人胰岛β细胞再生药物治疗糖尿病:过去的成就与未来的挑战。
Human Beta Cell Regenerative Drug Therapy for Diabetes: Past Achievements and Future Challenges.
机构信息
The Diabetes Obesity Metabolism Institute, The Icahn School of Medicine at Mount Sinai, New York, NY, United States.
The Division of Pediatric Endocrinology, The Icahn School of Medicine at Mount Sinai, New York, NY, United States.
出版信息
Front Endocrinol (Lausanne). 2021 Jul 16;12:671946. doi: 10.3389/fendo.2021.671946. eCollection 2021.
A quantitative deficiency of normally functioning insulin-producing pancreatic beta cells is a major contributor to all common forms of diabetes. This is the underlying premise for attempts to replace beta cells in people with diabetes by pancreas transplantation, pancreatic islet transplantation, and transplantation of beta cells or pancreatic islets derived from human stem cells. While progress is rapid and impressive in the beta cell replacement field, these approaches are expensive, and for transplant approaches, limited by donor organ availability. For these reasons, beta cell replacement will not likely become available to the hundreds of millions of people around the world with diabetes. Since the large majority of people with diabetes have some residual beta cells in their pancreata, an alternate approach to reversing diabetes would be developing pharmacologic approaches to induce these residual beta cells to regenerate and expand in a way that also permits normal function. Unfortunately, despite the broad availability of multiple classes of diabetes drugs in the current diabetes armamentarium, none has the ability to induce regeneration or expansion of human beta cells. Development of such drugs would be transformative for diabetes care around the world. This picture has begun to change. Over the past half-decade, a novel class of beta cell regenerative small molecules has emerged: the DYRK1A inhibitors. Their emergence has tremendous potential, but many areas of uncertainty and challenge remain. In this review, we summarize the accomplishments in the world of beta cell regenerative drug development and summarize areas in which most experts would agree. We also outline and summarize areas of disagreement or lack of unanimity, of controversy in the field, of obstacles to beta cell regeneration, and of challenges that will need to be overcome in order to establish human beta cell regenerative drug therapeutics as a clinically viable class of diabetes drugs.
正常功能的胰岛素产生的胰腺β细胞的数量不足是所有常见类型糖尿病的主要原因。这是尝试通过胰腺移植、胰岛移植以及源自人类干细胞的β细胞或胰岛移植来替代糖尿病患者的β细胞的基本前提。虽然在β细胞替代领域的进展迅速且令人印象深刻,但这些方法昂贵,并且对于移植方法,受到供体器官可用性的限制。出于这些原因,β细胞替代不太可能为全世界数以亿计的糖尿病患者提供。由于大多数糖尿病患者的胰腺中仍有一些残留的β细胞,因此逆转糖尿病的另一种方法是开发药物方法来诱导这些残留的β细胞再生和扩张,从而使其恢复正常功能。不幸的是,尽管当前糖尿病武器库中有多种类别的糖尿病药物广泛可用,但没有一种药物具有诱导人类β细胞再生或扩张的能力。此类药物的开发将对全世界的糖尿病护理产生变革性影响。这种情况已经开始改变。在过去的五年中,出现了一类新型的β细胞再生小分子: DYRK1A 抑制剂。它们的出现具有巨大的潜力,但仍存在许多不确定和挑战。在这篇综述中,我们总结了β细胞再生药物开发领域的成就,并总结了大多数专家都认同的领域。我们还概述并总结了该领域存在分歧或缺乏共识的领域、存在争议的领域、β细胞再生的障碍以及为了将人类β细胞再生药物治疗确立为一种可行的糖尿病药物类别而需要克服的挑战。