日本癌症基因和细胞治疗产品临床开发的最新趋势。

Current Trends in Clinical Development of Gene and Cellular Therapeutic Products for Cancer in Japan.

机构信息

Translational Research Center, The University of Tokyo Hospital, Tokyo, Japan.

Department of Translational Research Promotion Incubation Center for Advanced Medical Science, Kyushu University, Fukuoka, Japan.

出版信息

Clin Ther. 2019 Jan;41(1):174-184.e3. doi: 10.1016/j.clinthera.2018.11.003. Epub 2018 Dec 7.

DOI:10.1016/j.clinthera.2018.11.003

PMID:30528048

Abstract

PURPOSE

In Japan, gene therapy and cellular therapy are categorized as regenerative medicine products based on the Pharmaceuticals and Medical Devices Law that was implemented in 2014. In this new law, regenerative medicine products were newly defined, and a conditional and term-limited approval system for regenerative medicine products was instituted. In addition, the Ministry of Health, Labour and Welfare instituted the SAKIGAKE (meaning pioneer or forerunner in Japanese) designation system in 2015. This designation is similar to the breakthrough therapy designation in the United States. These new regulatory frameworks have stimulated clinical development of new gene and cellular products in Japan. In fact, oncolytic virus therapy for glioblastoma and NY-ESO-1 (T-cell receptor) T-cell therapy for synovial sarcoma were granted SAKIGAKE designation in 2016 and 2018, respectively. Oncolytic virus therapy and genetically engineered T-cell therapy for cancer are being actively developed and examined in investigator-initiated trials.

METHODS

This review analyzes the domestic and international clinical trial registries to comprehensively collect information on clinical trials of gene and cellular therapeutic products for cancer in Japan.

IMPLICATIONS

Current trends in clinical development of gene and cellular therapeutic products for cancer in Japan are discussed.

摘要

目的

在 2014 年实施的《药品和医疗器械法》中，日本将基因治疗和细胞治疗归类为再生医学产品。在这部新法律中，对再生医学产品进行了新的定义，并建立了再生医学产品的有条件和有限期批准制度。此外，厚生劳动省于 2015 年设立了 SAKIGAKE（意为先锋或先驱）指定制度。该指定类似于美国的突破性治疗指定。这些新的监管框架刺激了日本新型基因和细胞产品的临床开发。事实上，溶瘤病毒治疗脑胶质瘤和 NY-ESO-1（T 细胞受体）T 细胞治疗滑膜肉瘤分别于 2016 年和 2018 年获得了 SAKIGAKE 认定。溶瘤病毒治疗和基因工程 T 细胞治疗癌症正在进行积极的研究和临床试验。