The pharmaceutical industry is at a critical juncture

Four decades of molecular biology and genomics have fundamentally advanced our understanding of human disease, but this huge effort has had far less impact on therapy than promised, largely because turning genomic and biochemical data into treatments is slow and difficult. Without a radical improvement in efficiency, the industry is almost certain to shrink, even as the needs of an aging population grow.

The missing link between basic science and new drugs is pharmacology, the study of drug actions

The Harvard Medical School (HMS) Laboratory of Systems Pharmacology (LSP) is a  multi-disciplinary effort within HiTS to reinvent the fundamental science underlying the development of new medicines and their use in individual patients.

Every cell is an extensively interconnected ecosystem. Any given gene may influence dozens or hundreds of other genes. Systems Pharmacology investigates drug action at the level of entire biochemical and genetic networks, rather than looking at the effect of any single element.

The LSP  brings together investigators in mathematical and experimental disciplines from multiple academic institutions (Harvard, MIT, Tufts) and research hospitals (Dana Farber, MGH, BWH) and eventually visiting scientists from the FDA and local drug companies to integrate computational and systems approaches into all phases of drug discovery and development.  READ MORE at Harvard Magazine about Director Peter Sorger and his approach to improving drug development.

At the LSP, we are modeling complex systems to predict drug action

Scientists already have robust tools for quantitatively analyzing biological characteristics such as genome sequence, gene expression, metabolism or cell proliferation.  Researchers at the LSP are now collecting these data at a massive scale and using mathematical and computational approaches to thread these lines of evidence together into sophisticated models. These models will in turn offer a richer picture of disease biology, and inform the development of medicines that deliver greater therapeutic benefit with fewer safety concerns.

Putting this model to work: the LSP Safety Science project

Safety Medicine is a major LSP initiative that looks to improve toxicity screening across the entire biotech/pharmaceutical industry.

Every year, hundreds of promising drugs fail in trials because they produce side effects that can cause significant harm to the human liver, kidneys, or other vital organs.  Although each of these drugs may be targeting a different disease, their harmful side effects follow common pathways.

The goal of our Safety Science (toxicology) project is to discover these common pathways and to develop human cell-based assays that can quickly identify toxic chemical compounds that will always harm the human body–no matter the purpose of the drug.  By testing drugs early on, researchers would be able to eliminate harmful compounds and turn to investigating alternatives, thus saving time and money while developing more effective therapies.