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在死亡谷中生存:转化学术药物发现的机遇与挑战

Surviving in the Valley of Death: Opportunities and Challenges in Translating Academic Drug Discoveries.

机构信息

SPARK Translational Research Program, Stanford University School of Medicine, Stanford, California 94305, USA; email:

Department of Chemical and Systems Biology, Stanford University School of Medicine, Stanford, California 94305, USA.

出版信息

Annu Rev Pharmacol Toxicol. 2019 Jan 6;59:405-421. doi: 10.1146/annurev-pharmtox-010818-021625. Epub 2018 Sep 12.

DOI:10.1146/annurev-pharmtox-010818-021625
PMID:30208282
Abstract

With pharmaceutical companies shrinking their research departments and exiting out of efforts related to unprofitable diseases, society has become increasingly dependent on academic institutions to perform drug discovery and early-stage translational research. Academic drug discovery and translational research programs assist in shepherding promising therapeutic opportunities through the so-called valley of death in the hope that a successful new drug will result in saved lives, improved health, economic growth, and financial return. We have interviewed directors of 16 such academic programs in the United States and found that these programs and the projects therein face numerous challenges in reaching the clinic, including limited funding, lack of know-how, and lack of a regional drug development ecosystem. If these issues can be addressed through novel industry partnerships, the revision of government policies, and expanded programs in translational education, more effective new therapies are more likely to reach patients in need.

摘要

随着制药公司缩减其研究部门,并退出与无利可图的疾病相关的研发,社会越来越依赖学术机构开展药物发现和早期转化研究。学术药物发现和转化研究项目有助于引导有前途的治疗机会通过所谓的死亡之谷,以期成功开发出新药,从而拯救生命、改善健康、促进经济增长和带来经济回报。我们采访了美国 16 个这样的学术项目的主任,发现这些项目及其内部的项目在进入临床阶段面临着许多挑战,包括资金有限、缺乏专业知识和缺乏区域药物开发生态系统。如果这些问题能够通过新颖的行业合作、政府政策的修订以及扩大转化教育项目来解决,那么更有效的新疗法就更有可能惠及有需要的患者。

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