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THE NEXT

DECADE Speeding Discoveries to Patients Building High-Yield Partnerships The Power of Philanthropy

www.sanfordburnham.org | PORTAL

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FOUNDERS

A Message from Board Chairman Greg Lucier 1

Dr. William H. and Lillian Fishman

Crafting the 10-Year Vision 2

HONORARY TRUSTEES

Building High-Yield Partnerships 4

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

The Power of Philanthropy: The Hervey Family Gift 6

TRUSTEES AND OFFICERS

Gregory T. Lucier

Speeding Discoveries to Patients 2

Q&A with Trustee Bill Gerhart 8 Lake Nona Celebrates First Endowed Chair 8 Remembering Lillian Fishman 10 2013 Gala: On the Track to Discovery 11

CHAIRMAN

The Next Generation 12

Duane J. Roth († 2013)

2013: The Year in Headlines 13

VICE CHAIRMAN

Partners in Science: Grant Hill Back Cover

Kristiina Vuori, M.D., Ph.D. PRESIDENT AND INTERIM CEO 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, M.S., J.D.

VICE PRESIDENT, COMMUNICATIONS

SECRETARY

Lorenzo Berho James C. Blair, Ph.D. 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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Deborah Robison

VICE PRESIDENT, EXTERNAL RELATIONS

THE NEXT

DECADE Speeding Discoveries to Patients

Edgar M. Gillenwaters

VICE PRESIDENTS, PHILANTHROPY

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

Building High-Yield Partnerships

Kate Callen

The Power of Philanthropy

CONTRIBUTORS

Karolyn Baker Patrick Bartosch James Short

ON THE COVER

DESIGN

www.sanfordburnham.org

Toll-free: 1-877-454-5702

Our 10-year vision will build on Institute strengths to redefine the medical research organization of the 21st century.

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 the Chairman Gregory T. Lucier Last year, Sanford-Burnham’s leadership team met with a select group of advisors to map out a future strategy for expanding the Institute’s global impact. I was leaving the Board of Trustees after completing my term as chair, but at the urging of then-chair Wain Fishburn, vice chair Duane Roth and president Kristiina Vuori, I came back to the Institute to participate in the strategic planning initiative. It was an amazing experience. We took a fresh look at Sanford-Burnham’s mission and core strengths, and we envisaged how we could redefine the biomedical research organization of the 21st century. As you will read in this issue of Portal, the resulting 10-year vision is targeted, tactical, and aspirational. Over the next decade, Sanford-Burnham will parlay its worldclass scientific prowess to new heights of translational research breakthroughs. We are now on track to achieve two paramount goals: • First, we will leverage our expertise in human biology to speed discoveries to patients. • Second, we will create new highyield partnerships in health care and drug development. This fall, I returned to the Board to take up Duane’s mantle as chair. Duane believed that the pursuit of biomedical discoveries is so important that it must be done big and it must be done right. Carrying out the 10-year vision that he helped create will honor his memory, and it will propel us toward the fulfillment of our Institute’s quest: “From Research, The Power To Cure.”

“The 10-year vision is targeted, tactical, and aspirational.” I strongly believe in the 10-year vision because I have such profound confidence in the entire Sanford-Burnham team of scientists, executives, partners, and supporters. Our benefactors are key players in the collaborative process. Their generosity and enthusiasm are fueling our optimism for the future. The next decade will be an extraordinary chapter in the history of this remarkable Institute. I’m so glad that you are joining us at this threshold moment, and I thank you for everything you do for Sanford-Burnham. Sincerely,

