Fall 2013

YOUR DOORWAY TO SCIENTIFIC DISCOVERY

Unlocking the Mysteries of the Brain

The Role of Big Data in Medical Research Developing Nanotechnology Therapies for Traumatic Brain Injuries

VOLUME 3 NUMBER 4 | FALL 2013

www.sanfordburnham.org | PORTAL

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FOUNDERS

A Message from Trustee Peter Preuss 1

Dr. William H. and Lillian Fishman

Unlocking the Mysteries of the Brain 2

HONORARY TRUSTEES

Talking with a Scientist: Judith Altarejos, Ph.D. 5

Roberta and Malin Burnham Joe Lewis Conrad T. Prebys T. Denny Sanford

Big Data and Medical Research 6

Trustees and Officers

Gregory T. Lucier

Partners in Science: Gayle Wilson 4

Nanotechnology Therapies for Traumatic Brain Injuries 7 Partnership Highlights 8 Presidential BRAIN Initiative 9 Remembering Duane Roth 10

CHAIRMAN

Recent Events 11

Duane J. Roth († 2013)

Upcoming Events 12

Vice CHAIRMAN

Community Outreach 13

Kristiina Vuori, M.D., Ph.D. President and Interim CEO

Join us for a Tour of Sanford-Burnham Back Cover

PROFESSOR AND PAULINE AND STANLEY FOSTER PRESIDENTIAL CHAIR

Gary F. Raisl, M.B.A., Ed.D. Executive Vice President Chief Administrative Officer Chief Financial Officer

Interim Senior Vice President, External Relations

Treasurer

Blair Blum

Margaret M. Dunbar, J.D. Secretary

YOUR DOORWAY TO SCIENTIFIC DISCOVERY

Lorenzo Berho James C. Blair Shehan Dissanayake, Ph.D. Daniel J. Epstein M. Wainwright Fishburn, Jr. Pauline M. Foster Patrick J. Geraghty Bill Gerhart Alan A. Gleicher Jeanne L. Herberger, Ph.D. Brent Jacobs James E. Jardon II J. Bernard Machen, D.D.S., Ph.D. Hank Nordhoff Douglas Obenshain Peter Preuss Ze’ev Ronai, Ph.D. Andrew J. Viterbi, Ph.D. Allen R. Weiss Luder G. Whitlock, Jr. Gayle E. Wilson Todd Golub, M.D. ScienTIFIC Advisory Board Chairman EX-OFFICIO

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Vice President, CoMmunications

Deborah Robison

VICE PRESIDENT, EXTERNAL RELATIONS

Edgar M. Gillenwaters

VICE PRESIDENTS, PHILANTHROPY

Paul Baker Philip Graham, M.B.A. Editor

Patrick Bartosch

Unlocking the Mysteries of the Brain

Contributors

The Role of Big Data in Medical Research Developing Nanotechnology Therapies for Traumatic Brain Injuries

VOLUME 3 NUMBER 4 | FALL 2013

Karolyn Baker Kate Callen Molly Townsend

ON THE COVER

Design

www.sanfordburnham.org

Toll-free: 1-877-454-5702

Rendering of the human brain with neurons in the background.

Creative Fusion

Sanford-Burnham Medical Research Institute

10901 North Torrey Pines Road, La Jolla, CA 92037 • 858-646-3100

Sanford-Burnham Medical Research Institute at Lake Nona 6400 Sanger Road, Orlando, FL 32827 • 407-745-2000

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A Message from

Trustee Peter Preuss At Sanford-Burnham, scientists are investigating a wide variety of health problems associated with the brain. We are exploring innovative treatments to improve the lives of patients with brain disease and injury. In this issue of Portal, you will learn about the challenges facing scientists in the field of brain-health research, and the dynamic strategies that are leading to new understandings of this crucial part of the human body.

The brain gives us consciousness, interprets our senses, and allows us to think – and we are just now beginning to discover its many other functions.

