TAG: "Translational medicine"

Big data in biosciences, health care is focus of new UCLA research center


Institute for Quantitative and Computational Biosciences will advance biomedical sciences.

Alexander Hoffmann and his colleagues will collaborate with mathematicians to make sense of a tsunami of biological data. (Photo by Reed Hutchinson, UCLA)

By Stuart Wolpert, UCLA

A new research institute at UCLA may eventually provide doctors with tools to more accurately tailor medicines for individual patients, which could both improve quality of care and minimize the side effects associated with today’s medicine.

The Institute for Quantitative and Computational Biosciences will employ multidisciplinary research to study how molecules and genes interact. Its goal: unlocking the biological basis of health and disease by tapping the power of big data and computational modeling.

“UCLA’s Institute for Quantitative and Computational Biosciences will have a major, positive impact on human health,” said UCLA Chancellor Gene Block. “It will engage exceptional faculty from the life sciences and physical sciences, and our David Geffen School of Medicine and Henry Samueli School of Engineering and Applied Science to ensure that UCLA is at the forefront of research that will help usher in a new era of personalized health care, and to transform research and education in the biosciences.”

The institute is led by Alexander Hoffmann, professor of microbiology, immunology and molecular genetics in the UCLA College, whose research aims to understand how our genes interact to ensure health or produce disease — and the roles played by such factors as food, environmental stresses, infectious agents and pharmaceuticals. Among the diseases for which Hoffmann’s research may lead to significant progress are cancer and immune disorders, because they are caused by errors in cellular decision-making.

Hoffmann says that biology’s million-dollar question is how genes and environment interact to ensure health or cause disease, he said. As UCLA researchers work to answer that question, they will collaborate with UCLA mathematicians who will create mathematical models that help them make sense of a tsunami of biological data.

“Biology is entering a new phase,” Hoffmann said. “So far, biology has been much less math-based than the other sciences. Since the sequencing of the human genome in the early 2000s, there has been an irreversible change in the way biology and biomedical research are being done. At UCLA, we will lead research in that direction and connect basic and applied sciences in an unprecedentedly productive collaboration.”

Victoria Sork, dean of the UCLA Division of Life Sciences, said the institute’s approach represents the “new life sciences” and predicts that the new center will accelerate discovery and translational application in many areas, including medicine, the environment, energy, and food production and food safety.

“Technological breakthroughs are enabling scientists to analyze not only one gene at a time, but how hundreds or thousands of genes work together,” Sork said. “Combined with big data, new knowledge of critical gene networks will lead us to a better understanding of what makes humans healthy.”

The road to “precision medicine”

Dr. A. Eugene Washington, vice chancellor of UCLA Health Sciences and dean of the David Geffen School of Medicine at UCLA, said the new era of personalized medicine will offer higher-quality health care — and possibly lower-cost care — because genetic information will give health providers better knowledge about individual patients.

“We are likely to see significant change in health care in the coming years as genetic data for individuals become more widely available,” Washington said.

In fact, big data already has begun to transform health care. In the past, doctors treating people with a certain disease might have relied solely on their own or their colleagues’ experience treating others with the same disease. Now, instead of relying on a small number of case studies, physicians can turn to mountains of data to guide their approach.

“We haven’t yet begun to fully tap into the knowledge we have about how we have treated millions of patients,” said Dr. Steven Dubinett, director of the UCLA Clinical and Translational Science Institute, and UCLA’s senior associate dean for translational research and associate vice chancellor for research.

“Now, with the rise of big data, we have the capability to utilize a network of brains in a highly sophisticated manner so that all our experience at UCLA, in the University of California system and the many other hospitals with which we share data can be brought to bear on patient treatment in a way that was not possible before.”

The result may be not only personalized health care, but “precision medicine”—the ability for doctors to accurately predict positive health outcomes for patients, Dubinett said.

The move to big data also is dramatically changing the skill sets required for life sciences and biomedical researchers: Increasingly, backgrounds in mathematics, computer science and physics will be highly sought after. Already, UCLA is planning new programs through which computational scientists will train clinicians so they can understand how to work with large sets of data and apply the insights they gain to treating patients.

In addition, UCLA has established a doctoral program in bioinformatics, and the Clinical and Translational Science Institute, in which UCLA is one of four partner institutions, is at the forefront of utilizing big data in clinical care — including developing new pharmaceuticals and bringing important new discoveries into the community.

Much of the data UCLA faculty will work with will come from the University of California Research eXchange, which manages an extremely large repository of clinical data — more than 12 million patient records. Dubinett said UCReX is in the process of adding millions of additional records through partnerships with other Los Angeles medical institutions and, eventually, other academic medical centers in California and throughout the U.S. (Patients’ identities are not released to researchers.)

Dubinett said UCLA will be a national leader in this revolution in personalized health care, in part because UCLA’s medical center is part of its main campus — something that is not the case at many other research universities. That close proximity makes it easier for doctors to collaborate with experts in biomedical informatics and other fields, and has been a lure for many of the exceptional scientists joining the effort.

To strengthen the new institute, UCLA has hired nine faculty members since July 2011 and has plans to hire additional faculty in the next several years. One of the new hires was Leonid Kruglyak, who came to UCLA from Princeton University in 2013. Kruglyak uses big data in his genetics research and, according to Sork, is a “brilliant superstar of the highest stature.”

Among the other outstanding faculty members UCLA has hired, Sork said, are two at the cutting edge of computational biology: Matteo Pellegrini, professor of molecular, cell and developmental biology, and Xinshu (Grace) Xiao, an associate professor of integrative biology and physiology. Both are in the UCLA College.