Gregory T. Lucier CHAIRMAN, SANFORD-BURNHAM BOARD OF TRUSTEES CHAIRMAN AND CEO, LIFE TECHNOLOGIES

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THE NEXT DECADE

Speeding Discoveries to Patients Crafting the

10-Year Vision When Sanford-Burnham leaders and advisors sat down to envision the next decade, they began with the core elements of the Institute’s phenomenal success over nearly four decades: teamwork, technology, and talent. With its “no silos” culture Wainwright Fishburn Jr. of collaboration, investment in high-throughput robotic technology, and recruitment of all-star scientists, Sanford-Burnham is poised to accelerate into the future. The 10-year vision lays out specific strategies to achieve new heights of distinction. “We will double down on what we do best,” said Wainwright Fishburn Jr., who guided the 2012 initiative as Board chairman. “We will broaden our approach to translation. We will catapult drug development in ways never done before. We will emerge as the partner of choice for pharmaceutical companies.” In addition to Fishburn, a partner in Cooley LLP with expertise in creating life-science companies, the members of the Special Advisory Committee consulting with Kristiina Vuori, M.D., Ph.D., President of Sanford-Burnham, and John C. Reed, M.D., Ph.D., then-CEO of Sanford Burnham, were: • Jim Blair, Ph.D., founding partner, Domain Associates • Shehan Dissanayake, CEO, Tavistock Ventures • Greg Lucier, chairman and CEO, Life Technologies • James Madara, M.D., CEO and executive vice president, American Medical Association • John Mendelsohn, M.D., past president, MD Anderson Cancer Center • Steven Paul, M.D., past president, Lilly Research Laboratories

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Robert Wechsler-Reya Robert WechslerReya, Ph.D., is one of the nation’s top stem-cell scientists. As he explains below, his vocation and his passion are the same: shorten the distance between his discoveries and new treatments for pediatric cancer.

Medulloblastoma is a childhood brain tumor that develops when signals that control cell division break down and cells divide too much. Drugs used to eradicate cancer can leave children with cognitive impairments for the rest of their lives. Research in the Tumor Initiation and Maintenance Program has focused on eradicating the cells that sustain tumor growth, the cancer stem cells. We recently found that cancer stem cells in medulloblastoma have higher levels of certain enzymes that allow cells to divide. This presents a new route to attack tumors more strategically. We reported this fall that we could use a new set of drugs to curb these regulatory enzymes and block the growth of cancer cells. We hope to move our research into the realm of personalized medicine by tailoring therapies to each patient’s unique tumor. I work with clinicians at Rady Children’s Hospital here in San Diego. We can take tumor cells straight from the operating room to the laboratory and implant them into mice to create “avatars” that we can use to preserve


and study each tumor. Then, with the high-throughput screening capacity of our Conrad Prebys Center for Chemical Genomics, we can quickly test thousands of drugs to determine which agents are effective at killing the tumor in that child. I couldn’t have envisioned doing this kind of work before I came here. At SanfordBurnham, scientists studying different aspects of cancer biology come together to connect the dots, asking questions like, “What if we look at your pathway in my cancer model?” And the drugscreening power of the Prebys Center gives us a platform for translational discoveries that is paradigm shifting. Two days ago, I met a woman whose daughter died of a rare brain cancer. She’s starting a foundation to support research to fight the disease that killed her child. I told her our research findings might be applicable. When you look into the eyes of a parent who has endured this, you have to ask, “How can we not do everything in our power to help these families?”

Pamela Itkin-Ansari

Pamela Itkin-Ansari, Ph.D., studies diseases of the pancreas in Sanford-Burnham’s Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research. As she describes below, she has come up with a novel treatment approach to freeing children with diabetes from insulin dependence. Patients with type 1 diabetes do their best to control their blood sugar with multiple insulin injections a day. But that can’t match the regulation that a working pancreas performs every second of the day. We can achieve that control by

transplanting pancreatic islet cells into diabetic patients. But those patients need to be immunosuppressed to prevent the body from rejecting the new cells. That’s not a good therapeutic trade-off for a child. As we searched for a way to deliver insulin-producing cells without triggering the immune system, we became interested in a pouch-like device made of a material similar to Gore-Tex®. It was developed 20 years ago to encapsulate and deliver islet cells, but the cells couldn’t survive inside it. So it was set aside, and it has been sitting on the shelf for two decades. We realized that the device might work with new stemcell technology. So we investigated encapsulating stem cell-derived pre-islet pancreatic cells. Those cells are destined to become islet cells, and they are hardier than mature islet cells. We hoped they would survive inside the pouch after transplantation and mature to become functional islet cells. That is exactly what has happened in our pre-clinical research. By the time children with type 1 diabetes turn 6, they may have had 10,000 insulin shots and 16,000 finger pricks to test blood sugar. I’ve gotten to know many of them, and I want to relieve them of the constant anxiety about what they’ve eaten or whether they’ve taken their insulin. I’m thrilled to think that, by linking an old device and a new technology, we may have a therapy that could give these children carefree lives. www.sanfordburnham.org | PORTAL

