Mitochondrial replacement therapy: the UK and US regulatory landscapes
Mitochondrial replacement therapy: the UK and US regulatory landscapes
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DOI：
10.1093/jlb/lsw051
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发表时间：
2016-11-01
影响因子：
3.4
通讯作者：
Castro, Rosa J.
中科院分区：
文献类型：
--
作者：
Castro, Rosa J.
2015年10月29日，英国关于线粒体捐赠的法规（UK 2015 法规）生效。 1 英国人类受精和胚胎学法案（“HFE 法案”）的这项修正案允许使用线粒体捐赠技术作为体外受精 (IVF) 治疗的一部分。线粒体替代疗法（MRT）旨在防止线粒体疾病从母亲传染给其遗传相关的孩子。在美国，MRT 的使用受到美国食品和药物管理局 (FDA) 的监督，其批准需要根据研究性新药申请进行临床试验，目前已通过 2016 年国会拨款法案中包含的附加条款暂停。 2本说明讨论了英国的 MRT 监管框架，并将其与美国的情况进行了比较。它重点关注两国的监管和伦理讨论，为有关编辑人类生殖细胞的争论寻找一些教训。 3 第一部分介绍线粒体的一些生物学特征及其对线粒体疾病、医疗干预以及伦理和监管问题的影响。第二部分讨论英国 2015 年法规通过的监管途径以及批准文本的主要特点。第三部分考虑了美国当前的监管环境。第四部分讨论 MRT 提出的一些监管和生物伦理问题。
On October 29, 2015, the United Kingdom’s regulations on mitochondrial donation (UK 2015 Regulations) came into force. 1 This amendment to the UK Human Fertilisation and Embryology Act (the ‘HFE Act’) allows the use of mitochondrial donation techniques as part ofin vitrofertilization (IVF) treatments. Mitochondrial replacement therapies (MRT) aim at preventing the transmission of mitochondrial disease from a mother to her genetically related children. In the USA, the use of MRT falls under the oversight of the US Food and Drug Administration (FDA), and its approval, which would require clinical trials under an investigational new drug application, has been currently halted through a rider included in the 2016 Congressional Appropriations Act. 2This note discusses the UK regulatory framework for MRT and compares it to the US landscape. It focuses on the regulatory and ethical discussions in both countries to find some lessons for debates about editing human germ cells. 3 The first section introduces some biological characteristics of mitochondria and their implications for mitochondrial diseases, medical interventions and ethical and regulatory questions. The second section discusses the regulatory pathway leading to the adoption of the UK 2015 regulations and the main features of the approved text. The third section considers the current regulatory landscape in the USA. The fourth section discusses some regulatory and bioethical questions raised by MRT.