UCSF

Goal 1: Create a new framework for drug discovery and development

Amplify and integrate our expertise in chemistry, physics, and math to more deeply understand health and disease

Advance our understanding of protein structure and function; discover failures in biological systems that are associated with complex diseases; deploy the power of computation to make sense of vast quantities of biological and pharmaceutical data

Milestones

  • Recruited and hired new faculty physicists (Adam Abate, PhD; Xiaokun Shu, PhD) and new faculty members with chemistry expertise (Michael Fischbach, PhD; William Degrado, PhD; Zev Gartner, PhD; Bo Huang, PhD; Danica Fujimori, PhD) to amplify our expertise in the physical sciences.
  • Hired faculty members with mathematical research programs (Ryan Hernandez, PhD, whose research is in computational genomics; Rada Savic, PhD, whose research is in pharmacometrics).
  • Let the formation of the Quantitative Biosciences Consortium to facilitate PhD training in the quantitative biosciences at UCSF. This umbrella graduate group involves five major graduate programs: Bioengineering, Biological and Medical Informatics, Biophysics, Chemistry and Chemical Biology, and Pharmaceutical Sciences and Pharmacogenomics.

Create new research tools

Apply nanoscience to develop tools and devices to diagnose and treat diseases; develop small molecules to probe biological processes and serve as therapeutic agents; develop advanced computational tools that will transform understanding of living systems; use physical methods to understand normal and pathological biological processes at the molecular level

Milestones

  • Created the Small Molecule Discovery Center to assist UC researchers in the identification of small molecules that modulate biochemical or cellular processes and have the potential to alter disease states.
  • Was selected to lead a UCSF research collaboration under the National Cancer Institute to use small molecules to "search" the surfaces of cells for possible new "druggable" sites. The goal is to develop whole new classes of drugs to target cancer.
  • Chosen as one of three renal device projects nationwide to pilot a U.S. Food and Drug Administration (FDA) regulatory approval program called Innovation Pathway 2.0. The Kidney Project at UCSF is creating an implantable bioartificial kidney under the national leadership of Shuvo Roy, PhD, Department of Bioengineering and Therapeutic Sciences. The program will involve close contact among the FDA and device developers early in the development process, in order to identify and address potential scientific and regulatory hurdles and create a roadmap for project approval. The goal is to improve the projects' overall chance of success and maintain safety, while reducing the time and cost of FDA review.
  • Created the Biomedical Microdevices Laboratory to apply microelectromechanical systems (the microelectronics, microfabrication, and micromachining technologies known collectively as MEMS) and associated nanotechnologies to clinical applications such as surgical instruments, tissue repair, artificial organs, diagnostic tools, and drug delivery systems.
  • Created the Therapeutic Micro and Nanotechnology Laboratory for the design, fabrication, and use of advanced micro/nano biosystems for cellular integration and tissue engineering, biomimetic architectures for functional biomaterials, and therapeutic drug targeting and delivery.
  • Created a suite of computational tools that can be licensed (or are freely available) and used in drug discovery programs. These tools include a new version of DOCK and SEA (Similarity Ensemble Approach) from the laboratory of Brian Shoichet, PhD; Modeller and ModBase from the laboratory of Andrej Sali, PhD; and MutInf from the laboratory of Matt Jacobson, PhD.
  • Through a grant from the National Institutes of Health, began building the UCSF Antibiome Center. The build involves assembling an automated "production factory" that, along with facilities at the Universities of Chicago and Toronto (a consortium dubbed the Recombinant Antibody Network), will make antibodies that selectively bind to all of the approximately 1,500 human transcription factors-proteins that bind to DNA sequences and control the flow of genetic information that generates new proteins inside cells. The Network's antibodies will be a resource for all researchers studying transcription factors and gene regulation. They also may be developed as diagnostic tools and new pharmaceutical agents.
  • Co-led the cross-disciplinary Pediatric Device Consortium to guide the invention of new medical devices for children.
  • Acquired and applied a Thermo-Fisher LTQ Orbitrap Velos mass spectrometer to monitoring and measuring the molecular kaleidoscope of the human proteome.

Find new approaches to therapeutics discovery and delivery

Expand our ability to predict therapeutic effectiveness based on genetic profiles; use chip technology to evaluate how drugs will act in the body; find better ways to deliver drugs and novel therapies to targeted disease sites in the body

Milestones

  • Supported the exploration of promising new research areas, including: high-throughput biology using microfluidics, advanced light microscopy, pharmacometrics, de novo protein design, design of genetically engineered encoded probes for multicolor whole-body imaging, computational genomics, design of cellular networks, application of microelectromechanical systems to the development of therapeutics, enzymatic features responsible for the development of antibiotic resistance, and polypharmacology.
  • Launched the Center for Quantitative Pharmacology to accelerate predictive drug development. Recruited and hired an expert in pharmacometrics (Rada Savic,PhD). Began rebuilding the School's quantitative pharmacology leadership.