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THE NEXT DECADE

Building High-Yield Partnerships Michael Jackson

Michael Jackson, Ph.D., joined Sanford-Burnham in 2009 at a time when federal support for basic research was waning. One of his early priorities as the new vice president for drug discovery was to scout for potential partner organizations that shared the Institute’s mission and offered complementary strengths. He found such a partner in the world-renowned Mayo Clinic. Here, he speaks about the resulting alliance that has already begun to build a pipeline of potential therapeutics aimed at diseases with serious unmet medical needs. When we first entered into discussions in May 2012, Sanford-Burnham had a large National Institutes of Health (NIH) grant for academic high-throughput drug screening that was concluding, and Mayo was considering whether to pursue drug discovery by setting up its own screening capability. It became clear that our respective strengths — world-class laboratory research and early drug discovery at Sanford-Burnham and world-class clinical innovation and medical research expertise at Mayo — would be a powerful combination. Initially, we could conduct early drug discovery together. Ultimately, we could drive the discovery and development of “first in class” drugs all the way from the bench to “proof of concept” in patients. This partnership grew rapidly. Andrew Badley, M.D., Mayo’s associate dean of Research Resources, reached out to their seven research centers seeking clinician scientists who had ideas for early-stage drug

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development. We chose one pilot project per center to work on immediately. By the end of 2012, armed with sound preliminary data, we submitted grant applications to the NIH for five of the seven projects. One application has already resulted in an NIH grant for a full-scale early drug-discovery project conducted at both institutions. We are selecting projects with the greatest potential. Our overall goal is to identify new candidate drugs that impact disease. But we have a bigger picture in sight, a much grander aspiration. We will pursue new drug candidates with the passion of clinicians and scientists who have come up with these concepts and will stay engaged with them and see them through to clinical trials. Our partnership is patient-focused; its mission is to deliver effective and safe drugs to the people who need them. In some cases, we may discover new drugs to treat diseases, and in other cases, we may repurpose current therapies for different uses.

Steven Smith

The future of personalized medicine is unfolding in a state-ofthe-art facility in Orlando, Fla., where researchers and clinicians are joining forces to fight metabolic diseases. The Florida Hospital—SanfordBurnham Translational Research Institute for Metabolism and Diabetes (TRI-MD) is an innovative model designed to “crack the code” for the epidemics of diabetes and obesity by personalizing approaches to research and treatment. The TRI-MD’s scientific director Steven Smith, M.D., describes what makes the partnership unique.


“This partnership is designed to propel investigator-initiated research toward the creation of new therapies. These projects have resulted in new federal grants and novel intellectual property. Looking to the future, there is a clear recognition that this partnership has the very real possibility of creating new therapies to improve the health of patients worldwide.” — Andrew Badley, M.D., associate dean of Research Resources at Mayo Clinic, reflecting on the potential impact of the Sanford-Burnham/Mayo collaboration

Our collaborative approach is accelerating the cross-fertilization of ideas from bench to bedside and back again. Too often, discoveries at the bench are slow to reach clinical researchers and even slower to get into the clinic. The Florida Hospital—Sanford-Burnham partnership connects clinical investigators who are identifying the causes of metabolic diseases with basic scientists who are probing into the molecular origins of diseases and developing novel treatments. Our combined faculty regularly discusses not only Sanford-Burnham discoveries but also advances at a variety of other research institutes and centers. We bring industry colleagues into these discussions at every opportunity. And we prioritize — we converge on unmet medical needs that represent the mostpressing medical problems of the day.

Using the power of Sanford-Burnham’s advanced technologies in metabolomics and genomics, the TRI-MD team approaches each problem from multiple perspectives simultaneously, and we seek alignment between multiple platforms. In this way, we know the best next steps to take to confirm key findings. This creates confidence that we’re focusing our time and resources on problems that have the greatest relevance to the human condition. My aspiration for the TRI-MD over the next decade is simple: I want this to be the first place industry and academic researchers go when they are looking to forge productive bench-to-bedside-andback partnerships. And that is already starting to happen. People are coming to Orlando to see this innovative collaborative model up close and to understand how we are able to make it work so well. www.sanfordburnham.org | PORTAL

