Christopher P. Austin, M.D., became director of the National Center for Advancing Translational Sciences (NCATS), NIH’s newest component, in September 2012. Launched in December 2011, NCATS aims to transform the process by which human health interventions are developed and delivered, making it more efficient, productive and scientifically robust. Austin applies his experiences in academia, industry and government – including work in rural hospitals in Africa and Alaska – to lead NCATS.
What is your vision for NCATS?
NCATS will work with collaborators and stakeholders to transform translation from a largely empirical, trial-and-error process to a more science-based, predictive enterprise. We will focus on solving problems that are common to the translational process for all diseases, and so tend not to be a focus for others. Examples include developing models to help more accurately predict efficacy and toxicology, repurposing drugs, providing starting points for novel targets and untreatable diseases, improving clinical trial recruitment and endpoint criteria and Institutional Review Board harmonization.
Since translation is a team sport, NCATS will serve as a catalyst, connector and adaptor to complement – not compete with – the work of the other NIH Institutes and Centers, industry and the nonprofit sector.
How do collaborations advance NCATS’ global health research?
In NCATS’ preclinical programs, each project is a collaboration with an academic, industry or nonprofit researcher. The deliverable may be a novel target, a probe to test a therapeutic hypothesis, or an early-stage drug. For example, NCATS’ NIH Chemical Genomics Center (NCGC) has collaborated on the identification and development of early stage compounds to treat many tropical diseases, including Chagas disease, schistosomiasis, giardia, malaria, HIV and hepatitis C. These diseases affect millions globally. NCGC enables researchers who are experts in global health problems but not necessarily equipped or experienced in translation to access the industrial-scale assay development, small molecule and RNAi screening, informatics, and medicinal chemistry necessary to identify chemical probes and drug leads.
Collaborative projects in our Therapeutics for Rare and Neglected Diseases (TRND) and Bridging Interventional Development Gaps (BrIDGs) programs address the next stages of translation, aiming to deliver new interventions for testing in first-in-human trials. Current TRND projects include new, more potent treatments for cryptococcal meningitis, an infectious disease leading to hundreds of thousands of HIV-related deaths in sub-Saharan Africa each year, as well as the first drug candidate to target directly the molecular cause of sickle cell disease, a genetic disorder affecting millions worldwide. Through TRND, we also took on two projects targeting different biological pathways to treat schistosomiasis. Although these projects now have been discontinued based on preset project milestones, both advanced knowledge in schistosomiasis disease research.