TAG: "Translational medicine"

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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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 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.

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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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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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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MedImmune collaboration boosts UCSF’s Catalyst Awards


Partnership will support projects that translate research into treatments.

Roxanne Duan (left), MedImmune, and June Lee, UC San Francisco

The Clinical and Translational Science Institute (CTSI) at UC San Francisco has entered into a three-year collaboration with MedImmune, the global biologics research and development arm of AstraZeneca. The collaboration will focus on CTSI’s Catalyst Awards program, which solicits applications from university scientists who wish to move their translational research beyond the bench and into product development.

This marks the first industrial partnership for CTSI’s Catalyst Awards program’s therapeutic track, which focuses specifically on discovery and development of patient treatment options. The collaboration will benefit both MedImmune’s biologics and AstraZeneca’s small molecule portfolios and will call for proposals in therapeutic areas of interest to MedImmune and AstraZeneca, including cardiovascular and metabolic disease; oncology; respiratory, inflammation and autoimmunity; neuroscience; and infectious disease.

MedImmune and UCSF will collaborate to move forward the most promising research projects over the next three years with the option to extend the partnership. The collaboration will foster scientific exchange and expertise between UCSF and MedImmune scientists, and support projects that translate research into treatments that improve patient outcomes.

“UCSF’s innovative translational research capabilities combined with MedImmune’s industry experience in this area will help identify and nurture promising early science that can benefit MedImmune, UCSF researchers and ultimately patients,” said Dr. Bing Yao, senior vice president and head of MedImmune’s Respiratory, Inflammation and Autoimmunity Innovative Medicines Unit.  “We look forward to partnering with leading scientists at UCSF and growing our collective expertise within the Bay Area, one of the top global bioscience hubs.”

June Lee, M.D., F.A.C.C.P., director of CTSI’s Early Translational Research program, which manages the Catalyst Awards, echoed that sentiment. “This collaboration marks an important first step in cultivating critical industry-academic partnerships in our therapeutics track. Beyond that, it also supports the broader mandate of UCSF and the NIH Clinical and Translational Science Awards to accelerate research to improve health, for which industry and companies like MedImmune are very important partners.”

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University of Texas taps UCSF neurologist to lead new medical school


Clay Johnston to become inaugural dean of medical school at University of Texas at Austin.

Clay Johnston

Clay Johnston

Clay Johnston, M.D., Ph.D., a physician-scientist who expanded UC San Francisco’s patient-centered research through his leadership of the Clinical Translational Science Institute (CTSI) and the new Center for Healthcare Value, is leaving at the end of February to become the inaugural dean of the Dell School of Medicine at The University of Texas at Austin.

A neurologist and epidemiologist, as well as associate vice chancellor for research, Johnston has been at UCSF since his residency 20 years ago. He has published widely in his field – the prevention and treatment of stroke and transient ischemic attack – and treats patients with cerebral aneurysms, vascular malformations and stroke, in addition to directing the hospital stroke service.

Johnston is the principal investigator of a $112 million Clinical and Translational Science Award from the National Institutes of Health aimed at helping scientists bring experimental research into the clinic.

“Clay has played a singular role in UCSF’s drive to accelerate translational research to improve human health,” said Jeffrey Bluestone, Ph.D., executive vice chancellor/provost at UCSF. “He’s a steady and unflappable leader, and this, along with his research acumen, has enabled UCSF to forge critical partnerships in the biotech industry and with foundations and private funders.”

Deborah Grady

Deborah Grady

Johnston, who received his medical degree from Harvard and did his internship at Massachusetts General Hospital, said UCSF was a great training ground for him, and it will continue to be a place where those he mentored and supervised can develop.

“I’m proud of what the people of CTSI have done in the last few years,” he said. “I hate to leave such a strong team and so many great teachers and friends but know that in a place like UCSF their work will only accelerate.  ”

In his new job, he will be building a medical school and hospital, literally from the ground up. The first class of students will enter in the fall of 2016.

