From
Health Care and Biotech in the Bay Area, a publication of the San Francisco Chamber of Commerce
The pulse of translational research is quickening throughout UCSF. Among the numerous endeavors under way are several that represent different disease areas and tactics.
The California Institute for Quantitative Biomedical Research (QB3) is one of four institutes established by the State of California to catalyze collaborations among scientists at different UC campuses and those in the state's technology industries. QB3 is the only such institute devoted to biomedical research. It brings together the powerful quantitative tools of the physical sciences, engineering and mathematics to tackle complex biological problems critical to advancing human health. Its very mission is the translation of basic research to innovative patient treatments. Development of new light microscopes that reach resolutions previously thought to be physically impossible is underway at the Institutes, as are such projects as combining MRI imaging and spectroscopic analyses to allow precise mapping of chemical activity in tumors; and developing a systematic program to predict the folded structure of proteins and map all possible interactions between proteins and small molecules to speed the discovery of new drugs with lower side effects.
The Cardiovascular Research Institute is also moving in the direction of translational research, as exemplified in the work of John Kane, MD, PhD, and colleagues, who are searching for genes associated with such disorders as diabetes, arteriosclerosis and hypertension. The discovery of these genes will allow scientists to identify those people most at risk for the diseases, allowing for preventive measures in the appropriate individuals. It also will enable scientists to examine the molecular mechanisms of these diseases, which could potentially lead to new therapies. The team has identified 21 genes associated with myocardial infarction, or heart attack, and four in association with stroke. These genes are revealing previously unknown mechanisms of disease. Because of the velocity of studies in progress, it is likely that comprehensive applied genomic analysis will enter clinical use within five years.
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Josepth DeRisi |
Translational research is also exemplified in the work of Josepth DeRisi, PhD, whose ingenuity and collaborative spirit have made him a transformative force within UCSF. DeRisi designs and builds microarrays, tools in which gene activity - within viruses, bacteria, parasites and other biological samples - is revealed on a specially prepared glass slide. The chip draws on computer-chip technology, computation and bioinformatics. He is using the technology to make major advances in understanding such infectious diseases as SARS (severe acute respiratory syndrome) and malaria. He has also reached out to clinical researchers throughout UCSF to collaborate on the use of the microarray to explore the possible role of viruses in cancers, asthma and a host of other diseases. DeRisi's driving passion is eradicating the parasite that causes malaria. His team has made significant advances, including identifying the genetic activity of the third and critical stage in the parasite's life cycle. The discovery points to numerous new potential targets for drug and vaccine therapy.
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James McKerrow |
James McKerrow, PhD, is also focused on diseases of the developing world, and his work, as much as that of any scientist at UCSF today, illustrates the power of translational research. McKerrow is focused on microscopic worms and other parasites that kill or disfigure millions of people in the tropics every year. The pharmaceutical industry has not invested adequately in research on drugs for these diseases, so McKerrow has taken on the challenge himself, spearheading an unusual research and development effort within UCSF and with outside contacts to discover the parasites' vulnerabilities, identify drug candidates to kill them and test the drugs in clinical trials. The strategy is working. Based on his team's discoveries, Phase 1 clinical trials are under way in conjunction with OneWorldHealth, a nonprofit, to treat Chagas' disease, the leading cause of heart disease in Latin America, and a second is in development to treat Leishmaniasis, which infects 12 million and causes disabling lesions. McKerrow's commitment has led to support from a local, idealistic philanthropic group - the Sandler Family Supporting Foundation. His progress has since led them to fund a new center for basic research in parasitic diseases.
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Michael Cabana |
Pediatrician Michael Cabana, MD, MPH, is taking his research to the clinic. Cabana is leading a large, HIH-funded trial aimed at determining if "good bacteria," or "probiotics," normally found in the human intestine and many common foods (including yogurt) can be used to prevent the development of asthma in children. The study explores the theory that, in certain children, if the immune system is not stimulated in a certain way and at a certain time, the children will develop the disease. Scientists believe probiotic bacteria may be a safe and feasible way to stimulate the immune system for infants. Evidence already suggests that "probiotics" have certain health benefits, such as the prevention of diarrhea, and that they could promote the development of a balanced immune system in infants. The purpose of the study is to determine if newborn babies who are fed Lactobacillus GG (normally found in yogurt) supplements once a day for the first six-months of life have a decreased risk for developing early signs of asthma.
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Kathy Giacomini |
As co-chair of the committee that planned the Strategic Opportunities Support (SOC) Center, Kathy Giacomini, PhD, director of the Center for Pharmacogenomics in the UCSF School of Pharmacy, believes that UCSF can become the place where basic discoveries will be translated into clinical research and ultimately into practice. The center will be at the crossroads of this effort. As outlined in the proposal endorsed by the National Institutes of Health (NIH), the SOS Center will provide funds and support for pilot projects in clinical and translational research, multidisciplinary planning grants and mini-sabbaticals for faculty to develop expertise in clinical and translational research. Giacomini's research is emblematic of the translational activity that has stamped UCSF as a leader in transforming laboratory insights and discoveries into patient therapies. Her research, for example, focuses on membrane transporters, which facilitate the flux of drugs and important natural compounds into and out of cells. As principal investigator of Pharmacogenetics of Membrane Transporters, a major project funded by NIH, Giacomini is leading an effort to understand the implications of genetic variation in 25 membrane transporters on clinical drug response. Initial studies of the project have demonstrated that there are common variants of xenobiotic transporters that may be responsible for variation in drug response. Ultimately, the information obtained from these studies may be used before treatment to determine whether an individual is likely to benefit from a particular drug or to experience adverse effects, and may even be used to design drugs to better treat subsets of patients who do not respond to standard treatments. Giacomini and her colleagues are also seeking to determine whether genetic variation in transporters contributes to variation in sensitivity and resistance to anticancer drugs.
Related Links:
UCSF Set to Transform Itself into Engine of Translational Research
UCSF in the 21st Century: Translating Scientific Discovery to Patient Care
UCSF Leaders Reflect on Significance of Clinical and Translational Science Institute
Professor Describes Goals of Clinical and Translational Science Institute
The UCSF Clinical and Translational Science Institute: A View to the Future of Research
NIH Launches National Consortium to Transform Clinical Research
Clinical and Translational Science Awards to Transform Clinical Research