Recent scientific advances are redefining the way we understand brain function, and are narrowing the gap between laboratory research and patient care.

You may have heard about the recent findings by Sanford-Burnham’s Stuart A. Lipton, M.D., Ph.D. After decades of research, Dr. Lipton and his team have developed the first experimental drug to boost brain synapses lost in Alzheimer’s disease. This groundbreaking discovery sheds new light on how the brain works and how diseases of the brain develop. It will hopefully translate into therapies to help both early and later-stage Alzheimer’s patients.

I understand the urgency for new therapies to improve the lives of those suffering from brain disease. Through The Preuss Foundation for Brain Tumor Research, I have had the opportunity to create an environment for scientists to work collaboratively to tackle some of the world’s greatest medical challenges. Since 1985, the Preuss Foundation has hosted and

Sanford-Burnham president and interim CEO, Kristiina Vuori, M.D., Ph.D., with Peter Preuss

sponsored 38 seminars and conferences that serve as forums for the world’s preeminent brain-tumor researchers. These events have offered scientists, including some of Sanford-Burnham’s researchers, unique opportunities to discuss their research challenges and catalyze innovation.

The most recent seminar was hosted by Sanford-Burnham at the La Jolla campus.

I am inspired, and I hope you are too, by the Institute’s research into understanding the brain’s capacity to be strengthened and repaired. It is a promising time in this field of medical research and we are excited to share these stories with you.

– Peter Preuss honorary trustee

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Unlocking the

Mysteries of the Brain The brain may be the most fascinating organ of the human body, but it’s also one of the least understood. Diseases of the brain such as Alzheimer’s and Parkinson’s are often devastating, and scientists are only now starting to understand their causes and to treat them effectively.

The human brain is by far the most complex and most mysterious organ. An estimated 100 billion nerve cells (neurons) can be found in the human brain, all of them connected to hundreds or even thousands of other neurons through connection points known as synapses.

As the center of the nervous system, the brain can be viewed as the “control room” of our body.

It controls the other organs through nerve signals and by driving the secretion of hormones. It is also the physical structure that makes up the mind, thus defining our personality, making us who we are. If something goes wrong in the brain, the impact can be devastating.

This issue of Portal is devoted to the topic of brain health and offers an overview of current SanfordBurnham research into brain disease. Fueled by generous contributions from donors, our scientists are studying the causes of debilitating diseases such as Alzheimer’s or Parkinson’s and searching for the treatments and cures of tomorrow. Nearly every

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family has endured the heartache of a loved one becoming lost in the fog of cognitive impairment. We are pursuing ways to lift that fog and restore those patients to fully functioning lives.

A MULTI-DISCIPLINARY APPROACH TO STUDYING BRAIN HEALTH

Scientists in the Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research form the core of our brainrelated research. However, given the brain’s impact on other areas of the body, a number of laboratories are investigating how this crucial organ impacts other diseases. For instance, brain tumors are being studied in our NCI-designated Cancer Center and brain hormones’ influence on metabolism in our Diabetes and Obesity Center, among others. This epitomizes Sanford-Burnham’s multidisciplinary approach to studying disease across teams and centers.

In the Del E. Webb Center, researchers seek to understand the fundamental molecular changes responsible for cell

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Stuart Lipton, M.D., Ph.D., is professor and director of the Del E. Webb Center

Evan Snyder, M.D., Ph.D., is professor and director of the Stem Cells and Regenerative Biology Program

Huaxi Xu, Ph.D., is professor in the Degenerative Disease Research Program

and tissue degeneration in the nervous system and other organs. They strive to discover new medicines that protect the brain as well as develop regenerative brain, heart, and diabetes therapies using stem cells. Another focus is to understand the biology of embryonic stem cells, which give rise to the many different types of cells in the body. Scientists there also reprogram adult cells of any tissue type to become induced pluripotent stem cells (iPSCs), a specially engineered type of stem cells that have a lot in common with their embryonic cousins.

approaches have focused on plaques and aggregates of a certain protein in the brains of Alzheimer’s patients, but with little success. This study, however, focused on a completely different target.