From individual genes to entire ecologies

Pellegrini, co-director of the institute, said the move to big data also will enable scientists to significantly broaden the scope of their research.

“We’re going from a paradigm where scientists studied individual genes to one in which they will study organisms and even entire ecologies — sequencing the genomes of communities of organisms and understanding how they interact,” Pellegrini said. “Technology is making science very exciting, presenting enormous opportunities to revolutionize our understanding of biology at the genome-wide level and to apply these techniques to answer all kinds of questions.”

Hoffmann said that in the past, one of the major challenges in biology research was generating data. “Now, the challenge is how to make sense of a tsunami of scientific data, to discover the critical patterns and to tell the signal from the noise,” he said. “The opportunities to develop accurate predictions are unprecedented.”

“These examples are just the tip of the iceberg,” said Hoffmann. “The power of combining big data computational tools with computational modeling based on hard basic science is leading a revolution in the bio- and health sciences that provides unimagined opportunities to humanity.”

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Lens-free microscope can detect cancer at cellular level


UCLA researchers develop device that can do the work of pathology lab microscopes.

Tissue sample image created by a new lens-free microscope developed in the UCLA lab of Aydogan Ozcan. (Image by Aydogan Ozcan, UCLA)

By Bill Kisliuk, UCLA

UCLA researchers have developed a lens-free microscope that can be used to detect the presence of cancer or other cell-level abnormalities with the same accuracy as larger and more expensive optical microscopes.

The invention could lead to less expensive and more portable technology for performing common examinations of tissue, blood and other biomedical specimens. It may prove especially useful in remote areas and in cases where large numbers of samples need to be examined quickly.

The microscope is the latest in a series of computational imaging and diagnostic devices developed in the lab of Aydogan Ozcan, the Chancellor’s Professor of Electrical Engineering and Bioengineering at the UCLA Henry Samueli School of Engineering and Applied Science and a Howard Hughes Medical Institute professor. Ozcan’s lab has previously developed custom-designed smartphone attachments and apps that enable quick analysis of food samples for allergens, water samples for heavy metals and bacteria, cell counts in blood samples, and the use of Google Glass to process the results of medical diagnostic tests.

The latest invention is the first lens-free microscope that can be used for high-throughput 3-D tissue imaging — an important need in the study of disease.

“This is a milestone in the work we’ve been doing,” said Ozcan, who also is the associate director of UCLA’s California NanoSystems Institute. “This is the first time tissue samples have been imaged in 3-D using a lens-free on-chip microscope.”

The research is the cover article today (Dec. 17) in Science Translational Medicine, which is published by the American Association for the Advancement of Science.

The device works by using a laser or light-emitting-diode to illuminate a tissue or blood sample that has been placed on a slide and inserted into the device. A sensor array on a microchip — the same type of chip that is used in digital cameras, including cellphone cameras — captures and records the pattern of shadows created by the sample.

The device processes these patterns as a series of holograms, forming 3-D images of the specimen and giving medical personnel a virtual depth-of-field view. An algorithm color codes the reconstructed images, making the contrasts in the samples more apparent than they would be in the holograms and making any abnormalities easier to detect.

Ozcan’s team tested the device using Pap smears that indicated cervical cancer, tissue specimens containing cancerous breast cells, and blood samples containing sickle cell anemia. In a blind test, a board-certified pathologist analyzed sets of specimen images that had been created by the lens-free technology and by conventional microscopes. The pathologist’s diagnoses using the lens-free microscopic images proved accurate 99 percent of the time.

Another benefit of the lens-free device is that it produces images that are several hundred times larger in area, or field of view, than those captured by conventional bright-field optical microscopes, which makes it possible to process specimens more quickly.

“While mobile health care has expanded rapidly with the growth of consumer electronics — cellphones in particular — pathology is still, by and large, constrained to advanced clinical laboratory settings,” Ozcan said. “Accompanied by advances in its graphical user interface, this platform could scale up for use in clinical, biomedical, scientific, educational and citizen-science applications, among others.”

In addition to Ozcan, the principal authors of the research were Alon Greenbaum, a UCLA Engineering graduate student and a research fellow at HHMI, and Yibo Zhang, a UCLA Engineering graduate student. Other authors were UCLA Engineering graduate student Wei Luo, undergraduate researchers Alborz Feizi and Ping-Luen Chung, and Dr. Shivani Kandukuri of the department of pathology and laboratory medicine at the David Geffen School of Medicine at UCLA.

The research was supported by the Presidential Early Career Award for Scientists and Engineers, the National Science Foundation, the National Institutes of Health, the Army Research Office, the Office of Naval Research and the Howard Hughes Medical Institute.

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New drugs from fish oil could air artery repair


Interdisciplinary collaboration key to translating academic research into tangible innovations.

Kevin Lance, a graduate student in Tejal Desai's lab, holds up a tiny "wrap" that could be placed around an artery or vein and slowly release a fish oil-derived drug to prevent vascular scarring. (Photo by Kathleen Masterson, UC San Francisco)

By Kathleen Masterson, UC San Francisco

Every year, more than a half-million Americans undergo procedures to have a narrowed coronary artery propped open with a small metal mesh tube, or stent. The procedure is common for certain patients who’ve experienced a heart attack or other arterial blockages, and it helps to restore blood flow.

But in about 1 in 4 cases (1 in 3 if it’s your leg artery), the vasculature tissue starts renarrowing again after the procedure, effectively regrowing the blockage. The problem of excessive vascular scarring isn’t limited to stents, but also affects many other common procedures such as angioplasty, bypass surgery, and placement of fistulas or grafts for patients on dialysis.

