Without access to additional data, intellectual property or expertise, an exciting research project can quickly go cold. Last December, the U.S. National Institutes of Health launched its 27th center, the National Center for Advancing Translational Science (NCATS), to revive those cold cases and break down the barriers that impede progress in important biomedical research. BVGH spoke to Dr. Christopher Austin, director of NCATS' Division of Pre-Clinical Innovation, about the importance of translational research in solving the world's toughest health questions and spending the delivery of new drugs, vaccines, and diagnostics to those who need them most.

 

How is translational science different from other types of research and development, and what makes it important?

It is different because it is a science that is fundamentally about making connections across disciplines, as opposed to a science that digs deep in the mechanism of a particular biological phenomenon or disease. There are many different definitions of translational science, but the term literally means to "carry across" a project from one part of the research ecosystem to another. For example, a translational scientist might work to find links between a basic research finding to a diagnostic or therapeutic with initial evidence of safety and efficacy in humans. Or they might find synergies between research predominantly done in public institutions and research done in private ones. "Translation" is also used to describe the science required to move a drug from approval to appropriate use in the general population. The term is also used to describe the science of the translational process itself, from a scientific point of view (e.g., ways to better predict drug toxicity) or a systems point of view (e.g., innovative partnership structures to move projects across the relevant translational space more efficiently).

 

How can translational science help drive the development of new drugs, vaccines, and diagnostics for neglected diseases and other diseases that disproportionately impact the world's poorest?

Neglected diseases present particular opportunities and challenges to translational efforts, for both scientific and organizational reasons. From a scientific point of view, neglected diseases are often not as well studied or understood as diseases of the developed world, so the starting points for diagnostic or therapeutic development can be more precarious and the potential for unanticipated failures can be greater. From an organizational point of view, neglected diseases lack two important features that other diseases often have: organizations or foundations that represent those affected with the disease, raise money for disease research, and collect patient registry data to facilitate research; and anticipated return on investment, which makes the identification of partners to hand the projects off to more limited. The advent of organizations focused on coordinating research between public and private sectors have been enormously helpful with breaking down financial barriers that will then help to reduce the scientific barriers as well. With more data on neglected diseases, we can then focus on making the types of translational science connections that are being made for other "developed world" diseases.

 

Why is both public and private sector participation important in translational science important?

For the most part, scientific research begins in the public sector and ends up in the private sector. Because of this, translational science can only be successful when technological translational science research questions are defined by what both parties see as the roadblocks preventing the efficient movement of projects from basic discovery to product development and patient access.

 

How will NCATS work to break down key barriers to research and development through translational science? 

NCATS will help establish and advance public private partnerships that will speed the delivery of new drugs, diagnostics, and medical devices to patients. Working in concert with non-profit and private sectors, NCATS will develop innovative approaches, policies and methods to reduce, remove or bypass bottlenecks in translational science collaborations. NCATS will study why barriers to collaboration, such as issues around intellectual property, exist and what new models can help overcome them. Additionally, we will analyze successful academic and industry partnerships to help determine if NCATS should borrow or adopt those models of collaboration.

 

WIPO Re:Search -- a consortium of some of the world's leading pharmaceutical companies, academic and nonprofit research organizations -- works to promote translational research and collaborations among private- and public-sector product developers and neglected disease researchers. As a member of WIPO Re:Search, how might WIPO Re:Search help accelerate some of the NIH's and NCATS's projects?

There are many neglected disease projects within NCATS that are relevant to WIPO Re:Search, including within the Therapeutics for Rare and Neglected Disease program (TRND), projects on schistosomiasis and cryptococcal meningitis. We look forward to finding ways to work across sectors and with a variety of collaborators to advance these programs, and develop and deliver translational tools and technologies to those who need them most.