Small molecule metabolites produced by living organisms have been used for millennia in treatment of human diseases. Their use is incentivized by their typically local availability, ease of preparation and low costs of production. Given the vast chemical space occupied by the millions of metabolites produced in the living world, they were vigorously explored as a fountainhead of novel drugs in the 1990s. However, challenges such as the metabolites of interest frequently being part of complex extracts, their low abundance, untoward side-effects in high doses, and their origins in complex metabolic pathways have complicated traditional approaches to natural product drug discovery.

In the last decade, technological advances have created a unique opportunity to exploit naturally produced small molecules for drug discovery. Vast improvements in mass spectrometry allow for rapid detection of thousands of metabolites in complex mixtures, while availability of genomes, protein structures, pathway models, and informatic approaches allow for both pathway identification and reconstruction using synthetic biology methods. Novel in vitro technologies, such as organoid platforms and cells-on-chips, enable rapid screening of biological activities of metabolites prior to pre-clinical testing, and advancements in machine learning enable rational drug design from nature-inspired metabolites.

The bacterial, fungal, and plant kingdoms, which provide us with hundreds of our current antibiotics and other drugs, each produce hundreds of thousands of compounds, many of which are already known to have bioactive properties. Nonetheless, given a relative lack of communication between biochemists who identify such compounds, biomedical scientists who assay their molecular action, efficacy, and toxicity, and engineers who have the means to devise production systems of scale, these organismal chemical pools have been insufficiently explored for their drug potential. For example, several faculty at Cornell have identified biomedically relevant compounds but lack the means to test their activities, assess toxicities using mouse and/or human drug models and convert basic biological knowledge to marketable drugs.

Our aim in organizing this symposium is to bring together research communities with allied interests in discovering novel drugs from complex natural extracts. By assembling scientists from the NYC and Ithaca campuses into one venue, we will initiate discussions that can lead to new research collaborations, training grants, center grants and commercial startups. Thereby, this initiative will exploit unprecedented opportunities to use plants and other organisms as a resource for discovery of new medicines.