“When we operate on an artery it always causes an inflammatory reaction and a subsequent scarring response just like anywhere else on your body, even the skin,” said Michael S. Conte, M.D., chief of vascular & endovascular surgery at UCSF.

If this inflammation continues, the cells surrounding the tiny metal cage still treat the area as injured, and ultimately grow back in.

But a common class of naturally occurring molecules from fish oil could change all that.

It turns out that when the body heals naturally it’s a two-step process: first it generates compounds to promote inflammation, and when those wane, the body sends in a second set of compounds that actively stops inflammation.

These anti-inflammation signaling compounds are derived in the body from dietary fish oil, and Conte and collaborators Charles Serhan of Harvard University and Tejal Desai, Ph.D., chair of the UCSF Department of Bioengineering and Therapeutic Sciences, are using them to develop treatments to prevent ongoing inflammation in blood vessels.

Desai has been working on stents from a different angle; her lab at the School of Pharmacy focuses on therapeutic microtechnology and nanotechnology. If successful, their collaboration could help prevent arteries and veins from closing up again after surgeries, such as a stent implant or an angioplasty, a procedure where a balloon is temporarily inserted into the artery or vein to open it up.

“What we’re trying to do is turn an angioplasty into a mosquito bite: we want it to get injured and resolve, not get injured and scar,” said Conte.

Interdisciplinary collaboration like that of Desai and Conte is key to translating academic research into tangible innovations. Partnering with industry is also vital in getting technologies to patients: UCSF’s Innovation, Technology and Alliances supports hundreds of researchers to make science come alive by procuring licenses, partnering with industry sponsors and filing patents.

ITA helped Conte and Desai file a patent for their combination drug-device approach. Theirs is one of 48 patent filings this fiscal year, while overall UCSF holds a total of 631 active patents. Additionally, ITA negotiated nearly 400 industry-sponsored research deals and new clinical trial agreements.

Conte and Desai also won an NIH grant that specifically funds promising vascular research that could be translated into a medicinal use. The grant keeps them on a tight timeline to get their product up to speed for testing in humans.

To date, Conte has shown the drug reduces vascular scarring in mice and rabbits after they undergo an angioplasty. Not only did the drug lead to less scar tissue build-up, but they found a reduction in the presence of white cells weeks later, suggesting healing was accelerated.

With the help of UCSF’s Innovation, Technology & Alliances team, the researchers have filed a patent for their combination drug-device approach.

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UC translational medicine leaders chart course


UC BRAID holds annual retreat in San Diego.

UC BRAID program leaders (from left): Jennifer Grandis (UC San Francisco), Lars Berglund (UC Davis), Deborah Grady (UC San Francisco), Steven Dubinett (UCLA), Gary Firestein (UC San Diego) and Dan Cooper (UC Irvine). (Photo by Courtney McFall, UC San Francisco)

By Patti Wieser, UC San Diego

With plans to “think boldly” about the next phase of integrating resources and talent, representatives of the University of California Biomedical Research Acceleration, Integration, and Development (UC BRAID) program staked out future directions during the annual retreat Nov. 7 at UC San Diego. Plans on the horizon include integrating informatics across the UC enterprise, expanding UC Research Exchange (UC ReX – a federated multisite clinical data repository), developing industry partnerships, and expanding the systemwide network for clinical and translational research.

The meeting, which focused on innovation, collaboration and acceleration, drew more than 80 translational medicine researchers, administrative leaders, staff and faculty representing eight UC campuses. The participants also discussed major achievements and potential new areas of focus.

“We are extremely excited about our progress as we continue to create an environment that decreases barriers to biomedical research and creates new tools to facilitate research,” said Gary S. Firestein, M.D., UC BRAID chair, director of UC San Diego Clinical and Translational Research Institute (CTRI) and dean and associate vice chancellor of translational medicine at UC San Diego. “UC BRAID serves as a model for collaborative consortia.”

Established in 2010, UC BRAID, in collaboration with the University of California Office of the President (UCOP), is a joint effort of the five UC biomedical campuses to catalyze, accelerate and reduce the barriers for biomedical, clinical and translational research across the UC system. The UC BRAID consortium — UC Davis, UC Irvine, UCLA, UC San Diego and UC San Francisco — pools data, resources and expertise to reach this goal. UC Riverside, UC Santa Barbara and UC Santa Cruz and UCOP also participated in this year’s UC BRAID meeting.

Lars Berglund, incoming chair of UC BRAID, welcomed the retreat participants. “BRAID is not a goal. It is a means for reaching our goals,” said Berglund, M.D., Ph.D., director of the Clinical and Translational Science Center and senior associate dean of research at UC Davis. The retreat provided a snapshot of “who we are” and energized the participants to continue fulfilling BRAID’s mission. “Enhancing collaboration between the UC system partners will advance the translational research initiative by disintegrating barriers that have evolved,” he said.

Rachael Sak, R.N., M.P.H., director of UC BRAID, discussed the evolvement of UC BRAID during her presentation about leveraging a UC network. “We have a shared vision: to integrate resources and talent across UC to accelerate research that improves health. We are leveraging these, developing Institutional Review Board (IRB) and contracting metrics, and shaping into a collaborative network,” she said. Sak, noting how far the organization has progressed since it was established, cited the following two major successes of UC BRAID during this past year:

Cross-UC clinical trial recruitment: Building upon its accomplishments in cohort discovery and IRB reliance, UC BRAID is developing more advanced cross-campus participant recruitment strategies and services.

