Small molecules, often with molecular weights of 500 or below, have proven to be extremely important to researchers to explore function at the molecular, cellular, and in vivo level. Such molecules have also been proven to be 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.

The Molecular Libraries Roadmap, through one of its components, MLPCN, offers biomedical researchers access to the large-scale screening capacity, along with medicinal chemistry and informatics necessary to identify 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 that will enable researchers in the public and private sectors to validate new drug targets, which could then move into the drug-development pipeline. This is particularly true for rare diseases, which may not be attractive for development by the private sector.

Glossary

You can find a glossary of terms used in MLP here.

Screening Resources

The capacity for High Throughput Screening 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. As these HTS Technologies were not previously available to the public sector, many investigators may not be familiar with the components and requirements of high throughput screening.