专题论文

生物技术药物发展迅猛,突破性疗法认证推动药物创新——2017年新药研发热点回眸

  • 李子艳 ,
  • 黄瑶庆 ,
  • 周映红 ,
  • 毛艳艳 ,
  • 高柳滨
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  • 中国科学院上海药物研究所信息中心, 上海 201203
李子艳,馆员,研究方向为药物情报分析,电子信箱:zyli@simm.ac.cn

收稿日期: 2017-12-20

  修回日期: 2018-01-02

  网络出版日期: 2018-01-30

基金资助

中国科学院A类战略性先导科技专项(XDA12050201)

Hot topics in drug research and development in 2017: Biological drug rapid growth, breakthrough therapy certification

  • LI Ziyan ,
  • HUANG Yaoqing ,
  • ZHOU Yinghong ,
  • MAO Yanyan ,
  • GAO Liubin
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  • Information Center, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China

Received date: 2017-12-20

  Revised date: 2018-01-02

  Online published: 2018-01-30

摘要

新药研发集中体现生命科学和生物技术领域的新突破和新进展。当前,药物研发相关学科交叉日趋紧密,新方法、新技术层出不穷,在提升药物研发效率的同时快速革新疾病的治疗手段。从2017年美国食品药品监督管理局(FDA)批准的新药来看,生物技术药物发展异常迅猛,突破性疗法认证推动药物创新,临床上未满足的需求成为行业的追求目标。本文回顾2017年新药研发领域的突破性进展和研发热点,展示新药研发新趋势和新动向。

本文引用格式

李子艳 , 黄瑶庆 , 周映红 , 毛艳艳 , 高柳滨 . 生物技术药物发展迅猛,突破性疗法认证推动药物创新——2017年新药研发热点回眸[J]. 科技导报, 2018 , 36(1) : 116 -125 . DOI: 10.3981/j.issn.1000-7857.2018.01.014

Abstract

Drug R&D focus on breakthroughs and new progress in the field of life science and biotechnology. At present, drug R&D related disciplines become increasingly intersecting, new methods and technologies emerge rapidly, constantly improving the efficiency of drug R&D and innovating the way of disease treatment. From the perspective of new drugs approved by FDA in 2017, with the biotech drugs developing fast and the breakthrough therapy certification promoting drug innovation, unmet needs in the clinic are turning into the pursuit of the industry. This paper reviews the breakthrough progress and research hot topics in the field of drug R&D in 2017, exhibiting the direction and new trends to people in this field.

