Small probes and their bigger implications

Human genetics studies provide insight into how an ideal drug might target and modify a disease-causing protein. But, oftentimes, these “experiments of nature” indicate the need for drugs that act through novel mechanisms. A review by Broad Institute researchers Stuart Schreiber, Joanne Kotz, and colleagues published in Cell outlines recently completed, decade-long Molecular Libraries Program (MLP) — an NIH effort to identify small-molecule probes that could help explore human biology. From malaria to multiple sclerosis and diabetes, the authors describe how these probes are enabling the investigation of disease hypotheses that would be difficult to explore via other approaches, advancing therapeutic discovery.