CTSI’s co-director, Deborah Grady, M.D., M.P.H., will become interim director of the CTSI. Grady is a professor of medicine, as well as epidemiology and biostatistics. She directs the UCSF/Mount Zion Women’s Health Clinical Research Center and the UCSF Women’s Health Faculty Development Program.

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UCSF, Quest Diagnostics launch collaboration to advance precision medicine


Areas of focus will include autism, oncology, neurology and women’s health.

June Lee, UC San Francisco

June Lee, UC San Francisco

UC San Francisco and Quest Diagnostics, the world’s leading provider of diagnostic information services, have formed a collaboration to accelerate the translation of biomedical research into advanced diagnostics in the field of precision medicine, for improved patient care, treatment and outcomes.

Initial clinical areas of focus include autism, oncology, neurology and women’s health.

The collaboration, which combines the research discoveries and capabilities of UCSF with the national testing database and technical and clinical development capability of Quest Diagnostics, has an overarching aim of enabling holistic and integrated diagnostic solutions that close gaps in care or enable new clinical value.

Under the terms of the agreement, scientists will jointly research, develop and validate diagnostic innovations to solve specific clinical problems and provide actionable information to improve patient care. The organizations will focus on diagnostics to advance precision medicine, an emerging field of medical science that aims to integrate the most informative data from molecular, clinical, population and other research to create predictive, preventive and precise medical solutions for patients. Quest Diagnostics would independently develop and validate any lab-developed tests for clinical use that emerge from the collaboration’s research.

Researchers will utilize laboratory-based diagnostics, imaging procedures and population analysis based on Quest’s national Health Trends database, the largest private clinical database in the U.S., based on more than 1.5 billion patient encounters, to advance precision medicine.

The alliance is the first master agreement that UCSF’s Office of Innovation, Technology and Alliances has signed with a clinical laboratory testing company and augments the university’s efforts to translate laboratory research into new therapies. The broad agreement lays the groundwork for multiple projects between the two organizations.

“Advances in technology and science have identified many promising opportunities to improve outcomes through insights revealed by novel diagnostic solutions, yet fulfilling the full potential of these opportunities often hinges on translational clinical studies which validate their value,” said Jay Wohlgemuth, M.D., senior vice president, science and innovation, Quest Diagnostics. “This unique collaboration between UCSF and Quest brings together the finest researchers and clinicians in the country to accelerate the development of a ‘product pipeline’ of scientific discoveries as clinically valuable diagnostic solutions that enable precision medicine for improved outcomes.”

The collaboration is launching with two specific projects already under way. One project involves Quest’s national database of molecular testing data to facilitate participation in research and development efforts related to genetic variations of autism, based on Quest’s CGH microarray ClariSure technology, which can help identify genetic mutations associated with autism and other developmental disorders. While there currently is no treatment for autism, a test that aids its diagnosis could help identify individuals who might be appropriate candidates for research studies that could lead to future therapies.

The second project aims to identify biomarkers to determine which children with glioma brain tumors may benefit from a drug that is currently available to treat the disease. That project will integrate molecular biomarker testing with advanced MRI imaging technologies. This project is the first phase of larger collaborative studies to develop and validate integrated care pathways, which would include laboratory diagnostics, imaging data and other clinical information to be used in the management of patients with brain cancer and neurological diseases.

UCSF has been at the forefront of the movement toward precision medicine, for which UCSF Chancellor Susan Desmond-Hellmann, M.D., M.P.H., co-authored the initial National Academy of Sciences paper that defined the new field. That paper set the vision of harnessing the vast amounts of genetic, environmental and health data worldwide to make health care more predictive, precise and targeted.

“There are many diagnostics projects underway at UCSF for which Quest could partner and contribute a great deal of value in turning an isolated research project into a diagnostic service or other technology that directly benefits patients,” said June Lee, M.D., F.A.C.C.P., director of early translational research at the UCSF Clinical and Translational Science Institute, which initiated the collaboration with Quest after several scientists from both organizations had formed isolated, but successful, research collaborations. “This agreement will give UCSF researchers access to Quest expertise in developing diagnostics, as well as in understanding the market conditions for projects on campus.”

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