National leadership in NIH National Center for Advancing Translational Sciences (NCATS) projects: UC BRAID is at the core of two recent initiatives, Accrual to Clinical Trials and IRB Reliance, supported by NCATS to enable a national network that can conduct large, multicenter clinical trials.

William Tucker, interim vice president of research and graduate studies and executive director of Innovation Alliances and Services with UCOP, presented a talk, “Leveraging UC’s research enterprise for value: President Napolitano’s initiatives that involve research.” Tucker said these initiatives include stimulating research and discovery in areas of strategic importance that benefits California and the world, and improves human lives, the environment and the economy. He lauded BRAID for doing a “great job” of organizing itself and leveraging the system and common practices. Tucker’s takeaway message was: “Think boldly.”

Other presenters were Mike Palazzolo, director of UC BRAID Center for Accelerated Innovation; Doug Bell, chair of UC ReX; Mike Caliguiri, project director for IRB metrics; Eric Mah, project director for IRB reliance; and Dan Dohan, project director for EngageUC. Breakout sessions at the retreat focused on biobanking and biorepositories, child health, contracting, regulatory, drug and device discovery and development, and UC ReX.

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6 translational science trends that will improve your health


Trends highlighted in podcasts range from team science to big data for health.

By Deborah Grady, UC San Francisco

Translational science, also known as bench-to-bedside research, aims to translate biomedical discoveries into useful applications and treatments, such as a drug, device, diagnostic or behavioral intervention, that impact health and health outcomes.

At UC San Francisco, my colleagues and I at the UCSF Clinical and Translational Science Institute (CTSI) are collaborating and innovating in ways that are transforming health care as we know it.

We’re also looking ahead at the trends and influences that are reshaping – and more importantly, accelerating – translational science, all with a focus on improving health. We partnered with Carry The One Radio to produce podcasts on each of the trends.

Learn more about the podcasts or listen to the full playlist here.

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Bridge builder


Ellen Olshansky excels at forging bonds between university, community health organizations.

“My goal is to build partnerships with community-based groups,” says Ellen Olshansky, professor and founding director of nursing science at UCI. “I want to ask ‘How can we work together?’ It’s the difference between doing research ‘with’ and doing research ‘on.’” (Photo by Steve Zylius, UC Irvine)

Since joining UC Irvine in 2007 to launch the nursing science program, Ellen Olshansky has flourished as a highly respected county leader for community-based research and women’s health policy. And much of it started at her kitchen table five years ago.

There, in her University Hills home, Olshansky brought together Susan Bryant and Karol Gottfredson of UCI and Allyson Sonenshine and Stephanie Kight of Planned Parenthood of Orange & San Bernardino Counties to share ideas about what they could do to champion women’s health issues. Brainstorming over white wine, cheese and crackers, they outlined what would become the Orange County Women’s Health Project.

With aggressive planning and outreach by these five women, the OCWHP kicked off in 2011. In May 2012, it hosted the inaugural Orange County Women’s Health Policy Summit, at which a UCI alumna presented “A Snapshot of Women’s Health in Orange County” – the first-ever such survey.

The project’s partners have since formed task forces for breast and cervical cancer, teen reproductive health, and health and domestic violence. The work is paying dividends: Earlier this year, Blue Shield awarded the OCWHP $2 million to establish a countywide, integrated and collaborative system that will strengthen healthcare response to domestic violence and streamline service.

“Ellen was instrumental in getting the project off the ground – introducing the vision and doing the outreach and creating momentum,” says Sonenshine, OCWHP director. “We’ve developed a wonderful model that’s focused on data analysis, policy and education, and we play an increasingly important role.”

Now Olshansky is applying her bridge-building talents at UCI’s Institute for Clinical & Translational Science.

Supported by a prestigious Clinical & Translational Science Award from the National Institutes of Health, the ICTS is dedicated to advancing efforts to turn scientific discoveries into new methods, treatments and cures to improve public health. One of its most important objectives is community engagement, and that’s where Olshansky comes in.

“My goal is to build partnerships with community-based groups,” she says. “I want to ask ‘How can we work together?’ It’s the difference between doing research ‘with’ and doing research ‘on.’”

ICTS Director Dr. Dan Cooper says Olshansky is the perfect person at the perfect time to lead the community engagement push.

“Ellen has a long history of collaborating with the community, and her work is based upon having real dialogue and understanding among groups that don’t always speak the same language,” Cooper says. “She has remarkable skills in translating and expressing to faculty the community needs that impact health directly. Being a facilitator between these two groups is invaluable to us.”

To boost outreach, the ICTS is a founding partner of the Orange County Alliance for Community Health Research, which consists of the leaders of local, community-based organizations; practicing physicians; healthcare agencies; governmental representatives; community groups; and UCI researchers.

The alliance’s purpose is to create an infrastructure in Orange County that increases the ability of community organizations and universities to engage in health research that’s designed by the community to meet the needs of the community. Olshansky serves on its advisory board.

“At the heart of the alliance is the belief that the community first expresses its health needs, and then university researchers work with these partners to find solutions,” she says. “That’s what community-based research is all about, and I’ll be working diligently to further establish those relationships in Orange County.”

Earlier this year, Olshansky stepped aside from directing UCI’s Program in Nursing Science, where over seven years she oversaw the initiation of the bachelor’s program and the approval and initiation of the master’s and doctoral programs. She also spearheaded the effort to include nurse practitioner concentrations in the master’s program.

