EMBO Molecular Medicine Highlights CTF’s Innovative Research Model

Aug 7, 2017, Posted in Latest News, Published, Science

The August issue of EMBO Molecular Medicine features an article authored by Children’s Tumor Foundation President and Chief Scientific Officer Annette Bakker, PhD, and Salvatore La Rosa, PhD, Children’s Tumor Foundation VP, Research & Development about the larger role foundations are playing within the rare disease research ecosystem. EMBO Molecular Medicine is a peer-reviewed, online open access journal dedicated to a new research discipline at the interface between clinical research and basic biology. The journal is read by molecular biologists, clinicians and medical doctors.

 

Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem

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.

To continue reading, click here for the online version or download the full article.