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Theme 1: Research
Theme 1 in Leading Change: Strategic Plan 2015–2022 is:
Research—driving the development of innovative and precise drugs, medical devices, and diagnostic tests
Overcome fundamental challenges in the discovery of new therapeutics to treat disease
Devise new computational approaches toward the understanding of biology and disease
Use genomic data to decipher disease and predict drug response
Create the next generation of enabling technologies vital for new discoveries
Strengthen our health services, economics, and epidemiology research
Progress to 2021, Changes to theme in 2021, Changes to theme in 2017
Progress to 2021
Research has been a centerpiece of our strategic plan progress from bench to bedside. The School has led the nation in NIH funding, among pharmacy schools, for 41 consecutive years. Since 2015, the School has also experienced a 30% increase in NIH funding while maintaining the number of faculty members at 95. Consistent with this increase in funding, School researchers’ work routinely appears in the premier peer-reviewed journals, including Science, Cell, and the New England Journal of Medicine, among others.
Progress toward overcoming challenges in the discovery of new therapeutics (1.1) and devising new computational approaches (1.2) is best exemplified by the School’s creation of the first organized research unit (ORU) at UCSF in many decades, the Quantitative Biosciences Institute (QBI). QBI has secured numerous large center grants since its inception and serves as the hub of quantitative science at UCSF and beyond. During the pandemic, QBI created the QBI Coronavirus Research Group (QCRG), a scientific collaboration between 75 international laboratories that seeks to develop new approaches in the treatment of COVID-19.
Our success using genomic data to better understand disease and treatment (1.3) is epitomized by the work of the Asthma Collaboratory and multiple pharmaceutical science/pharmacogenomics studies performed in the Department Bioengineering and Therapeutic Sciences. Our researchers recently published the genomes of a vast and diverse group of over 50,000 people, enabling the study of how people of different ethnic backgrounds develop various diseases and respond to different drugs.
Creating the next generation of discovery technologies (1.4) is exemplified by countless efforts in the Department of Pharmaceutical Chemistry to make drug discovery a more efficient and precise process. An adaptation of virtual reality technology, developed in the UCSF Resource for Biocomputing, Visualization, and Informatics (RBVI), has enabled researchers to use virtual reality to hunt for potential drug compounds that precisely inhibit cancer-causing processes. Similarly, a collaboration between UCSF, GlaxoSmithKline, and two national laboratories is combining their collective computing resources to accelerate the development of new cancer therapies. Our researchers have also recently released a library of over 250 million drug compounds that can be easily screened for activity against biological targets.
The Department of Bioengineering and Therapeutic Sciences leads the way on the UCSF campus with respect to the goal of amplifying bioengineering research (1.5). The Kidney Project’s artificial kidney was shown to be safe in a large animal model, offering hope to millions with end-stage renal disease, and an injectable biomaterial was developed to guide CAR-T cell cancer therapies to hard-to-reach tumors.
We have made substantial progress in our leadership of the regulatory sciences (1.6) via our creation and continued development of CERSI (UCSF-Stanford Center of Excellence and Innovation), the only regulatory science and innovation center on the West Coast. UCSF-Stanford CERSI hosted its first conference, featuring scientists, clinicians, and representatives from industry and the U.S. Food and Drug Administration (FDA), in 2020, creating unique opportunities to speed the bench-to-bedside process.
The final area of our research theme—health services, economics, and epidemiology (1.7)—has been bolstered by the continued development of the UCSF Medication Outcomes Center (MOC), a unit intended to evaluate the appropriate use of drugs in the health system, and TRANSPERS, a unit devoted to genomics and health policy. Research from TRANSPERS has identified the slow emergence of insurance coverage for genetic testing as a key barrier to its use in health care, highlighting how the field can make itself more accessible to patients and health care providers in the future.
Goal 1: Overcome fundamental challenges in the discovery of new therapeutics to treat disease
Develop new approaches to treat diseases such as cancers, neurodegenerative diseases, and infectious diseases … by inventing strategies for new or highly challenging drug targets in the body that drugs activate or inhibit.
Develop new approaches to treat diseases such as cancers, neurodegenerative diseases, and infectious diseases … by inventing novel ways to intervene and disrupt the course of diseases.
Goal 2: Devise new computational approaches toward the understanding of biology and disease
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).
Continue computing and analyzing models of biomolecular structures and networks that facilitate a deeper understanding of biology and biomedicine … by developing and applying methods for integrative multi-scale modeling and visualization of models.
Devise new approaches to the design of drugs for neurodegenerative diseases … by developing new computational and experimental methods addressing key challenges, such as penetrating the blood-brain barrier.
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.
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.
Evaluate distant relationships between protein structure and function … by developing new computational approaches to protein bioinformatics.
Goal 3: Use genomic data to decipher disease and predict drug response
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.
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.
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.
Goal 4: Create the next generation of enabling technologies vital for new discoveries
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.
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 (QB3-UCSF).
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.
Create platforms for high-throughput screening, directed evolution, and DNA sequencing … by developing microfluidic approaches and droplet-based microfluidics.
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 anti-cancer drug treatment.
Goal 5: Amplify our research in bioengineering
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.
Develop a bioartificial kidney for the treatment of end stage renal disease patients … by combining ultrafiltration with cell therapy, resulting in a safer, more effective alternative to traditional dialysis.
Improve efficacy, compliance, and safety of therapeutics for chronic and acute diseases … by engineering injectable and implantable nanoscale drug delivery platforms.
Goal 6: Lead nationally in the regulatory sciences
Fully establish a robust Center of Excellence in Regulatory Science and Innovation (CERSI) with Stanford University and the U.S. Food and Drug Administration (U.S. 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.
Goal 7: Strengthen our health services, economics, and epidemiology research
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).
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.
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.
Changes to theme in 2021
Added summary School progress to 2021 for this theme
Archived driver and collaborator assignments from objectives
Archived 2017 progress updates from objectives
Updated plan title to reflect extension to 2022
Changes to theme in 2017
Rewrote Goal 1 from Uncover the deep biology of health and disease to Overcome fundamental challenges in the discovery of new therapeutics to treat disease
Condensed original objectives 1.1.1, 1.1.3, 1.1.4, 1.1.5 into two objectives: 1.1.1, 1.1.2; expanded list of drivers
Relocated original 1.1.2 objective to 1.5.3, the last objective of Research Goal 5
Deleted original objective 1.5.1; it is covered elsewhere