And during a recent sabbatical, Olshansky put the finishing touches on her latest book, Women’s Health & Wellness Across the Lifespan. Set for release on Dec. 11, it offers a historical and comprehensive look at women’s health – politically, socially, legally and medically – through contributions from leading experts across the country.

“This book is important because it explains the reasons why it’s necessary to have a focus on the issue of women’s health,” Olshansky says. “There has been such political push-back on sexual and reproductive health services, and we need to be sure that we continue to provide and strengthen these services.

“But many people believe that’s all women’s health is about. In fact, it’s much more than that, and this book goes in depth into the many other important issues women face. It’s written mostly for primary care physicians and nurse practitioners, but I believe anyone interested in women’s health will benefit from reading it.”

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UCSF sugar science initiative launched


Researchers highlight strong links between sugar and chronic disease.

By Kristen Bole, UC San Francisco

Researchers at UC San Francisco have launched SugarScience, a groundbreaking research and education initiative designed to highlight the most authoritative scientific findings on added sugar and its impact on health.

The national initiative is launching in partnership with outreach programs in health departments across the country, including the National Association of City and County Health Organizations and cities nationwide.

Developed by a team of UCSF health scientists in collaboration with scientists at UC Davis and Emory University School of Medicine, the initiative reflects an exhaustive review of more than 8,000 scientific papers that have been published to date on the health effects of added sugar.

The research shows strong evidence of links between the overconsumption of added sugar and chronic diseases, including Type 2 diabetes, heart disease and liver disease. It also reveals evidence linking sugar to Alzheimer’s disease and cancer, although the team assessed that more research is needed before those links can be considered conclusive.

Laura Schmidt, UC San Francisco

“The average American consumes nearly three times the recommended amount of added sugar every day, which is taking a tremendous toll on our nation’s health,” said Laura Schmidt, Ph.D., a UCSF professor in the Philip R. Lee Institute for Health Policy Studies and the lead investigator on the project. “This is the definitive science that establishes the causative link between sugar and chronic disease across the population.”

The initiative aims to bring scientific research out of medical journals and into the public domain by showcasing key findings that can help individuals and communities make informed decisions about their health. For example, SugarScience.org cites research showing that drinking just one can of soda per day can increase a person’s risk of dying from heart disease by nearly one-third, and can raise the risk of getting Type 2 diabetes by one-quarter.

More than 27 million Americans have been diagnosed with heart disease, which is the nation’s leading cause of death. Another 25.8 million Americans have Type 2 diabetes, caused by the body’s resistance to the hormone insulin coupled with the inability to produce enough insulin to regulate blood sugar levels. Of greatest concern is the rising number of children suffering from these chronic diseases.

Kristen Bibbins-Domingo, UC San Francisco

“Twenty years ago, Type 2 diabetes was unheard of among children, but now, more than 13,000 children are diagnosed with it each year,” said Kirsten Bibbins-Domingo, M.D., Ph.D., a UCSF professor of medicine, epidemiology and biostatistics, and director of the UCSF Center for Vulnerable Populations at San Francisco General Hospital and Trauma Center. “Diabetes is a devastating disease and we know that it is directly related to the added sugar we consume in food and beverages.”

Another rising concern is the impact of added sugar on Non-Alcoholic Fatty Liver Disease (NAFLD), which affects 31 percent of adults and 13 percent of children, and can lead to cirrhosis and liver failure.

“As pediatricians, we had evidence of the connection between sugar and diabetes, heart disease, and liver disease for years, but we haven’t had this level of definitive scientific evidence to back up our concerns,” said Robert Lustig, M.D., M.S.L., a pediatric endocrinologist at UCSF Benioff Children’s Hospital San Francisco and a member of the SugarScience team. “Our goal is to make that science digestible to the American public, and take the first step toward a national conversation based on the real scientific evidence.”

Robert Lustig, UC San Francisco

While there are no federal recommended daily values for added sugar, the American Heart Association recommends consuming less than 6 tsp. (25 g) for women and 9 tsp. (38 g) for men. Guidelines for children depend on caloric intake, but range between 3-6 tsp (12-25 g) per day. Americans currently consume 19.5 tsp. of added sugar, on average, every day.

Added sugar is defined as any caloric sweetener that is added in food preparation, at the table, in the kitchen or in a processing plant. It can be difficult for people to know how much sugar they are consuming, since roughly 74 percent of processed foods contain added sugar, which is listed under at least 60 different names on food labels.

The 12-member SugarScience team will continue to monitor scientific research about added sugar and will track findings at SugarScience.org. The initiative harnesses the power of UCSF’s extensive health sciences enterprise, which ranges from basic laboratory research to clinical, population and policy sciences, with an emphasis on translating science into public benefit. All four of UCSF’s graduate schools – dentistry, medicine, nursing and pharmacy – lead their fields in research funding from the National Institutes of Health, reflecting the caliber of their research. It also is aligned with the UC Global Food Initiative, which seeks to harness UC resources to address global food needs.

SugarScience is made possible by an independent grant from the Laura and John Arnold Foundation. It is supported by the Clinical and Translational Science Institute and the Philip R. Lee Institute for Health Policy Studies at UCSF.

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UCLA hosting 24-hour invention competition to meet health care needs


Inventathon encourages teams of young inventors to develop innovative solutions.

A team of UCLA students working on their project during the 2013 Inventathon competition. (Photo by Samantha Le, UCLA)

Just a stone’s throw from Silicon Beach — the epicenter of technology in Los Angeles — the Business of Science Center at UCLA, with support from the Center for Advanced Surgical and Interventional Technology and Center for Digital Behavior, is spurring innovation as the organizer of the second-annual Inventathon.

