The field of biological research that focuses on the science of small molecules, called chemical biology, is relatively new. Its coemergence with modern human genetics is leading to enormous opportunities for synergies between these fields. In addition to information on genotype, or the inherited instructions carried by a human or other organism, modern human genetics is shining a bright light on the phenotype of diseases—that is, the change in morphology, development, or behavior of an organism as a result of disease. With chemical biology, we can provide powerful methods for studying phenotypes of disease and making sense of what we find. That's where we can provide valuable information on likely targets to pharmaceutical companies.

Under a new chemical biology program called the Novel Therapeutics Initiative, the Broad Institute is further refining our thinking on effective drug discovery processes. Rather than trying to convince the pharmaceutical industry that an experimental lab technique is useful, we prefer to demonstrate the capabilities of our overall process in the context of especially challenging diseases, such as schizophrenia, diabetes, and cancers. If we can find highly unusual, highly effective therapeutic agents for these diseases that could not have been discovered otherwise, the pharmaceutical industry likely will pay attention. We're encouraged by the outcomes with mTOR and HDAC inhibitors.

We don't expect the pharmaceutical industry, which has spent millions of dollars and decades on its current drug discovery approaches, to slow down their process and switch to a new one. My hope is that applying chemical biology research and methods to drug discovery may serve as a way for the academic community to broaden its ties with the pharmaceutical industry, and demonstrate a path forward for developing better and safer drugs.

The head of the chemical biology program at the Broad Institute, Stuart Schreiber received his B.A. in chemistry from the University of Virginia and his Ph.D. in organic chemistry from Harvard University.

Interview by Lori Valigra

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