Q and A with Dr Christopher P Austin, NCATS director

March / April 2013 | Volume 12, Issue 2

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.

In what other ways does NCATS support global health research?

NCATS' Clinical and Translational Science Awards (CTSA) program, which supports a consortium of more than 60 academic institutions nationwide, is focused principally on the clinical and implementation stages of translational research, including areas critical to global health. For example, a team at the University of California, San Francisco (UCSF), is working to track and treat tuberculosis in developing countries by applying implementation science techniques from a CTSA-supported clinical research training program. Focused on translating findings from the clinic into real-world tuberculosis interventions, the team is testing several approaches in Ugandan health centers to improve the quality of treatment and using a cell phone-based LED microscope in Vietnam to decentralize diagnosis. The lead investigator learned broad translational skills through a CTSA-supported career development award and mentoring.

In addition, the NIH Office of Rare Diseases Research (ORDR), now housed at NCATS, was instrumental in starting the International Rare Diseases Research Consortium, which began as a joint effort between the European Commission and NIH to link global rare disease communities. By 2020, this consortium aims to develop 200 new therapies and diagnostic tests for most rare diseases. And, through ORDR's Rare Diseases Clinical Research Network, scientific collaborations continue with international sites in Australia, Canada, Europe, Iceland and India. ORDR also is connected with European and Asian countries to develop components of the Global Rare Diseases Registry and Data Repository, and ORDR staff from the Genetic and Rare Diseases Information Center respond to inquiries from patients, families, and providers around the globe.

NCATS will continue to evolve over the next several years to address the enormous needs and opportunities in translational science. We look forward to partnering with Fogarty to make that vision a reality for global health.

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