This event is a unique 24-hour competition designed to develop solutions for pressing health care needs using the latest device technology and mobile applications.

Watches that track more than time and augmented reality glasses worn like conventional glasses, but that also house a tiny computer, are just the latest examples of wearable devices. Inventathon is designed to help young inventors harness similar technologies for use in the healthcare field.

Inventathon kicks off Oct. 15 with the announcement of the health care need to be addressed. Teams then have a couple of days to assemble before the actual competition starts on Oct. 17. Once the competition begins the teams will work around the clock to develop and eventually present their ideas to a panel of judges. The product could be a mobile app, conceptual drawing or embedded or wearable device. Mentors from UCLA and industry will be available during the entire process, which is designed to help participants hone their research and entrepreneurial skills.

The 24-hour inventing marathon serves as the concluding event of UCLA Innovation Week, organized by Bruincubate, a collection of 14 different groups at UCLA dedicated to promoting entrepreneurship. Bruincubate is hosted by the UCLA Office of Intellectual Property and Industry Sponsored Research. Innovation Week brings together UCLA’s entrepreneurial organizations to help students, faculty, and staff explore and grow their ideas into tangible products. In addition to the Inventathon, events include talks, a career fair and mixers.

The Inventathon competition will take place at the UCLA California NanoSystems Institute. “This event supports future inventors and entrepreneurs,” said Shyam Natarajan, a Business of Science program director and a Center for Advanced Surgical and Interventional Technology researcher, who helped launch the event last year. “We are excited to see raw science talent paired with business and design expertise to develop and jumpstart ideas.”

Medical technology inventors of all levels, from undergraduates and graduate students from UCLA and other universities are welcome. Organizers encourage the teams, consisting of three to five participants, to include a wide range of skills from the medical field, engineering, art, design and business.

During the 24-hour competition, the teams will have access to tools such as 3-D printers, augmented reality glasses that can be used to help design and test applications for wearable devices, and special boards to help make mini computer chips, which are the brains behind the applications.

“Competitions like Inventathon get students to think there are no walls that will inhibit them,” said Roy Doumani, a professor at the David Geffen School of Medicine at UCLA and executive director of the Business of Science Center. “The experience is invaluable in developing the skill set needed to succeed in developing and pitching a product. Participants are mentored throughout the competition and we want to thank our mentors for their extremely valuable support and time.”

Additional programs on UCLA’s campus help students even after the competition. The Business of Science Center offers a course called Advancing Bioengineering Innovations designed to teach medical device design and to develop practical solutions for unmet medical needs. The program is a collaboration among the Department of Bioengineering in the UCLA Henry Samueli School of Engineering and Applied Science, the David Geffen School of Medicine at UCLA and the UCLA Anderson School of Management.

“There is huge potential for the latest remote monitoring applications and devices to support and track health care needs,” said Sean Young, assistant professor of family medicine and executive director of the Center for Digital Behavior at UCLA. The center brings together academic researchers and private sector companies to study how social media and mobile technologies can be used to predict and change behaviors that impact health. “Events like Inventathon are a great resource and learning opportunity for students.”

The second annual Inventathon will start on Wednesday, Oct. 15, with a kickoff event to announce the type of health need to be solved and to start assembling teams. Competition begins at 4 p.m. on Friday, Oct. 17 and the competition concludes Saturday, Oct. 18 at 6 p.m.

The public is invited to watch the final pitches to the judges and the announcement of the winners, which will take place from 4 to 6 p.m. on Saturday.

The UCLA Clinical Translational Science Institute is a collaborator on the event. This project received support from the following NIH/NCATS grant to the UCLA Clinical Translational Science Institute: UL1TR000124.

Inventathon sponsors include: Option3 LLC; Cardiovascular Systems; Epson America; SparkFun Electronics; UCLA Blum Center for Poverty and Health in Latin America; KARL STORZ Endoscopy-America; Hitachi Aloka Medical America; UCLA Center for World Health; Lob; California NanoSystems Institute, UCLA AIDS Institute and UCLA Health.

For more information about Inventathon and sponsorship opportunities, please visit www.UCLAideas.com.

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New effort to fight autoimmune disorders


Major partnership including UC San Diego will focus first on rheumatoid arthritis and lupus.

The Division of Rheumatology, Allergy and Immunology at the UC San Diego School of Medicine has been named a key site in a national, multi-institution, multiyear $41.6 million program to speed drug discovery, development, diagnostics and therapies for patients with autoimmune disorders, primarily rheumatoid arthritis (RA) and lupus erythematosus, which affect millions of Americans.

“We will be looking to pinpoint the genes, proteins, chemical pathways and networks involved in these diseases at the single cell level,” said Gary S. Firestein, M.D., professor, dean and associate vice chancellor of translational medicine. “This approach allows us to make comparisons across many diseases, revealing new insights and aspects of the disease process. We hope to better understand why some RA patients, for example, respond to therapy and others do not – and develop new therapies that target their condition based upon their particular genetic and environmental variables.”

The effort is part of a five-year, $230 million program called Accelerating Medicines Partnership (AMP), a collaboration between the Food and Drug Administration, the National Institutes of Health (NIH), 10 biopharmaceutical companies, and several nonprofit organizations. It will initially focus upon autoimmune disorders, type 2 diabetes and Alzheimer’s disease, with other diseases and conditions added in the future. The program for RA and lupus is managed through the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).