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THE NEXT DECADE

The Power of Philanthropy

The Hervey Family Gift When a research team headed by Ze’ev Ronai, Ph.D., identified a specific gene that gives rise to melanoma, they realized they had found a new target for treating the deadly disease. They wanted to follow the scientific trail with even greater urgency, but they needed additional funding. Enter the Hervey family of San Diego. Just weeks after Ronai’s discovery was published, the Herveys made a Ze’ev Ronai $1 million gift to advance his breakthrough research. The timing was serendipitous. The family had personal experience with skin cancer, and they were eager to join the fight against it. Working with The San Diego Foundation, the Herveys assessed various melanoma research projects currently being conducted in San Diego. Intrigued and excited by Ronai’s work, they wanted to provide their support. “The Hervey family’s gift will allow us to achieve a new level of collaborative studies with an established team of investigators providing complementing expertise; together, they will be able to further advance our discoveries,” said Ronai, who is professor and scientific director of Sanford-Burnham in La Jolla. “I truly appreciate the Herveys’ generosity, and I am excited by the possibilities they have opened up for us.”

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Jean and Jim Hervey

The donor is equally excited. “When we looked at Dr. Ronai’s research, we realized that there was a gap in funding, and we decided to fill that gap,” said Matthew Hervey, whose parents, Jim and Jean Hervey, established The Hervey Family Fund. “Our gift will help move his scientific work to the patient’s bedside. We think that’s a great investment, and we’re glad to have an opportunity to share in the excitement of his future discoveries.” Ronai’s laboratory has been working for more than a decade on fundamental signaling pathways in melanoma development. In collaboration with a Yale University


pathology group led by Marcus Bosenberg, M.D., Ph.D., they determined that the PDK1 gene, which plays a key role in normal cell processes, is indispensable to the formation and spread of melanoma cells. In mouse disease models, they showed that inhibiting PDK1 shrunk melanoma tumors, blocked metastasis, and prolonged survival. Armed with this knowledge, the Ronai team, which includes Randal Kaufman, Ph.D., and Michael Jackson, Ph.D., of Sanford-

“Our gift will help move his scientific work to the patient’s bedside.”

— Matthew Hervey

Burnham, will zero in on the proteins that are regulated by the PDK1 gene. “We need to pinpoint which of numerous PDK1 targets is the culprit in mediating melanoma development,” Ronai said. “We are designing actual experiments to pursue this urgently over the next few months. The project will ultimately involve five laboratories with complementary areas of expertise. Such team science is where the future of research lies.” Ronai anticipates that the team’s work will not only pave new roads in defining the importance of the PDK1 pathway in melanoma but also identify those patients who would benefit most from this PDK1-focused approach. The best possible therapeutics for clinical evaluations of PDK1 will then be assessed.

“The Hervey gift,” he said, “is an example of how private philanthropy can drive transformational research initiatives.” The gift is the Hervey Fund’s first foray into support for biomedical research, and it was made with careful consideration. “A lot of philanthropy has personal beginnings,” said Matthew Hervey. “Skin cancer has been a personal issue for my parents and siblings. We’ve known an awful lot of people who have suffered from skin cancer and some who have died from it.” Geography was another factor. “In a broader context, life in San Diego and Southern California is about being outdoors and going to the beach,” he added. “That puts all of us here at greater risk of developing skin cancer.” Melanoma is the most deadly form of skin cancer. The National Cancer Institute ranks it as the fifth most common type of new cancer diagnosis in American men and the sixth in American women. An estimated 76,700 will be diagnosed with the disease and 9,500 will die of it in 2013. And, as Matthew Hervey noted, melanoma has a disproportionate impact on San Diego. In a study of melanoma rates in the 100 largest U.S. cities, The Daily Beast compiled a ranking of “20 Sunburn Capitals” where risks and deaths were highest. San Diego made the list and posted the highest incidence of melanoma at 29.1 per 100,000. Bob Kelly, president and CEO of The San Diego Foundation, also has a personal interest in the Hervey gift to the Ronai team. “While I led the American Cancer Society, we had a mission to rid out cancer entirely,” he said. “That drive has never faltered in me, having seen so many people battle with this silent but devastating disease. Scientists and researchers have advanced light-years, and this grant gets us even closer to a cure.” www.sanfordburnham.org | PORTAL

