The New Vision of Drug Discovery and Development

Given the challenges within the pharmaceutical industry and the need for innovation in the industry, it was clear to us that an academic drug discovery institute should explore new approaches to the discovery of novel therapeutics, in addition to supporting more traditional approaches requested by faculty collaborators.  Our strategy has been to harness the main strengths in Pittsburgh that include outstanding computational and systems biology at both the University of Pittsburgh (Pitt) and Carnegie Mellon University (CMU), an NIH-funded Big-Data-To-Knowledge  (BD2K) Center joint between Pitt, CMU, the University of Pittsburgh Medical Center (UPMC), and the Pittsburgh Supercomputing Center (PSC),  a new initiative between Pitt, CMU and UPMC named the Pittsburgh Healthcare Data Alliance created to dramatically change the practice of medicine through medical informatics, as well as strong therapeutic area researchers and clinicians with access to crucial patient samples.  These strengths encouraged us to pursue the emerging field of quantitative systems pharmacology (QSP) as a central theme.


The UPDDI has three major classes of activities: a) integrated programs that apply QSP to metastatic cancer, Huntington’s Disease (as a first focus area to be extended  to other neurodegenerative diseases) and liver diseases; b) projects based on ligand- or target-centric discovery that are driven by faculty collaborators/ RO1 grants covering many therapeutic areas; and c) translational technology development including companion diagnostics, computational biology/chemistry tools, analytics to identify and quantify heterogeneity in experimental and clinical samples and advanced human tissue models for discovery.  The UPDDI is positioned in an outstanding environment to translate key scientific and clinical advances into novel therapeutics.  The Pittsburgh Cancer Institute, the Brain Institute along with the Pittsburgh Institute for Neurodegenerative Diseases and the historically strong research and clinical efforts in liver diseases are key elements of our integrated program strategy.


The University of Pittsburgh (Pitt) has the breadth and depth to support a major effort in drug discovery and development and in cooperation with the University of Pittsburgh Medical Center (UPMC) and Carnegie Mellon University (CMU), the ability to translate basic research to clinical practice. The UPDDI has created a centralized facility and core staff with a combination of academic and industrial experience, to help collaborators translate outstanding basic science into drug discovery programs. Additionally, many of the specific functions of the drug discovery and development process are distributed across the Pitt and UPMC campuses in distinct departments, centers and institutes; especially in the Schools of Medicine and Pharmacy, as well as the Colleges of Arts and Sciences and Engineering.


The UPDDI is committing major efforts in "Quantitative Systems Pharmacology"(QSP) in addition to traditional drug discovery and development methods.

Quantitative Systems Pharmacology (QSP): Combination of computational and experimental methods to elucidate, design, validate and apply new pharmacological concepts and strategies to the development and use of therapeutics and diagnostics. QSP provides an integrated "systems" approach assisted by high-content screening techniques to determining mechanisms of action during discovery, development and in patients. QSP will also create the knowledge required to alter complex cellular networks with single or combination therapy, as well as to alter the pathophysiology of disease in order to optimize the therapeutic benefit, while minimizing toxicity.

Chemistry and Molecular Biophysics: Design and synthesis of novel, drug-like chemical scaffolds, with the associated medicinal chemistry, as well as the development of peptidomimetics, Chinese herbal medicines and biologics to address the therapeutic needs. In addition, application of computational chemistry, chemical proteomics and molecular biophysics methods to predict and to define target proteins, druggable sites, and potential binding affinities, and to facilitate structure-based characterization, design and/or engineering of optimal drug-target interactions.

Therapeutic Areas

The UPDDI accepts collaborative projects in any therapeutic area where the collaborator has a significant interest and commitment to the program, while focusing internal efforts on cancer, neurodegenerative diseases and liver diseases. There is a major commitment to the use of experimental animals (e.g., yeast, C. elegans, Drosophila, and zebrafish), as well as human-derived cell/engineered tissue models early in the discovery process.