The unusual cross-sector partnership will emphasize finding tell-tale “biomarkers” for these disease areas, which are molecules that can be helpful for diagnosis or selecting treatment. The group also will identify promising drug targets and ways to reduce the time and cost of developing new therapeutics. A critical component of the effort, say officials, is that industry partners, such as Merck and Pfizer, will make AMP data and analyses publicly accessible to the broad biomedical community.

“To date, treatments for RA and lupus have been aimed at decreasing inflammation and pain,” said Stephen I. Katz, M.D., Ph.D., director of NIAMS. “For the first time, we are bringing together multidisciplinary research teams to achieve a broad, systems-level understanding of these diseases, setting the stage for the development of more effective diagnostic and treatment approaches.”

In a consortium with the University of Colorado, the University of Nebraska and Cedars Sinai Medical Center, UC San Diego will be responsible for using the latest genomics and epigenomics research tools to collect and process tissue and blood samples from patients with RA.

RA is primarily an inflammatory disease of the joints, affecting an estimated 1.5 million Americans, or almost 1 percent of the U.S. adult population. While it most often diagnosed in middle age and occurs with increased frequency in older people, it also strikes children and young adults. Symptoms include pain, selling, stiffness and loss of function in joints. RA typically becomes chronic. There are many treatments, but no cure. Research at UC San Diego in RA has contributed to the discovery of several novel therapies that are currently being used. Despite these advances, many patients still have pain and diminished quality of life.

Both RA and lupus belong to a larger group of autoimmune disorders that includes multiple sclerosis, Crohn’s disease, ulcerative colitis, type 1 diabetes and psoriasis. These diseases, say researchers, share common flaws in immune function and regulation, leading to inflammation that destroys tissues and results in reduced quality of life, disability and increased risk of death.

If successful, the researchers said the combined efforts of academia, the NIH and biotechnology companies can change the way research is performed and create multidisciplinary teams that can be more effective than individual groups.

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UC autism summit offers hope for help


First step toward collaborating systemwide to address need for treatments.

Leonard Abbeduto, director of the UC Davis MIND Institute, speaks at the UC summit on autism in Sacramento. (Photo by UC Davis)

By Alec Rosenberg

Researchers from across the University of California convened Thursday (Aug. 14) for a first-ever summit on autism — an initial step toward collaborating systemwide to address the urgent need for treatments.

Epidemiologists and geneticists joined neuroscientists and psychiatrists as more than 50 researchers from five UC campuses participated in the daylong summit at the UC Davis MIND Institute in Sacramento. They shared what they are doing in autism research and discussed ways to increase coordination and have a greater impact in improving the lives of children with autism and their families.

Autism spectrum disorder is one of health’s toughest challenges — a lifelong developmental condition with varying symptoms and severity that can affect social interactions, behavior and the ability to think, learn and problem solve. It has no single known cause and no known cure, though early behavioral-based treatments can help. And its prevalence is rising rapidly: Estimates are that autism affects more than 3 million individuals in the U.S., increasing the need for breakthroughs.

“How can we provide high-quality care for kids when the numbers are increasing dramatically and the resources are not?” said Leonard Abbeduto, Tsakopoulos-Vismara Endowed Chair and director of the UC Davis MIND Institute. “We’re all here because we want to impact the lives of kids and families.”

The autism summit, sponsored by the UC Office of the President, is the first step in an 18-month process aimed at accelerating progress toward treatments and strategies for prevention. The effort will include drafting a strategic plan for a coordinated approach to UC autism research, identifying research opportunities, increasing the number of multicampus grants and launching a series of public statewide autism forums to discuss ways of translating research into improved services.

“These are ambitious goals, but this is the group to make it happen,” Abbeduto said.

Harnessing UC’s expertise

The campuses participating in the summit — Davis, Irvine, UCLA, San Diego and San Francisco — are those with interdisciplinary autism research programs, integrated health care systems and programs that train pediatric health care professionals. Other UC campuses will participate in follow-up meetings.

The summit arose from discussions within the systemwide UC BRAID (Biomedical Research Acceleration, Integration and Development), which identified autism as an area of expertise that was ripe for increased coordination.

“This is really the beginning,” said Dan Cooper, chair of the Department of Pediatrics and director of the Institute for Clinical and Translational Science at UC Irvine. “The summit is designed to harness the unique basic science and translational research talent across the UC system in a way that will profoundly benefit children and adults with autism and related disorders.”

As one of the world’s largest and most prestigious research institutions, UC is uniquely positioned to address the mysteries surrounding autism.

“If we combine and band together, the promise is tremendous,” said Elysa Marco, a cognitive and behavioral child neurologist at UC San Francisco. “I think this represents a wonderful opportunity for us to do something greater.”

A timely collaboration

Thomas Insel, director of the National Institute of Mental Health, who delivered a summit presentation by videoconference, encouraged UC’s efforts, which he said could be a model at the statewide level.

“If you can get a group of people across the state sharing things, that’s a great way to accelerate our understanding and development of treatments,” said neurologist Jeffrey Neul of UC San Diego.

Autism is considered a public health crisis, with an incidence that has increased by more than 600 percent during the past two decades. The U.S. Centers for Disease Control and Prevention estimates that autism now affects 1 in 68 children. More than 350,000 Californians live with autism today.

Los Angeles Unified School District alone has 10,000 students with autism, said James McCracken, the Joseph Campbell Professor of Child Psychiatry and director of the Division of Child and Adolescent Psychiatry at UCLA. He’s optimistic that the UC autism summit will lead to positive outcomes.