参考文献

[1] Siegel R L, Miller K D, Jemal A. Cancerstatistics[J]. CA:A Cancer Journal for Clinicians, 2017, 67(1):7-30.
[2] Kakimi K, Karasaki T, Matsushita H, et al. Advances in personalized cancer immunotherapy[J]. Breast Cancer, 2017, 24(1):16-24.
[3] Ludin A, Zon L I. Cancer immunotherapy:The dark side of PD-1 receptor inhibition[J]. Nature, 2017, 552(7683):41-42.
[4] Wartewig T, Kurgyis Z, Keppler S, et al. PD-1 is a haploinsufficient suppressor of T cell lymphomagenesis[J]. Nature, 2017, 552(7683):121-125.
[5] Jennifer C F. Powerful new cancer drugs are saving lives, but can also ignite diabetes or other autoimmune conditions[EB/OL]. (2017-11-15)[2017-12-05]. http://www.sciencemag.org/news/2017/11/powerful-new-cancer-drugs-are-saving-livescan-also-ignite-diabetes-or-other-autoimmune.
[6] Vétizou M, Pitt J M, Daillère R, et al. Anticancer immunotherapy by CTLA-4 blockade relies on the gut microbiota[J]. Science, 2015, 350(6264):1079-1084.
[7] Botticelli A, Zizzari I, Mazzuca F, et al. Cross-talk between microbiota and immune fitness to steer and control response to anti PD-1/PDL-1 treatment[J]. Oncotarget, 2017, 8(5):8890-8899.
[8] KYMRIAH (tisagenlecleucel)[EB/OL]. (2017-08-30)[2017-12-05]. https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM574106.pdf
[9] Mullard A. FDA approves first CAR T therapy[J]. Nature Reviews. Drug Discovery, 2017, 16(10):669.
[10] YESCARTA (axicabtagene ciloleucel)[EB/OL]. (2017-10-18)[2017-12-05]. https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM581259.pdf.
[11] Kingwell K. CAR T therapies drive into new terrain[J]. Nature Reviews. Drug Discovery, 2017, 16(5):301-304.
[12] Pembrolizumab (Keytruda) 5-10-2017[EB/OL]. (2017-05-10)[2017-12-05]. https://www.fda.gov/drugs/informationondrugs/approveddrugs/ucm558048.htm.
[13] Garber K. Oncologists await historic first:A pan-tumor predictive marker, for immunotherapy[J]. Nature Biotechnology, 2017, 35(4):297-298.
[14] Dudley J C, Lin M T, Le D T, et al. Microsatellite instability as a biomarker for PD-1 blockade[J]. Clinical Cancer Research:An Official Journal of the American Association for Cancer Research, 2016, 22(4):813-820.
[15] Melissa H. Immunotherapy drug opens a new era of precision medicine for cancer[EB/OL]. (2017-05-26)[2017-12-05]. http://www.latimes.com/science/sciencenow/la-sci-sn-cancerimmunotherapy-drug-20170526-story.html?from=singlemessage&isappinstalled=0.
[16] Liu L, Li X, Wang J, et al. Two distant catalytic sites are responsible for C2c2 RNase activities[J]. Cell, 2017, 168(1/2):121-134.
[17] Eyquem J, Mansilla-Soto J, Giavridis T, et al.Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection[J]. Nature, 2017, 543(7643):113-117.
[18] Patel S J, Sanjana N E, Kishton R J, et al. Identification of essential genes for cancer immunotherapy[J]. Nature, 2017, 548(7669):537-542.
[19] Wan L, Wen H, Li Y, et al. ENL links histone acetylation to oncogenic gene expression in acute myeloid leukaemia[J]. Nature, 2017, 543(7644):265-269.
[20] Manguso R T, Pope H W, Zimmer M D, et al. In vivo CRISPR screening identifies Ptpn2 as a cancer immunotherapy target[J]. Nature, 2017, 547(7664):413-418.
[21] Committee on human gene editing:Scientific, medical, and ethical considerations. Human Genome Editing:Science, Ethics, and Governance (2017)[R]. Washington D C:National Academies of Sciences, Engineering, and Medicine, 2017.
[22] Ma H, Marti-Gutierrez N, Park SW, et al. Correction of a pathogenic gene mutation in human embryos[J]. Nature, 2017, 548(7668):413-419.
[23] Jiang C, Mei M, Li B, et al. A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo[J]. Cell Research, 2017, 27(3):440-443.
[24] Basu S, Adams L, Guhathakurta S, et al.A novel tool for monitoring endogenous alpha-synuclein transcription by NanoLuciferase tag insertion at the 3'end using CRISPR-Cas9 genome editing technique[J]. Scientific Reports, 2017, 8:45883.
[25] O'Neil N J, Bailey M L, Hieter P. Synthetic lethality and cancer[J]. Nature Reviews. Genetics, 2017, 18(10):613-623.
[26] Banerjee S, Kaye SB, Ashworth A.Making the best of PARP inhibitors in ovarian cancer[J]. Nature Reviews. Clinical Oncology, 2010, 7(9):508-519.
[27] FDA approves PARP inhibitor for ovarian cancer[J]. Nature Biotechnology, 2017, 35(5):398.
[28] Mullard A. Synthetic lethality screens point the way to new cancer drug targets[J]. Nature Reviews. Drug Discovery, 2017, 16(10):736.
[29] Wang T, Yu H, Hughes N W, et al. Gene essentiality profiling reveals gene networks and synthetic lethal interactions with oncogenic Ras[J]. Cell, 2017, 168(5):890-903.
[30] Barbieri I, Tzelepis K, Pandolfini L, et al. Promoter-bound METTL3 maintains myeloid leukaemia by m6A-dependent translation control[J]. Nature, 2017, 552(7683):126-131.
[31] MIT Technology Review. 10 Breakthrough Technologies 2017[EB/OL]. (2017-02-24)[2017-12-05]. https://www.technologyreview.com/lists/technologies/2017/.
[32] Spark Therapeutics. FDA advisory committee unanimously recommends approval of investigational LUXTURNATM (voretigeneneparvovec) for patients with Biallelic RPE65-mediated Inherited retinal disease[EB/OL]. (2017-10-12)[2017-12-05]. http://ir.sparktx.com/news-releases/news-release-details/fda-advisory-committee-unanimously-recommends-approval.
[33] Bennett J, Wellman J, Marshall K A, et al. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations:A follow-on phase 1 trial[J]. Lancet, 2016, 388(10045):661-672.
[34] Russell S, Bennett J, Wellman J A, et al. Efficacy and safety of voretigeneneparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy:A randomised, controlled, open-label, phase 3 trial[J]. Lancet, 2017, 390(10097):849-860.
[35] Morrison C. Landmark gene therapy poised for US approval[J]. Nature Reviews. Drug Discovery, 2017, 16(11):739-741.
[36] Sangamo Therapeutics. Corporate Presentation[EB/OL]. (2017-08-21)[2017-12-05]. https://investor.sangamo.com/presentations.
[37] Jocelyn K. A human has been injected with gene-editing tools to cure his disabling disease. Here's what you need to know[EB/OL]. (2017-11-15)[2017-12-05]. http://www.sciencemag.org/news/2017/11/human-has-been-injected-geneediting-tools-cure-his-disabling-disease-here-s-what-you.
[38] Mendell J R, Al-Zaidy S, Shell R, et al. Single-dose gene-replacement therapy for spinal muscular atrophy[J]. The New England journal of medicine, 2017, 377(18):1713-1722.
[39] Silver D, Schrittwieser J, Simonyan K, et al. Mastering the game of Go without human knowledge[J]. Nature, 2017, 550(7676):354-359.
[40] Baskin I I, Winkler D, Tetko I V. A renaissance of neural networks in drug discovery[J]. Expert Opinion on Drug Discovery, 2016, 11(8):785-795.
[41] Mullard A. The drug-maker's guide to the galaxy[J]. Nature, 2017, 549(7673):445-447.
[42] Smalley E. AI-powered drug discovery captures pharma interest[J]. Nature Biotechnology, 2017, 35(7):604-605.
[43] Novel drug approvals for 2017[EB/OL]. (2017-11-30)[2017-12-05]. https://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/ucm537040.htm.
[44] Hunter N L, Rao G R, Sherman R E. Flexibility in the FDA approach to orphan drug development[J]. Nature Reviews. Drug Discovery, 2017, 16(11):737-738.
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