“This is very exciting because everyone is looking for an earlier treatment of the disease,” Lipton says.

“These findings actually mean that you might be able to intercede not only early but also a bit later.” That means Alzheimer’s patients may be able to have synaptic connections restored even with plaques and tangles already in their brains.

treatment. It’s amazing,” says Xin Wang, a graduate student in Xu’s lab and first author of the study. “Gene therapy of this sort hasn’t really panned out in humans, however. So we’re now screening small molecules to look for some that might increase SNX27 production or function in the brain.” If effective, these molecules could one day form the basis of a new drug.

A NEW WAY TO TREAT ALZHEIMER’S?

FINDING THE MOLECULAR CAUSE OF DOWN SYNDROME

stroke, neurodegeneration, developmental delay, and others. We are also interested in using stem cells in a dish to help discover better drugs with which to treat patients.”

Recently, a consortium of researchers, including Sanford-Burnham, showed that transplanting neural stem cells into the spinal cord of mice with ALS, an untreatable and fatal disease of the nerve cells, slows disease onset and progression, improves motor function, and significantly prolongs survival.

You can expect more interesting brain-related news from Sanford-Burnham in the near future. Our scientists are currently working on promising new approaches to treating Parkinson’s disease as well as spinal cord injury and stroke, among others. They’re also growing cells with certain diseases in a dish to study neuropsychiatric disorders such as bipolar disorder, schizophrenia and autism.

Make sure to visit our science blog, Beaker (beaker. sanfordburnham.org), to stay updated on Institute news.

Lots of exciting news has recently emerged from our laboratories. Stuart Lipton, M.D., Ph.D., professor and director of the Del E. Webb Center, and a team of scientists from other institutions published a study in June showing that an experimental drug (NitroMemantine) reversed the loss of synapses in animal models of Alzheimer’s disease. In the past, treatment

In another study, a research team led by Huaxi Xu, Ph.D., found that the extra chromosome inherited in Down syndrome impairs learning and memory because it leads to low levels of a certain protein (SNX27) in the brain. They found that when SNX27 is elevated, cognitive function can go back to normal in animal models.

“Everything goes back to normal after SNX27

USING STEM CELLS TO TREAT NEUROLOGICAL DISEASES

The use of stem cells holds particular promise for the treatment of brain diseases.

“We believe many diseases of the brain and nervous system may be viewed as rooted in stem cells failing to do their job or going awry,” says Evan Y. Snyder, M.D., Ph.D., director of our Stem Cells and Regenerative Biology Program. “Therefore, we study how to fix those stem cells that are dysfunctional or how to replace them with stem cells and their derivatives that are normal.

This technique may present therapeutic options for a variety of diseases, including cancer, injury,

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PARTNERS IN SCIENCE

Gayle and Pete Wilson, the former governor of California

Gayle Wilson Many San Diegans will recognize Gayle Wilson as the former First Lady of California, but what they may not know is her lifelong advocacy for science. As an avid supporter of SanfordBurnham for over two decades, and a Trustee since 2007, Wilson believes in the value of investing in science and appreciates the critical role science education plays for our nation’s youth.

Her interest in science began early on while growing up in Phoenix, Arizona. As a high-school senior, she was named one of 40 national finalists in the Westinghouse Science Talent Search (now known as the Intel Science Talent Search), a researchbased science competition that provides the opportunity for aspiring scientists to present original research to nationally recognized

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professional scientists. The award had a remarkable impact on Wilson’s academic ambition, and led her to pursue a Bachelor of Arts degree in Biology from Stanford University. After Stanford, she married Pete Wilson and later moved to San Diego, where she became involved with both the Junior League and the Achievement Rewards for College Scientists (ARCS).

