UCSF

Theme 1: Research—2015 Original

Note: This is an archived version of the original Leading Change: Strategic Plan 2015–2020.

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Theme 1 in Leading Change: Strategic Plan 2015–2020 is:

Research: driving the development of innovative and precise drugs, medical devices, and diagnostic tests

Goals

  1. Uncover the deep biology of health and disease
  2. Devise new computational approaches toward the understanding of biology and disease
  3. Use genomic data to decipher disease and predict drug response
  4. Create the next generation of enabling technologies vital for new discoveries
  5. Amplify our research in bioengineering
  6. Lead nationally in the regulatory sciences
  7. Intensify our health services, economics, and epidemiology research

Goal 1: Uncover the deep biology of health and disease

1.1.1

Develop approaches to treat infectious pathogens … by targeting pathogen enzymes that promote drug resistance or disable host-immune responses.

Driver: William DeGrado, PhD

With: Department of Pharmaceutical Chemistry faculty

Completed by: 2020

1.1.2

Develop potential antibody approaches to treating and detecting cancers and gauging treatment effectiveness … by (a) identifying cell surface proteins that change during oncogene transformation and creating antibodies to these proteins as potential therapeutics, and (b) exploring how these antibodies could be used as potential biomarkers to detect cancers and the effectiveness of anticancer drug treatment.

Driver: James Wells, PhD

With: Department of Pharmaceutical Chemistry faculty

Completed by: 2020

1.1.3

Develop therapeutics for cancer and infectious diseases that function at the level of gene expression (epigenetic therapeutics) … by identifying druggable sites and developing small molecules that control enzymatic protein and nucleic modifications.

Driver: Danica Fujimori, PhD

With: UCSF faculty colleagues

Completed by: 2020

1.1.4

Design hybrid antimalarials that target disease while greatly reducing side effects … by taking advantage of a chemical reaction inside infected cells that allows drugs to be deposited inside only infected cells and not uninfected cells.

Driver: Adam Renslo, PhD

With: Department of Pharmaceutical Chemistry faculty

Completed by: 2020

1.1.5

Develop new drug leads for HIV/AIDS and cancer … by developing new methods to exploit human proteases.

Driver: Charles S. Craik, PhD

With: Department of Pharmaceutical Chemistry faculty

Completed by: 2020

1.1.6

Improve efficacy, compliance, and safety of therapeutics for chronic and acute diseases … by engineering injectable and implantable nanoscale drug delivery platforms.

Driver: Tejal Desai, PhD

With: Department of Bioengineering and Therapeutic Sciences faculty, California Institute for Quantitative Biosciences-UCSF, UCSF Clinical and Translational Science Institute

Completed by: 2018

Goal 2: Devise new computational approaches toward the understanding of biology and disease

1.2.1

Drive forward the application of computation, mathematics, and statistics to better understand large and complex problems in biology associated with disease—with the ultimate goal of developing new therapies … by leveraging the potential of the Quantitative Biosciences Institute (QBI).

Driver: Nevan Krogan, PhD

With: QBI Executive Committee

1.2.2

Create computer models of biomolecular structures and networks that will facilitate a deeper understanding of biology and biomedicine … by developing and applying integrative multi-scale methods.

Driver: Andrej Sali, PhD

With: William DeGrado, PhD; James Fraser, PhD; Kathy Giacomini, PhD; Matt Jacobson, PhD; Tanja Kortemme, PhD; Brian Shoichet, PhD; James Wells, PhD; David Agard, PhD (School of Medicine); Nevan Krogan, PhD (School of Medicine); Robert Stroud, PhD (School of Medicine); Stanley Prusiner, MD (School of Medicine); additional faculty members from Department of Bioengineering and Therapeutic Sciences, Department of Pharmaceutical Chemistry, and Department of Clinical Pharmacy; California Institute for Quantitative Biosciences-UCSF

Completed by: 2020

1.2.3

Devise computational methods to improve the design of drugs for neurodegenerative diseases … by defining and codifying the properties of molecules, allowing them to penetrate and move across the blood–brain barrier.

Driver: Matt Jacobson, PhD

With: Department of Pharmaceutical Chemistry faculty

Completed by: 2020

1.2.4

Create designer molecules that precisely control biological behavior … by developing new technologies that integrate computer models with the perturbations of molecules, cells, tissues, and organisms.

Driver: Tanja Kortemme, PhD

With: Department of Bioengineering and Therapeutic Sciences and Department of Pharmaceutical Chemistry faculties, California Institute for Quantitative Biosciences-UCSF, Precision Medicine Initiative leadership

Completed by: 2020

1.2.5

Bring physical and computational sciences to drug discovery, with a particular focus on drugs that affect communication across cell membranes (g-protein coupled receptors) and are targets for respiration and the relief of pain, hypertension, and depression … by developing new agents for drug targets and new targets for drugs.

Driver: Brian Shoichet, PhD

With: Department of Pharmaceutical Chemistry faculty

Completed by: 2020

1.2.6

Evaluate distant relationships between protein structure and function … by developing new computational approaches to protein bioinformatics.

Driver: Patricia Babbitt, PhD

With: Department of Bioengineering and Therapeutic Sciences and Department of Pharmaceutical Chemistry faculties

Completed by: 2018

Goal 3: Use genomic data to decipher disease and predict drug response

1.3.1

Understand the role of gene regulatory sequences in human disease, drug response, and evolution … by applying genomic technologies, mouse and fish genetic engineering, human patient samples, regulatory element analysis, and development of massively parallel reporter assays.

