Ricordi C, Japour A
Department of Surgery and Diabetes Research Institute, University of Miami, Miami, FL, USA.
Medical Director, ICON Plc, Miami, FL, USA.
CellR4 Repair Replace Regen Reprogram. 2019;7. doi: 10.32113/cellr4_201910_2768. Epub 2019 Oct 24.
Type 1 diabetes, which affects 1.25 million American children and adults, and more than 20 million people around the world, is a challenging chronic disease caused by the body's inability to make insulin. Among its most severe forms is brittle diabetes. People with brittle diabetes frequently experience large swings in blood sugar that can quickly move from too high to too low or vice versa. Severely low blood sugar, called hypoglycemia, can cause sudden and unexpected seizures, coma, heart attacks, and even death. Insulin is the main treatment for this common disease. But it isn't a cure. A type of cell transplant that comes close to a cure for some people with type 1 diabetes, a technique pioneered and tested in the United States, is now available in many countries but is still deemed an experimental procedure in the U.S., making it almost impossible to get. More than a decade ago, the United Kingdom's National Health Service approved islet cell transplantation for type 1 diabetes - an approval based on an extensive review of the evidence generated by clinical trials conducted in the United States. Our federal dollars supported that research, and this treatment ought to be available to U.S. citizens. Islet cell transplantation is not a panacea for all forms of type 1 diabetes. And transplantation of any organ, including islet cells, requires the use of anti-rejection drugs that can have a range of adverse side effects. Nevertheless, individuals with severe brittle diabetes who are fully informed of the risks and benefits should have the ability to access this lifesaving treatment option. We fully understand the FDA's efforts to rein in companies marketing unapproved stem cell products that have little or no evidence to support their use and that may put patients at risk. Yet the FDA should stay equally focused on its commitment to approving evidence-based transformative treatments for devastating diseases and conditions, including brittle diabetes.
1型糖尿病影响着125万美国儿童和成年人，以及全球超过2000万人，它是一种具有挑战性的慢性疾病，由身体无法分泌胰岛素引起。其最严重的形式之一是脆性糖尿病。脆性糖尿病患者经常经历血糖的大幅波动，血糖水平可能迅速从过高变为过低，反之亦然。严重低血糖，即低血糖症，可导致突然和意外的癫痫发作、昏迷、心脏病发作，甚至死亡。胰岛素是这种常见疾病的主要治疗方法。但它并不能治愈。一种接近治愈某些1型糖尿病患者的细胞移植技术，这项在美国开创并进行了测试的技术，现在在许多国家都已可用，但在美国仍被视为一种实验性程序，几乎无法获得。十多年前，英国国家医疗服务体系批准了1型糖尿病的胰岛细胞移植——这一批准是基于对美国进行的临床试验所产生证据的广泛审查。我们的联邦资金支持了这项研究，这种治疗方法应该提供给美国公民。胰岛细胞移植并非对所有形式的1型糖尿病都是万灵药。而且任何器官的移植，包括胰岛细胞移植，都需要使用抗排斥药物，这些药物可能会产生一系列不良副作用。然而，那些充分了解风险和益处的严重脆性糖尿病患者应该有能力获得这种挽救生命的治疗选择。我们完全理解美国食品药品监督管理局（FDA）为控制那些推销未经批准的干细胞产品的公司所做的努力，这些产品几乎没有或根本没有证据支持其使用，而且可能会使患者面临风险。然而，FDA同样应该专注于其承诺，即批准针对包括脆性糖尿病在内的毁灭性疾病和病症的基于证据的变革性治疗方法。