Marrying then-Senator Pete Wilson in 1983, Wilson moved to Washington, D.C., where she pursued her interests in math and science education and early childhood health. As First Lady of California during Pete Wilson’s tenure as governor from 1991-1999, Wilson served as the founding chairwoman of The California State Summer School for Mathematics and Science (COSMOS), a merit-based summer school designed to encourage talented highschool students to continue and expand upon their interest in math and science.

“COSMOS offers students the opportunity to explore topics beyond the typical high-school curriculum,” she says, “It provides students a chance to develop the skills, inspiration, and confidence vital to keeping up with the fast-paced world of science.” Now in its 15th year, COSMOS is preparing students for careers in mathematics and sciences on four campuses of the University of California – Davis, Irvine,

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San Diego, and Santa Cruz.

When Wilson first became involved with Sanford-Burnham in the early 1980s, she was most impressed by the overall quality of the Institute’s fundamental research into the molecular causes of disease.

“I believe strongly in supporting basic research that is the underpinning of major scientific discoveries,” she says.

“These advances will lead to curing disease, prolonging lives, and improving the overall quality of life.”

Wilson recognizes the challenges many scientists are facing in the field of brain health and has witnessed the impact of brain diseases like Alzheimer’s and Parkinson’s on her community. She believes in the power of providing support to medical research, which can work to cultivate scientific innovation and lead us closer to cures.

“The private sector must be a strong partner with the government in supporting research. As a Board member, I feel it’s critical to show my support for Sanford-Burnham through personal monetary contributions as well as by recruiting others to support this great institution.”

An act of generosity on the part of one caring person can transform the lives of so many people. With the help of philanthropists like Wilson, our scientists can accelerate the progress of their work, catalyzing the translation of scientific research into life-saving therapies.

Talking with a Scientist:

Judith Altarejos, Ph.D. The prevalence of obesity in the U.S. is a key contributor to the incidence of metabolic-related diseases such as diabetes, cardiovascular disease, and even cancer.

The American Medical Association (AMA) recently acknowledged obesity’s role by classifying it as a disease – a decision that has fueled renewed interest in discovering the biology of the disorder.

“We’ve known for a long time that the brain controls body weight and blood sugar,” says Judith Altarejos, Ph.D., assistant professor in Sanford-Burnham’s Diabetes and Obesity Research Center. However, investigating the brain’s role fell out of favor as researchers shifted their attention to organs and tissues when it became apparent that the pancreas produces the blood sugar-regulating hormone, insulin. The discovery of leptin, a hormone secreted by fat, in the mid-1990s, sparked renewed interest in the brain’s ability to control body weight and blood-sugar levels.

The brain has the capacity to modulate appetite, blood glucose levels, and energy expenditure. Different regions of the brain regulate body weight homeostasis, while other regions have an important role in regulating metabolism. However, in both obesity and diabetes the brain communication pathways may not be functioning properly. By studying the brain’s complicated circuits, researchers hope to figure out where a switch is broken and then fix, or bypass, this malfunctioning

Top: Judith Altarejos, Ph.D., is assistant professor in the Diabetes and Obesity Research Center. Inset: Altarejos and her lab team.

switch to restore the brain’s ability to regulate body weight or blood glucose.

Altarejos says that the process is extremely daunting because of the complexity of the brain. Her research focuses on CRTC1, a protein that regulates body weight through its functions in the hypothalamus. Stored body fat releases leptin, which turns on CRTC1 activity in the brain and in turn decreases appetite. In obesity, communication between leptin and CRTC1 is lost and the brain doesn’t get the message to reduce hunger, effectively telling the body to keep eating. Greater understanding of the wiring in the brain will lead to specific therapies targeting misfiring brain circuits.

Brain imaging will be a valuable diagnostic technique to overcome the challenge of examining the human brain. “We’re trying to answer disease-related questions about a complex system that is hard to mimic in a petri dish,” says Altarejos. While scientists are making strides in resolving some pieces of the puzzle, it will be necessary to innovate research techniques in order to probe the brain’s intricate networks. www.sanfordburnham.org | PORTAL

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TALKING WITH A DONOR

Developing Nanotechnology Therapies for Traumatic Brain Injuries

Big Data and Medical Research When scientists first sequenced the human genome a decade ago, it was hailed as an achievement that would transform biology and the way scientists tackle new problems.