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Q&A with

Trustee Bill Gerhart Bill Gerhart didn’t know much about life sciences when he was asked by an old business school classmate in 2000 to help evaluate a biotechnology company. But he learned fast, and he quickly developed a knack for looking at old pharmaceutical compounds with new entrepreneurial eyes. He has been the founder and/or CEO of several biotech companies, including, most recently, Elevation Pharmaceuticals, which was sold to a large Japanese pharmaceutical company in 2012. With a track record of biotech entrepreneurship, he brings unique insights to the Institute as a donor and a new trustee. Why Sanford-Burnham? What drew you to this organization? I am impressed not only with SanfordBurnham’s premiere reputation for basic medical research but also with its 10-year vision for becoming one of the most productive independent drug discovery institutes in the nation. As federal funding for basic research shrinks, productivity at medical research institutes will increasingly be measured by success at translating discoveries into drugs, as well as by

Bill Gerhart

significant scientific breakthroughs. With a substantial investment in drug discovery capabilities, an awareness of the importance of culture as a change agent, and seasoned industry drug discoverers like Michael Jackson, Sanford-Burnham is well-positioned to leverage its strength in basic science to discover new drugs based on internal research programs as well as forge drug discovery partnerships with pharmaceutical companies.

Lake Nona Campus Celebrates First Endowed Chair We at Sanford-Burnham know how important philanthropy is to the discovery of cures. There are many different ways to support our science; one is the establishment of an endowed chair. On September 12, we installed the first endowed chair at our Orlando, Fla., campus – the Tavistock Distinguished Professor Chair for the Scientific Director of Sanford-Burnham at Lake Nona. The inaugural holder of the chair, Daniel Kelly, M.D., has played a key role in establishing the Diabetes and Obesity Research Center and

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Kristiina Vuori, Daniel Kelly, Rasesh Thakkar

recruiting top scientists from around the world. The $3 million endowment of the chair was made possible with funding from Tavistock Group, an international private investment organization that manages Medical City at Lake Nona. The Tavistock


“Translating discoveries into drugs will result in new breakthrough therapies.” — Bill Gerhart What have you learned about the Institute in your first months as a trustee? I understood the strategy and goals of the Institute. But I wasn’t aware of how ambitious the plan is for strengthening its financial base to accelerate the discovery of important disease targets and the development of new therapies. I like that. If we’re not moving forward aggressively, we’re moving backwards. Greg Lucier’s recent return as chairman will be a huge asset to Kristiina Vuori and her leadership team. Greg is one of the most accomplished life-sciences executives in our region, and he has a well-deserved reputation for making things happen.

from drug discoveries. Finally, we must evolve the culture. The new initiative, STRIVE, which seeks to educate Sanford-Burnham scientists on the criteria and process of drug discovery, is a major step forward.

In your view, what are the Institute’s top priorities going forward? High-quality basic research is a given; that’s the reputation we have now. This core competency should be nurtured wherever possible. Second, we must strengthen the financial base through partnerships with pharmaceutical companies, generous support from philanthropists, and eventually, revenue

Where do you see SanfordBurnham a decade from now? I see a future where the new paradigm at Sanford-Burnham for translating discoveries into drugs results in new breakthrough therapies for cancer, brain disease, heart disease, and diabetes – areas where we have core competencies. In 10 years, I expect that Sanford-Burnham will have migrated from validating molecular targets and discovering new molecules to validating drug candidates and building clinical proof of safety and efficacy. This will accelerate therapies to market, and it will exponentially drive value to provide a more robust revenue source for Sanford-Burnham. This vision is completely realizable given the very capable and energized leadership at the Institute. And it’s a vision I am eager to help facilitate in any way that I can.

Distinguished Professor Chair is named in honor of Tavistock Group and the Tavistock Foundation, which focuses its philanthropic support on medical services, education, and medical research. “Endowed chairs carry prestige and honor for the holders and the persons or organizations for which they are named,” said Kristiina Vuori, M.D., Ph.D., Sanford-Burnham’s president and interim CEO, at the ceremony. “They also help to advance scientific research by offering stability in a way that grant funding may not. We are extremely appreciative of this gift and the legacy that it will bestow.” Kelly is credited with attracting a prestigious faculty to the Lake Nona campus and helping

to fuel rapid growth of the region’s emerging life-science cluster. His leadership has established a state-of-the-art facility where translational research, team science, and partnerships advance discoveries to benefit patients. During the ceremony, Rasesh Thakkar, senior managing director of Tavistock Group, acknowledged Kelly’s role in putting Orlando’s Medical City on the map. He also praised the Institute for fighting today’s most challenging diseases: “Sanford-Burnham’s gift back to us as a community will far exceed what we have given. The Tavistock family feels honored to be affiliated with Dr. Kelly. He will make discoveries that we can’t even imagine yet.” www.sanfordburnham.org | PORTAL

