

下午6点15分（中部时间）

Dr. Zhao will present the findings at the late-breaking poster session presentation sessions:

赵博士将在晚间的海报会议中展示这些发现：

Immune-Shielded Islets from Engineered Human Pluripotent Stem Cells for Potential Allogeneic Therapy

来自工程化人类多能干细胞的免疫屏蔽胰岛用于潜在的同种异体治疗

Presented on

呈现于

Sunday, June 22

6月22日，星期日

在

12:30 p.m. CT

中午12点30分（美国中部时间）

About the ADA's Scientific Sessions

关于ADA的科学会议

The ADA's 85

ADA的85

Scientific Sessions, the world's largest scientific meeting focused on diabetes research, prevention, and care, will be held in

科学会议，全球最大的专注于糖尿病研究、预防和护理的科学会议，将举行在

Chicago, IL

伊利诺伊州芝加哥市

, on June 20–23. Thousands of leading physicians, scientists, and health care professionals from around the world are expected to convene both in person and virtually to unveil cutting-edge research, treatment recommendations, and advances toward a cure for diabetes. Attendees will receive exclusive access to thousands of original research presentations and take part in provocative and engaging exchanges with leading diabetes experts.

，6月20日至23日。预计将有数千名来自世界各地的顶尖医生、科学家和医疗保健专业人士亲临现场或通过虚拟方式齐聚一堂，揭示糖尿病领域的前沿研究、治疗建议以及迈向治愈的进展。与会者将获得独家访问数千份原创研究展示的机会，并参与与糖尿病领域权威专家进行的富有启发性和吸引力的交流。

Join the Scientific Sessions conversation on social media using #ADASciSessions..

使用 #ADASciSessions 在社交媒体上加入科学会议的对话。

About the American Diabetes Association

关于美国糖尿病协会

The American Diabetes Association (ADA) is the nation's leading voluntary health organization fighting to end diabetes and helping people thrive. This year, the ADA celebrates 85 years of driving discovery and research to prevent, manage, treat, and ultimately cure—and we're not stopping. There are 136 million Americans living with diabetes or prediabetes.

美国糖尿病协会 (ADA) 是全美领先的志愿健康组织，致力于终结糖尿病并帮助人们茁壮成长。今年，ADA迎来了推动预防、管理、治疗并最终治愈糖尿病的第85个年头，而且我们不会停下脚步。目前有1.36亿美国人患有糖尿病或糖尿病前期。

Through advocacy, program development, and education, we're fighting for them all. To learn more or to get involved, visit us at .

通过宣传、项目开发和教育，我们正在为他们所有人而战。要了解更多信息或参与其中，请访问我们的网站。

diabetes.org

糖尿病.org

or call 1-800-DIABETES (800-342-2383). Join us in the fight on Facebook (

或拨打1-800-DIABETES（800-342-2383）。加入我们在Facebook上的抗争（

American Diabetes Association

美国糖尿病协会

), Spanish Facebook (

），西班牙Facebook（

Asociación Americana de la Diabetes