In 2012, the National Institute of General Medical Sciences (NIGMS) achieved a momentous feat, turning 50. While the Institute has much to celebrate, it has never been more committed to serving its role as NIH's "basic research institute." NIGMS plans to maintain its time-tested strategy of long-term, stable research support for highly creative scientists committed to building a broad and deep foundation of discovery. Through NIGMS-funded research, the spark of individual bright minds produces knowledge and therapies that are saving the lives of people in every corner of America every day.
Investing in Basic Research
A crucial outcome of basic research is knowledge—today's currency of global competitiveness. NIGMS-funded researchers working in various areas of science have shattered paradigms about what we thought we knew about basic molecules and cells, and this information has expanded our understanding with regard to health and disease. For example, natural sugar molecules (glycans) are critical for defining cell and organ function, but much about these key molecules remains a mystery (see Program Portrait). We also know that RNA, DNA's molecular cousin, plays a starring—not simply a supporting—role in a wide range of biological functions. Clarifying the structure and function of glycans and RNA remains a priority for NIGMS research support, as scientists continue to explore the activities of these basic ingredients of our cells in cancer, development, infections, and other health-related processes.
Animal models of disease—laboratory mice in particular—are valuable tools for biomedical discovery. However, researchers have recently confirmed laboratory mice do not represent the diversity of either wild mice or humans, because they are bred from a very small gene pool. This limits the ability of scientists to connect mouse-related findings to human health. NIGMS is supporting the Collaborative Cross, a research effort that aims to vastly increase the diversity of mouse data available for study. The project has generated an online database of mouse genetic variation that represents approximately four times the genetic variation of the world's human populations and is poised to facilitate research more applicable to human diseases.
Accelerating Discovery Through Technology
Created in 2000, the Protein Structure Initiative (PSI) is a federal, university, and industry effort aimed at dramatically reducing the costs and lessening the time it takes to determine a three-dimensional protein structure. The PSI remains a highly successful NIGMS program that has accelerated discovery through technology. Its most recent iteration, PSI:Biology, began in 2010 to harvest the fruits of earlier methods-based development work to address significant problems in biomedicine. A key component of PSI:Biology is a publicly accessible repository that provides PSI-generated biological resources to researchers everywhere, significantly catalyzing discovery in various areas of health and disease. The Biomedical Technology Research Centers are another example of the synergistic interaction of technical and biomedical expertise. These Centers promote the widespread and routine application of cutting-edge imaging and computer-based technologies across the full spectrum from bench to bedside.
NIGMS continues to invest in computer modeling approaches for various health-related applications, such as understanding and preventing disease outbreaks. In recent work, scientists with the Models of Infectious Disease Agent Study (MIDAS) investigated the cholera outbreak that occurred after the earthquake in Haiti. The models revealed that, had a large stockpile of oral cholera vaccine been available, and given to the Haitians most at risk after the earthquake, cholera-induced illness and death could have been slashed by about half. As this study clearly demonstrates, MIDAS research findings continue to have significant policy implications for managing public health crises that can emerge without warning.
Advancing Translational Sciences
Developing a single new medicine from concept to consumer is an extremely costly and time-consuming process—often taking more than a decade and up to $1 billion. Approaches that can save time and costs could accelerate this process. In recent work, NIGMS-funded scientists searched through public databases of medical research information to see how 100 diseases affected the activity of thousands of genes. This enabled them to develop various genetic "signatures" that they subsequently matched to approved drugs that were known to affect the same genes. This study suggested that an epilepsy drug may work better than existing treatments for inflammatory bowel disease, and an anti-ulcer medication might be useful for lung cancer treatment. While the computer results were confirmed in animal studies, further analyses will require testing in humans.
In another example of advancing basic science toward patient care, a group of scientists created a family-wide genetic risk report. Their findings, which build on a 2010 study1 that reported the first whole-genome medical risk assessment for an individual, uncovered an inherited tendency for blood clots. The new information enables family members to pursue lifestyle changes and medications to prevent future illness. This essential proof-of-principle study lays important groundwork for future studies that will enable disease risk prediction more broadly.
Encouraging New Investigators and New Ideas
In April 2011, NIGMS issued Investing in the Future, a strategic plan for its biomedical and behavioral research training programs. In implementing the plan, NIGMS strongly encourages high-quality mentorship and recognizes the diversity of successful careers open to well-trained scientists. The Institute has recently established a new organizational component, the Division of Training, Workforce Development, and Diversity, which integrates training and diversity activities across Institute programs and oversees the Institutional Development Award (IDeA) program that broadens the geographic distribution of NIH funding.