While it has yet to fulfill the prophecy of revolutionizing the diagnosis and treatment of most, if not all, human diseases, it has helped scientists better understand the health and medical needs of people based on their individual genetic blueprints. It has also spawned the creation of vast information sets that have come to be known as “Big Data.”

Big Data refers to our ability to collect and analyze the massive amounts of information we now generate. For medical researchers, the goal is to use that information to take a macroscopic view of health, including the ability to recognize patterns of disease development. These new scientific endeavors are generating immense amounts of information.

Scientists at Sanford-Burnham use technology platforms, called the “omics”, that are generating an expansive amount of data. In a field called systems biology, these platforms look at the larger picture of biology in a more holistic fashion, including genomics (the study of genes and their functions), proteomics (the study of an organism’s complete complement of proteins), and metabolomics (the study of the differences between biological samples based on their metabolite profiles), among others.

Analyzing this data will lead to a better understanding of disease processes and potentially more solutions for treating myriad conditions. The data will be useful for diagnostics, especially early cancer detection.

Profiling a person’s proteome or metabolome

Erkki Ruoslahti, M.D., Ph.D., and his team study nanotechnologies as treatments for brain injuries

can help clinicians see global changes in the body far in advance of symptoms. Big Data will also have a major impact on personalized medicine. Rather than a one-size-fits-all approach, therapies will be tailored to an individual’s disease based on his or her genetic, proteomic, or metabolomic profile.

While it has become an indispensable tool for researchers, Big Data carries challenges for the scientific community. Collecting and storing data are easier tasks than figuring out what information is important and, more critically, how to use the information.

The question is how to harness the vast quantities of data that will be generated in the future. Big Data already speeds up every process scientists currently perform, so they can focus on the creative part of their work.

And, in the end, that pays off for everyone.

The U.S. Defense Advanced Research Projects Agency (DARPA) recently awarded $6 million to a team of researchers to develop nanotechnology therapies for the treatment of traumatic brain injury and associated infections. The award brings together a multi-disciplinary team of renowned experts in laboratory research, translational investigation, and clinical medicine. The team includes SanfordBurnham’s Erkki Ruoslahti, M.D., Ph.D., and is led by Michael J. Sailor, Ph.D., from

the University of California, San Diego.

Ballistics injuries that penetrate the skull accounted for 18 percent of battlefield wounds sustained by troops who served in Iraq and Afghanistan. A major contributor to the mortality associated with a penetrating brain injury is the elevated risk of intracranial infection.

Projectiles drive contaminated foreign materials into neural tissue. Under normal conditions, the brain is protected from infection by a physiological system called the blood-brain barrier.

Unfortunately, those same natural defense mechanisms make it difficult to get antibiotics to the brain once an infection has taken hold.

Several types of nanoparticles have already been approved for clinical use in patients, but none for treatment of trauma or diseases in the brain. This is due in part to the inability of nanoparticle formulations to cross the blood-brain barrier and reach their intended targets.

“Poor penetration into tissues limits the application of nanoparticles to the treatment of many types of diseases,” said Ruoslahti, distinguished professor in our NCIdesignated Cancer Center. “We are trying to overcome this limitation by using targeting molecules that activate tissue-

specific transport pathways to deliver nanoparticles.”

Treating brain infections is becoming more difficult as drug-resistant strains of viruses and bacteria have emerged. Because drugresistant strains mutate rapidly, researchers must constantly adjust their approach to treatment. In an attempt to hit an evolving target, the team is creating modular delivery systems, so they can be reconfigured “on the fly” with the latest therapeutic advances.

Nanocomplexes that contain genetic material known as short interfering RNA, or siRNA, will be key to this aspect of the team’s approach.