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Remembering Lillian Fishman She was a radiant woman with a passion for big science and a devotion to young talent. Together with her husband and a modest grant, she launched a new kind of institute for high-yield biomedical research. In her final visits to her beloved campus, junior scientists left their benches to gather around her for inspiration. The extraordinary life of Lillian Fishman ended August 24 when she died peacefully at her home in La Jolla. Described by U–T San Diego as “one of the most prominent of San Diego’s biomedical pioneers,” the Institute co-founder was eulogized at a September 9 memorial service by her children Joel and Nina, her friends, and her protégés. President and interim CEO Kristiina Vuori recalled that when she joined the La Jolla Cancer Research Foundation in 1996, “we postdocs were welcomed personally by Lillian, and she let us know that our individual contributions were vital to the organization’s success.” Addressing Joel and Nina directly, Vuori said, “Your mother was a remarkable, courageous, and kind lady. Thank you for sharing her with us for so many years.” Honorary trustee Malin Burnham remembered meeting William and Lillian Fishman shortly after they opened the Institute with $180,000 in National

Cancer Institute funding. “They created an atmosphere of family and networking with no silos, and I just took to it,” he said. “Collaboration was in the DNA of this place.” Three decades later, he said, “San Diego is known as having that DNA in its whole corridor, and much of that came from the seed that was planted by the Fishmans.” The final two speakers, Joel and Nina, conveyed their mother’s deep pride in the Institute. Said Nina, “She believed that the value at the heart of Sanford-Burnham was to give scientists space and freedom to advance their research, and this has made it a built-to-last organization.” Joel concluded, “From her many friendships, she built a larger family, and all those who worked with her on this great cause will miss her dearly.”

When Lillian Soared

In 2010, Lillian Fishman traveled with family and friends to her alma mater, the University of Alberta, to receive a Distinguished Alumna Award. Malin Burnham, who arranged the trip, recalled that the 95-year-old “was the strongest I’d ever seen her.” On the flight home to San Diego, she was invited into the cockpit to sit in the jump seat between the two pilots. “Lillian, in effect, landed that airplane,” said Burnham, “and her smile went from ear to ear. She was appreciative of and interested in every experience life offered. It really was a joy to be with her.”

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Terrific friends, top-notch entertainment and tremendous generosity scored a trifecta at the 2013 Sanford-Burnham “On the Track to Discovery� gala. Held in the heart of horse country at the Del Mar Country Club, the elegant gathering delivered a big payout, raising $2.1 million for Institute research support. Honorary gala chairs Roberta Burnham, Pauline Foster, and Madeleine Pickens orchestrated a magical evening that showcased the merriment, high energy, and inspirational spirit of the Triple Crown of Thoroughbred Racing.

Denny Sanford, Kristiina Vuori, Stuart Tanz

Debra Turner, Conrad Prebys

Roberta Burnham, Renee Roth, Stephanie and Steve Williams

Heather Kowalski, Elizabeth Dewberry, Denny Sanford, Craig Venter

Garth Powis, Jeanne Herberger, Lynn Kirkpatrick

Lisa and Steven Cassidy

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“What he could not finish, we will finish.” – Shiyu Wang

Shiyu Wang

The Next Generation Eric Dudl, Ph.D., was studying cancer as a Sanford-Burnham postdoctoral fellow when a medical diagnosis revealed that he had the disease himself. The oncology investigator was just 33 when he passed away in August 2006, but he had already published several research papers elucidating how cancer works at the cellular level. Jim and Barbara Dudl chose to honor their son by establishing the annual Eric Dudl Scholarship to support top Sanford-Burnham postdocs. Each year, the Dudl family comes to campus to bestow the award and meet its recipient. At this year’s Sept. 26 ceremony, 2013 Dudl Scholarship winner Shiyu Wang, Ph.D., made a personal pledge to Eric’s parents. “What he could not finish, we will finish,” she told them. “We will continue his journey along the path to discovery, and he will live on in our work and in our hearts.” Wang works in the laboratory of Randal Kaufman, Ph.D., in the Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research. She is studying the endoplasmic reticulum, or ER stress response, which is implicated in diseases ranging from cancer to neurodegeneration. The path that led

