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让任何人都能将具有临床相关性的想法转化为治疗方法。

Enabling Anyone to Translate Clinically Relevant Ideas to Therapies.

作者信息

Ekins Sean, Diaz Natalie, Chung Julia, Mathews Paul, McMurtray Aaron

机构信息

Collaborations Pharmaceuticals, Inc., 5616 Hilltop Needmore Road, Fuquay-Varina, Noth Carolina, 27526, USA.

Phoenix Nest, Inc., P.O. BOX 150057, Brooklyn, New York, 11215, USA.

出版信息

Pharm Res. 2017 Jan;34(1):1-6. doi: 10.1007/s11095-016-2039-5. Epub 2016 Sep 12.

Abstract

How do we inspire new ideas that could lead to potential treatments for rare or neglected diseases, and allow for serendipity that could help to catalyze them? How many potentially good ideas are lost because they are never tested? What if those ideas could have lead to new therapeutic approaches and major healthcare advances? If a clinician or anyone for that matter, has a new idea they want to test to develop a molecule or therapeutic that they could translate to the clinic, how would they do it without a laboratory or funding? These are not idle theoretical questions but addressing them could have potentially huge economic implications for nations. If we fail to capture the diversity of ideas and test them we may also lose out on the next blockbuster treatments. Many of those involved in the process of ideation may be discouraged and simply not know where to go. We try to address these questions and describe how there are options to raising funding, how even small scale investments can foster preclinical or clinical translation, and how there are several approaches to outsourcing the experiments, whether to collaborators or commercial enterprises. While these are not new or far from complete solutions, they are first steps that can be taken by virtually anyone while we work on other solutions to build a more concrete structure for the "idea-hypothesis testing-proof of concept-translation-breakthrough pathway".

摘要

我们如何激发能带来罕见病或被忽视疾病潜在治疗方法的新想法,并让有助于促成这些想法的意外发现出现?有多少潜在的好想法因从未得到检验而被埋没?要是这些想法本可带来新的治疗方法和重大医疗进展又会怎样?如果临床医生或其他任何人有一个想要检验的新想法,想要研发一种可转化至临床的分子或疗法,在没有实验室或资金的情况下他们该怎么做?这些并非无意义的理论问题,而解决这些问题可能会给各国带来潜在的巨大经济影响。如果我们未能捕捉想法的多样性并对其进行检验,我们可能也会错失下一个重磅治疗方法。许多参与构思过程的人可能会灰心丧气,根本不知道该何去何从。我们试图解决这些问题,并描述有哪些筹集资金的选择、即使是小规模投资如何能促进临床前或临床转化,以及有几种将实验外包给合作者或商业企业的方法。虽然这些并非全新或完备的解决方案,但它们是几乎任何人都可以迈出的第一步,与此同时我们致力于寻找其他解决方案,为“想法 - 假设检验 - 概念验证 - 转化 - 突破路径”构建更具体的架构。

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