The function of this type of RNA is that it specifically interferes with processes in a diseased cell. The advantage of RNA therapies is that they can be quickly and easily modified when a new disease target emerges.

But effective delivery of siRNA-based therapeutics in the body has proven to be a challenge because the negative charge and chemical structure of siRNA makes it very unstable in the body, and it has difficulty crossing into diseased cells. To solve these problems, scientists developed nanoparticles that form a protective coating around siRNA. The nanocomplexes they Continued on Page 8

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Partnership Highlights

Presidential BRAIN Initiative to Transform Brain Science

Johnson & Johnson (Janssen Pharmaceutical)

Johnson & Johnson subsidiary Janssen Pharmaceutical Research and Development is collaborating with SanfordBurnham in a three-year effort to identify lead compounds targeting novel pathways involved in Alzheimer’s disease and major psychiatric disorders like depression, schizophrenia, and bipolar disorder. These diseases together affect more than seven million Americans and, although some treatment options exist, a high unmet medical need remains.

Intrexon

Sanford-Burnham is partnering with Intrexon Corporation, a leading synthetic-biology company, to accelerate the Institute’s stem-cell research. As part of the agreement, Sanford-Burnham scientists provide Intrexon with expertise in the field of induced pluripotent stem cells (iPSCs; stem cells produced in the laboratory by genetically reprogramming any adult cell, such as a skin cell). In turn, Intrexon shares its knowledge of how best to use its technologies. SanfordBurnham is currently building a vast collection of human iPSCs generated from individual patients and healthy volunteers.

Pfizer

We recently announced a new collaboration with Pfizer to identify therapeutic targets for insulin resistance and diabetes. The research teams will utilize Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics to robotically analyze hundreds of thousands of compounds in search of the few that may help treat disease. When promising compounds have been identified, Sanford-Burnham and Pfizer scientists will characterize and further study the “hit” compounds to understand their mechanism of action. These compounds will then be used as “probes” to identify novel targets for the treatment of diabetes.

Continued from Page 7

Nanotechnology are developing shield the negative charge of RNA and protect it from nucleases that would normally destroy it.

DARPA often uses the term ‘DARPA-hard’ to refer to problems that are extremely tough to solve. What makes this a DARPA-hard problem is the fact that it is so difficult to deliver therapeutics to the brain. But for scientific pioneers like Ruoslahti, a tough biomedical problem is a very exciting research opportunity – and, in this case, a chance to repay a nation’s debt to young heroes who have sacrificed so much.

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When President Obama announced the formation of the BRAIN (Brain Research through Advancing Innovative Neurotechnologies) Initiative in April 2013, he said the $100 million initiative would be a “transformative” vehicle that would give scientists the tools they need to unlock the “enormous mystery” that is the human brain.

The BRAIN Initiative is part of a presidential focus aimed at revolutionizing our understanding of how individual cells and complex neural circuits in the brain act in both time and space. “Many regard understanding how the human brain works as the last great frontier in biomedical research,” said Story C. Landis, director of the National Institute of Neurological Disorders and Stroke (NINDS), which oversees the project, in a statement.

“Much remains to be done before we have a dynamic picture of the brain in action and understand how we are able to see, hear, reason, learn, and remember.”

Among the possible long-term outcomes of the project, NINDS says, are improved treatments for neurodegenerative diseases and the development of solutions to prevent, treat, or possibly reverse the effects of post-traumatic stress disorder and traumatic brain injuries.

“When the president of the United States declares that imaging the brain is a high priority, it says that understanding the nervous system—the brain, in specific—is a national imperative,” says Evan Snyder, M.D., Ph.D., professor in the Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research at Sanford-Burnham.

While the BRAIN Initiative has several key private-sector partners—the Allen Institute for Brain Science, the Salk Institute, the Kavli Foundation, and the Howard Hughes Medical Institute—other research organizations,

including Sanford-Burnham, will benefit from and participate in the project.