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her to Sanford-Burnham was circuitous and fortuitous. As she puts it, “It was my destiny to join the Institute.” In 2007, as a new Ph.D. molecular biologist, Wang was interested in joining the SanfordBurnham laboratory of Stuart Lipton, M.D., Ph.D., a pioneer in ER-stress response research, but their discussions were cut off when massive wildfires swept through San Diego County. She chose instead to join the University of Michigan laboratory of another leader in her field, Randal Kaufman. Three years later, Kaufman called a meeting of his lab and made a surprise announcement. Wang recalled, “He said, ‘I have some important news. I’m going to Sanford-Burnham, and I want you to join me.’” “I was SO thrilled!” she added. “I knew this would open up exciting new avenues for my research.” “Eric is an inspiration to every postdoc here at Sanford-Burnham, and especially to those of

Eric Dudl

us lucky enough to be Eric Dudl Scholarship recipients,” Wang told his family. “I became interested in science as a child because it was a path to learning new ideas. But now I think of science the way Eric did, as a path to discovering cures for diseases like cancer, which was his research focus.”


2013: The Year in Headlines The Huffington Post

January 29, 2013

Skin Stem Cells May Lead To Treatment For Heart Condition

A new stem-cell technology that transforms patient skin cells into heart cells allowed scientists to mimic a rare inherited heart ailment, paving the way for the development of possible new treatments. ARVD/C is a leading cause of sudden death among young athletes. Researchers at Sanford-Burnham led by Huei-Sheng Vincent Chen, M.D., Ph.D., and Johns Hopkins University collected skin samples from adult ARVD/C patients. They added molecules to reverse the development of the cells to their embryotic stage, manipulating them to produce an unlimited supply of heart muscle cells, [then] triggered the cells to behave like sick heart cells similar to those in patients with ARVD/C.

The Economist

The New York Times

April 8, 2013

Protein Linked to Development Problems Down syndrome is caused by the presence of an extra copy of chromosome 21. But it has remained a mystery why that results in impaired development. Now researchers at Sanford-Burnham led by Huaxi Xu, Ph.D., think they have found a clue. The scientists found that mice that lacked a protein known as SNX27 had many of the same learning and memory defects as mice with Down syndrome. Looking at the brains of people with the syndrome, the researcher discovered that they, too, lacked SNX27. Now the researchers are investigating molecules that might increase production of SNX27 in the human brain.

June 22, 2013

Understanding Alzheimer’s Disease

It has been widely presumed that if [amyloid beta] plaques can be removed, the confusion and loss of memory of Alzheimer’s will be relieved as well. It is just a question of inventing a drug that will do this. A paper published this week describes another attempt. Instead of attacking the peptide directly, Stuart Lipton of Sanford-Burnham Medical Research Institute and his colleagues are trying to stop its effects. In doing so, they have characterized in new detail the way the peptide wreaks its damage.

The White House Office of Science and Technology Policy

September 27, 2013

Basic Research, Game Changing Benefits

Last week, in Washington, D.C., distinguished guests gathered at the second annual Golden Goose Awards to honor six federally-funded researchers whose work has positively transformed technology, medicine, and countless lives. With funding from the National Science Foundation, fellow Golden Goose honorees Dr. Thomas Brock and [SanfordBurnham’s] Dr. Hudson Freeze made discoveries that led to the field of biotechnology. Through their study and replication of DNA, they paved the way for a “genomics revolution”— including incredible developments in medical diagnostics, such as genetic tests.

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Nonprofit Organization U.S. Postage 10901 N. Torrey Pines Road, La Jolla, CA 92037

PAID

Sanford-Burnham Medical Research Institute

Grant Hill Visits Sanford-Burnham at Lake Nona

Grant Hill, a seven-time National Basketball Association All-Star, has devoted much of his post-retirement career to philanthropy and health advocacy.  Now a broadcast star with Turner Sports “NBA Inside Stuff,” Hill recently visited SanfordBurnham’s Lake Nona campus, where scientific director Daniel Kelly gave him a close-up look at how Institute research begins the journey to discoveries, new therapeutics, and new hope for patients.


Winter 2013