“It’s an exciting first step,” McCracken said.

Making an impact

At the summit, researchers split into six working groups aimed at tackling different aspects of autism: genetic risk factors; environmental risk factors; neurobiology; diagnosis, symptoms and developmental trajectories; treatment, pharmacology and services; and research infrastructure.

They identified opportunities for collaboration such as multicampus research projects, hosting workshops to share data and provide training, and developing a systemwide autism patient registry and research repository. They encouraged using the UC ReX (Research eXchange) Data Explorer, a UC BRAID effort that enables UC investigators to identify potential research study cohorts at the five UC medical centers. Several mentioned that offering incentives would spur broader collaborations.

Ultimately, UC summit participants want to help prevent autism and speed treatments and cures. They’re working on many fronts, from behavior to medications to stem cells.

UC Davis MIND Institute researcher Sally Rogers helped develop the Early Start Denver Model, an intensive early intervention therapy for children with autism that fuses play- and relationship-based approaches with teaching practices of applied behavior analysis (a model developed at UCLA).

The Early Start Denver Model is being used around the world. Last week, Rogers trained a group that included participants from Brazil, Canada, Mexico, Spain and Turkey. She wants to have an even bigger impact and sees potential in a universitywide autism collaboration.

“It’s about trying to enhance the quality of life,” Rogers said.

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UC hosting statewide autism summit


UC researchers collaborate across system to address urgent need for treatments.

Bringing together the research prowess of the University of California to address the increase in autism incidence, its public health impacts, and the need to speed the development of treatments for affected individuals and their families, internationally respected scientists from UC campuses at Irvine, Los Angeles, San Diego, San Francisco and Davis will converge at the UC Davis MIND Institute for a daylong summit on innovative translational neurodevelopmental research.

An initiative of the UC Office of the President, the University of California Summit on Translational Research in Autism Spectrum Disorders will be held Thursday (Aug. 14) from 8 a.m. to 5 p.m. at the UC Davis MIND Institute, 2825 50th St., Sacramento. The summit will include a presentation via video conference on “Pressing Issues for a Translational Science of Autism Spectrum Disorder” by Thomas Insel, director of the National Institute of Mental Health (NIMH). Participation in the summit is by invitation only.

“The increase in autism spectrum disorder cases has exceeded the capacity of public and private organizations to provide effective health care, education and treatment to affected families,” said Leonard Abbeduto, Vismara-Tsakopoulos Endowed Chair and director of the MIND Institute.

“We must develop new, more effective strategies for treatment and prevention that are informed by a deeper understanding of the etiology, mechanisms and manifestations of the disorder.”

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UCLA awarded $7M to unravel mystery genetic diseases


One of six institutions chosen by NIH to help tackle the most difficult-to-solve medical cases.

The David Geffen School of Medicine at UCLA is one of six institutions nationwide chosen by the National Institutes of Health to join the agency’s efforts to tackle the most difficult-to-solve medical cases and develop ways to diagnose rare genetic disorders.

Part of a $120 million initiative called the Undiagnosed Diseases Network, the $7.2 million grant to UCLA will support comprehensive “bedside-to-bench” clinical research to aid physicians in their efforts to give long-sought answers to patients living with mystery diseases.

“Undiagnosed diseases take a huge toll on patients, their families and the health care system,” said Katrina Dipple, a co-principal investigator on the UCLA grant with Stanley Nelson, Christina Palmer and Eric Vilain. “This funding will accelerate and expand our clinical genomics program, enabling us to quickly give patients a firm diagnosis and clarify the best way to treat them.”

Despite extensive clinical testing by skilled physicians, some diseases remain unrecognized because they are extremely rare, underreported or atypical forms of more common diseases. An interdisciplinary team of geneticists at each Undiagnosed Diseases Network site will examine and study patients with prolonged undiagnosed diseases.

“A vast number of children and adults suffer from severe, often fatal, undiagnosed disorders,” Vilain said. “This program will enable us to discover new genes causing ultra-rare medical conditions and to identify environmental factors that lead to disease or that interact with genes to cause disease.”

Patients will undergo an intensive weeklong clinical assessment that includes a clinical evaluation, consultations with specialists, and medical tests, including genome sequencing to identify genetic mutations. The team will also evaluate the impact on patients and families of genetic counseling and genomic test results to develop best practices for conveying this information.

The Undiagnosed Diseases Network capitalizes on the strengths of UCLA’s genetic medicine program, particularly its Clinical Genomics Center, which utilizes powerful sequencing technology to diagnose rare genetic disorders. Using a simple blood sample from a patient and both parents, the center can perform a test that simultaneously searches 37 million base pairs in 20,000 genes to pinpoint the single DNA change responsible for causing a patient’s disease. To date, a specific genetic explanation has been identified in a quarter of the cases evaluated with this test, as have a number of novel disease-causing genes.

UCLA is the only facility in the western U.S. and one of only three nationwide with a laboratory that can perform genomic sequence directly usable for patient care, and the university’s Medical Genetics Clinic cares for more than 750 new patients a year and offers comprehensive pre- and post-test genetic counseling.

All patient studies will take place at UCLA’s Westwood campus, at the Clinical and Translational Research Center of the Clinical and Translational Science Institute. Network investigators will share genomic and clinical data gleaned from patients with their research colleagues nationwide to enhance the understanding of rare and unknown diseases.

Patients interested in participating in the Undiagnosed Diseases Network may learn more at www.rarediseases.info.nih.gov/undiagnosed. Applications will be accepted beginning in the fall.

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