Stuart Lipton, M.D., Ph.D., director of the Del E. Webb Center, says Sanford-Burnham’s electrophysiology and optogenetics technologies, funded by NINDS, will play a role in the BRAIN Initiative by offering its resources to others involved in BRAIN research efforts. These technologies look at electrical activity in the brain through multi-electrode array devices that contain multiple plates through which neural signals are obtained and delivered, essentially serving as neural interfaces that connect neurons to the brain’s electrical circuitry.

“Through these resources, we look forward to participating in the BRAIN Initiative,” says Lipton. “The BRAIN Initiative talks about the subtlety of brain diseases, and through our technology, core facilities, and research, we do subtlety and nuance in brain diseases really well.”

While the BRAIN Initiative is focused more on the normal, healthy brain, Lipton says its basic-science approach will be important for disease states. And that melds nicely with Sanford-Burnham’s translational-oriented science, which aims to move basic discoveries from the laboratory to the patient’s bedside more quickly.

The Initiative will lead to an increased awareness of the brain, which Lipton and Snyder agree is essential. “This will have an effect on educating the public about the brain, and that’s very important,” says Lipton. And very transformative. www.sanfordburnham.org | PORTAL

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Recent RECENT EVENTS

“Duane’s involvement with the Institute inspired confidence and influenced me as I considered how I would contribute to medical research in San Diego.”

– Denny Sanford

“Duane was a great personal friend and colleague. On many fronts, his amazing leadership helped significantly elevate San Diego to the national stage.”

– Malin Burnham

Remembering

San Diego Padres VIP Event

Duane Roth

On August 6, a group of Sanford-Burnham donors enjoyed a day in the Owner’s Box at Petco Park to watch the San Diego Padres play the Baltimore Orioles. A moment of silence was held in memory of Duane Roth and participants shared their thoughts and memories of Duane.

By Kristiina Vuori, M.D., Ph.D., president and interim CEO

I was shocked and deeply saddened to hear that our friend and colleague Duane Roth passed away on August 3. He died from complications of head injuries suffered in a bicycling accident.

I speak on behalf of everyone at SanfordBurnham when I say that we will all miss him dearly. In a sad convergence of science and life, I am writing this tribute in an issue of Portal, that focuses on the topic of brain health.

He was an amazing friend, a leader, and a mentor to all of us, and a true giant in the San Diego community and beyond. We greatly appreciated the vision and experience he contributed to our Institute.

Duane was serving as vice chairman of Sanford-Burnham’s Board of Trustees and was to assume the role of Board chairman in September. While well positioned to continue its regular duties, the Board will soon decide on new Board leadership.

Of key importance this past year, Duane provided invaluable counsel in the development of Sanford-Burnham’s 10-year strategic vision. He worked closely with Institute leaders to develop a plan to enhance our stature as a world-class biomedical research institute while advancing the Institute’s drug discovery capabilities and translational focus.

Duane’s early input will guide the Institute’s strategic direction for years to come.

Duane’s devotion to making organizations thrive and his tireless efforts to make the world a better place has inspired many. We join all those whose saddened hearts go out to Duane’s wife and partner Renee, and to the Roth family.

We dedicate this issue of Portal to Duane.

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Left to right: Peter Seidler, Malin Burnham, The Honorable Pete Wilson, Ron Fowler, and Dan Epstein

Duane Roth was such a positive force for Sanford-Burnham and for San Diego. It’s going to be hard to maintain the same momentum without him. But I know Duane would have wanted us to be bold, to follow the strategic vision he helped construct, and to address the tough issues, as he always did.”

– Gayle Wilson

Careers in Science Symposium

In July, students and faculty from all over Florida attended the Central Florida Careers in Science Symposium at Sanford-Burnham at Lake Nona. The symposium included panel discussions with faculty and core directors, a networking lunch, and career-development presentations from established researchers.

Participants at this year’s Careers in Science Symposium

Graduate School Retreat

In late May, graduate students from the Sanford-Burnham Graduate School of Biomedical Sciences attended the 4th annual program retreat in Palm Springs, Calif.

