Children's Tumor Foundation, New York, NY, USA.
EMBO Mol Med. 2017 Sep;9(9):1179-1182. doi: 10.15252/emmm.201708203.
In recent years, medical foundations have become increasingly influential, and now play an instrumental and integral role in the research and development of their disease area of interest. While some foundations have directly invested in taking drug candidates to the clinic, others have focused on creating specific tools for accelerating the identification and development of effective treatments. Here, we describe a new model, developed by the Children's Tumor Foundation (CTF), by which foundations may play a role in the rare disease ecosystem. On the one hand, the CTF uses its position to build bridges between academic scientists, biotech and pharmaceutical companies, and patients, to accelerate the development of treatments that really matter most to patients. On the other hand, it acts as a niche investor to fund an integrated platform for critical R&D endeavors—including elements such as a patient registry, biobank, and open data platforms—which smoothen the transition from basic discovery to clinical benefit. Currently, the Children's Tumor Foundation is launching a call to top finance experts to collaborate on building an innovative model that will guarantee long‐term sustainability of this integrated platform (Fig 1).
近年来，医学基金会的影响力与日俱增，在其关注疾病领域的研究和开发中发挥了重要作用。一些基金会直接投资于将候选药物推向临床，而另一些基金会则专注于开发特定工具，以加速有效治疗方法的识别和开发。在这里，我们描述了一种由儿童肿瘤基金会（CTF）开发的新模式，通过该模式，基金会可以在罕见病生态系统中发挥作用。一方面，CTF 利用其地位在学术科学家、生物技术和制药公司以及患者之间架起桥梁，以加速开发对患者真正重要的治疗方法。另一方面，它充当利基投资者，为关键研发工作提供综合平台的资金支持，包括患者登记处、生物库和开放数据平台等要素，从而促进从基础发现到临床获益的顺利过渡。目前，儿童肿瘤基金会正在呼吁顶级金融专家合作，以建立一种创新模式，保证这个综合平台的长期可持续性（图 1）。