Driver: Nadav Ahituv, PhD

With: Department of Bioengineering and Therapeutic Sciences faculty

Completed by: 2018

1.3.2

Uncover genetic mechanisms underlying host-pathogen interactions and differences in drug response … by leveraging the theory-rich field of population genetics and the data-rich field of human genetics.

Driver: Ryan Hernandez, PhD

With: Department of Bioengineering and Therapeutic Sciences faculty

Completed by: 2018

1.3.3

Optimize a big data interpretive platform … by developing computational methods and integrated databases that predict drug action using multi-tiered datasets, and by developing computational methods to enable data-driven prescribing of drugs.

Drivers: Esteban G. Burchard, MD, MPH; Kathy Giacomini, PhD; Ryan Hernandez, PhD; Deanna Kroetz, PhD; Rada Savic, PhD

With: Department of Bioengineering and Therapeutic Sciences and Department of Pharmaceutical Chemistry faculties

Completed by: 2020

Goal 4: Create the next generation of enabling technologies vital for new discoveries

1.4.1

Rapidly build 3D human tissues for basic research, regenerative medicine, and the study of cancer … by developing next-generation strategies for precise tissue fabrication from primary tissue or renewable cell stocks.

Driver: Zev Gartner, PhD

With: UCSF faculty colleagues

Completed by: 2020

1.4.2

Develop new enabling tools and technologies in molecular, cellular, and tissue engineering; high-content cellular imaging; large-scale mapping of intracellular and interorganismal interactions; and super-resolution microscopy … by partnering with foundations, industry, and the California Institute for Quantitative Biosciences-UCSF.

Drivers: Adam Abate, PhD; Tejal Desai, PhD; Bo Huang, PhD

With: Department of Bioengineering and Therapeutic Sciences and Department of Pharmaceutical Chemistry faculties; California Institute for Quantitative Biosciences-UCSF; UCSF Office of Innovation, Technology, and Alliances

Completed by: 2020

1.4.3

Develop the next generation of biomedical technology … by establishing a “collaboratory” for medical device innovation that will facilitate interactions and prototype development among clinicians, scientists, and engineers.

Drivers: Tejal Desai, PhD; Shuvo Roy, PhD; Hanmin Lee, MD (School of Medicine); Joseph DeRisi, PhD (School of Medicine)

With: Department of Bioengineering and Therapeutic Sciences, Department of Surgery, and Department of Biochemistry faculties; California Institute for Quantitative Biosciences-UCSF

Completed by: 2020

1.4.4

Create platforms for high-throughput screening, directed evolution, and DNA sequencing … by developing microfluidic approaches and droplet-based microfluidics.

Driver: Adam Abate, PhD

With: Department of Bioengineering and Therapeutic Sciences faculty

Completed by: 2017

Goal 5: Amplify our research in bioengineering

1.5.1

Expand our work in therapeutic bioengineering (medical devices and diagnostics) … by developing sustainable research programs that enable the discovery of biomarkers used to identify and monitor pathological conditions.

Drivers: Tejal Desai, PhD; Shuvo Roy, PhD

With: Department of Bioengineering and Therapeutic Sciences and Department of Pharmaceutical Chemistry faculties

Completed by: 2020

1.5.2

Use bioengineering to improve the precise diagnosis and detection of disease and the monitoring of treatments … by collaborating across disciplines with engineers, clinicians, scientists, and industry partners.

Drivers: Tejal Desai, PhD; Shuvo Roy, PhD

With: Department of Bioengineering and Therapeutic Sciences faculty

Completed by: 2020

1.5.3

Develop a bioartificial kidney for the treatment of end stage renal disease (ESRD) patients … by combining ultrafiltration with cell therapy, resulting in a safer, more effective alternative to traditional dialysis.

Drivers: Tejal Desai, PhD; Shuvo Roy, PhD

With: Department of Surgery and Division of Nephrology faculties; William Fissell, MD (Vanderbilt University); H. David Humes, MD (University of Michigan); U.S. Food and Drug Administration; industry partners

Completed by: 2020

Goal 6: Lead nationally in the regulatory sciences

1.6.1

Fully establish a robust Center of Excellence in Regulatory Science and Innovation (CERSI) with Stanford University and the U.S. Food and Drug Administration (FDA) … by successfully competing for a three-year FDA CERSI grant; launching the center; establishing and implementing a strong center roadmap that includes education, research, and outreach programs in the regulatory sciences.

Driver: Kathy Giacomini, PhD

With: Russ Altman, PhD (Stanford University); U.S. Food and Drug Administration; industry partners

Completed by: 2018

Goal 7: Intensify our health services, economics, and epidemiology research

1.7.1

Assess how tobacco control influences public health … by evaluating changes in policies and tobacco industry marketing strategies, including the development and regulation of new products such as electronic nicotine delivery systems (e.g., e-cigarettes).

Driver: Dorie Apollonio, PhD

Completed by: 2020

1.7.2

Evaluate the economics of disease treatments … by using state-of-the-art comparative-effectiveness and cost-effectiveness analyses of new technologies, drugs, diagnostics, devices, and new practice methods for disease treatment.

Driver: Leslie Wilson, PhD

With: James Lightwood, PhD; World Health Organization (WHO) Collaborating Centre for Pharmaceutical Research and Science Policy

Completed by: 2020

1.7.3

Make precision medicine accessible … by building trans-disciplinary and cross-sector research that evaluates the impact of precision medicine on clinical care, health economics, and health policy.

Driver: Kathryn Phillips, PhD

With: Bani Tamraz, PharmD, PhD; UCSF Clinical and Translational Science Institute

Completed by: 2020


Back to current: Theme 1: Research