Students had the opportunity to present to their peers on the theme of “Translational Medicine: From Lab to Clinic.” The retreat encouraged collaboration, discussions, and offered students an opportunity to enhance their presentation skills.

Graduate students at their retreat in Palm Springs, Calif.

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Upcoming UPCOMING EVENTS

October 2-4

The Atlantic Meets the Pacific

Community Outreach Joe Negron Visit

In July, Florida State Senator Joe Negron visited Sanford-Burnham at Lake Nona. His visit included a tour of the Conrad Prebys Center for Chemical Genomics, led

by Drs. Stephen Gardell and Layton Smith.

Sanford-Burnham is taking part in The Atlantic Meets the Pacific, an annual event for top thought leaders in technology, the sciences, and health. The program takes place in San Diego and will showcase progress being made on the frontier of health research. Participants also have the opportunity to take a tour of our La Jolla campus for a behind-the-scenes look at our research labs.

Learn more at atlanticmeetspacific.com

October 26 & 27

Pedal the Cause

Florida State Rep. Joe Negron (right) with Sanford-Burnham’s Layton Smith, Ph.D.

Join us for Pedal the Cause San Diego, a cycling fundraiser that will provide funding for the best cancer research ideas at all three of San Diego’s NCI-designated Cancer Centers: UC San Diego Moores Cancer Center, Sanford-Burnham Medical Research Institute, and the Salk Institute. One hundred percent of the proceeds stay in San Diego to fund lifesaving research. Sign up today at pedalthecause.org. Not able to attend the race? Sign up as a virtual rider to show your support!

November 2

Join us On the Track to Discovery at our Annual Gala It’s time to place your bets and mark your calendars on Saturday, November 2, for SanfordBurnham’s annual gala, On the Track to Discovery, to be held at the Del Mar Country Club.

This year’s gala will showcase the merriment, high energy, and inspirational spirit of the Triple Crown of Thoroughbred Racing. You’ll be in the money for a trifecta event, with delectable drinks, exquisite eats, and dynamite dance music.

We look forward to your joining Honorary Gala Chairs Roberta Burnham, Pauline Foster, Madeleine Pickens, and fellow Sanford-Burnham fans for an unforgettable evening cheering research down the homestretch.

Visit sanfordburnham.org/gala

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The seminar takes place every second Thursday of the month.

Students from this summer’s research program.

Our Lake Nona summer interns in the lab.

Stem Cell Consortium Seminar Series

Preuss School UCSD Summer Research Program

Lake Nona Summer Interns

Sanford-Burnham’s Southern California Stem Cell Consortium seminar takes place every second Thursday of the month at the Sanford Consortium for Regenerative Medicine.

The seminars provide a venue for collaboration and discussion on biomedical research. Speakers are invited from across the country. The public is invited to attend.

The summer research program is an intensive two-week program that offers eleven Preuss School UCSD students the opportunity to participate in summer research at Sanford-Burnham’s La Jolla campus. Guided by Sanford-Burnham scientists, the high-school students learned about genetic analysis, applied sciences, and scientific research.

Seven students from Central Florida high schools were selected to participate in summer internships at Sanford-Burnham’s Florida campus. Students worked one on one with scientists to practice laboratory methods, basic chemistry concepts, and applied research techniques.

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Join us for a Tour of Sanford-Burnham in La Jolla

We received an overwhelming response to our last quarterly tours and we are looking forward to welcoming more new guests on Wednesday, December 4, 2013. We invite you to join us for a behind-the-scenes tour of our La Jolla campus to explore the world of medical research.

Tours are free of charge and include an overview of the Institute’s history, followed by a visit to our stem cell research center and the Conrad Prebys Center for Chemical Genomics, our ultra-high-throughput screening facility. Learn how robots screen chemical compounds by the millions to find the few that could potentially be developed into the medicines of tomorrow.

To reserve your spot on the 10 a.m. or 2 p.m. PT tour on December 4, please visit supportus.sanfordburnham.org/tours.

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