Small molecules, often with molecular weights of 500 or below, are extremely important for researchers to explore function at the molecular, cellular, and in vivo level. Such molecules have also proven valuable for treating diseases, and most medicines marketed today are from this class.
A key challenge is to identify small molecules effective at modulating a given biological process or disease state. Currently, researchers must systematically screen tens or hundreds of thousands of small molecules to find a successful match between a chemical and its target. This process is known as high-throughput screening or HTS. The capacity for HTS has been built within the pharmaceutical and biotechnology sectors for the purposes of drug development over the last ten years, but similar resources have not existed in the public sector.
The Molecular Libraries Program offers public sector biomedical researchers access to the large-scale screening capacity necessary to identify small molecules that can be optimized as chemical probes to study the functions of genes, cells, and biochemical pathways. This will lead to new ways to explore the functions of genes and signaling pathways in health and disease.
NIH anticipates that these projects will also facilitate the development of new drugs, by providing early stage chemical compounds to researchers in the public and private sectors for validation of new drug targets, which could then move into the drug-development pipeline. This model of drug development may be particularly useful for rare diseases and areas of unmet medical need, which may not be attractive for development by the private sector.
Three key technological advances drive NIH's effort to build small molecule libraries. First, the successful completion of the Human Genome Project has provided an enormous amount of human biology to be studied and innumerable potential drug targets to be discovered. Second, developments in chemistry have given researchers in the public sector the ability to rapidly and efficiently synthesize large numbers of related molecules, a capability previously available only to researchers in pharmaceutical and biotechnology companies. Third, advances in robotic technology and informatics now allow scientists to screen hundreds of thousands of compounds in a single day, a screening capacity that is orders of magnitude greater than a decade ago.
The Molecular Libraries Program has three components:
The Molecular Libraries Program also enhances the discovery and availability of small molecules for molecular imaging including imaging of molecules or molecular events in biological systems that span the scale from single cells to whole organisms. Ultimately, it is hoped that this effort will enable personalized profiles of cell and tissue function, which may lead to more individualized approaches to diagnosing and treating disease. By significantly enhancing the support of this emerging field, NIH will ensure that molecular imaging will become a powerful tool for biomedical research and will be a synergistic component of research in molecular medicine that promises landmark improvements in clinical care.
For more information on the Molecular Libraries Program, contact Geoff Spencer, Communications Specialist, National Human Genome Research Institute, (301) 402-0911, email@example.com; or Kate Egan, Chief, Science Writing, Press and Dissemination Branch, Office of Science Policy, Planning and Communications, National Institute of Mental Health, 301-443-4536, firstname.lastname@example.org.