Following a poster session, Christopher P. Austin, M.D., director of the National Center for Advancing Translational Sciences (NCATS), spoke to attendees on the role of NCATS in catalyzing translational innovation. “Translation is a team sport,” Austin said in his presentation, which stressed the need for collaboration, from both programs within the National Institutes of Health and those in external institutions, in order to speed the development of new treatments.
Austin’s role at NCATS brings together his work as a neurologist and his position at Merck, where he observed a team-based approach and a culture of deliverables in discovering novel targets and drugs but perceived a lack of deep biology to support research. The fact that people are unhealthier and that funders of research – both public and private – are impatient, points to a need to improve the translation of discoveries in the lab to benefit human health.
The number of new drugs approved by the FDA per billion dollars spent on research and development has halved roughly every nine years since 1950, Austin said. NCATS’ mission is to bring together methods, technologies, and tools to tangibly improve human health across a wide range of diseases. Examples of translational problems requiring a team approach include predictive toxicology; the efficacy of data interoperability; clinical trial networks; EHRs for research; and adaptive clinical trial designs.
NCATS seeks to fulfill its mission through the CTSA program, the Rare Disease Research and Therapeutics program, technology programs to re-engineer translational science, innovative training programs, the development of a robust academic discipline of translational research, and new models for engaging and partnering with communities. “NCATS has just begun to transform itself and its programs to benefit patients,” said Austin.
Richard Flavell, Ph.D., F.R.S., founding chair of the Department of Immunobiology, presented on the role of inflammasomes in health, microbial imbalance, and disease. Flavell explained research conducted by immunobiologists at Yale and other institutions that elucidates how altered microbiota in the gut is implicated in the pathogenesis of diabetes, rheumatoid arthritis, obesity, inflammation-induced cancer, and other diseases. This research sheds light on how diet, gut bacteria, and inflammation interact with one’s genotype in a cycle that leads to a wide variety of systemic diseases.
• Ellen Hoffman, M.D., associate research scientist in the Yale Child Study Center and a trainee in the Investigative Medicine Program, who presented her research on zebrafish to examine neuropathways in autism. She is using a new genetic engineering technology to knock out newly discovered autism genes and small-molecule screening to identify common pathways and relevant drugs.
• Serene Chen, a student at Yale School of Medicine in the TL1 program, who spent a year working on Variation in Recovery: Role of Gender on Outcomes of Young AMI Patients (VIRGO). VIRGO is a study examining heart disease in women aged 55 and under, a group that has about twice the risk of dying from a heart attack than men of similar age.
• Meagan Moore, M.D., Ph.D., postdoctoral fellow in internal medicine (pulmonary and critical care), who presented research on the role of the protein receptor Plexin C1 in pulmonary fibrosis—a disease that is both difficult to treat and unpredictable.
In his closing remarks, YCCI director Robert S. Sherwin, M.D., noted that YCCI’s success in training scientists capable of bridging the gap between basic science and clinical care is illustrated by the results of its educational programs. The 88 YCCI Scholars who have been awarded research and salary support since the program’s inception in 2006 have received $157 million in independent funding and published over 1,000 papers. Furthermore, about 90 percent of YCCI Scholars and Investigative Medicine alumni have remained in academic medicine.