Spring 2014

IGNITING the FUTURE of MEDICINE Landmark Gift Launches 10-Year Vision Hybrid Scientist Bridges Basic Research and Drug Discovery Key Advances in Therapeutics Target Rare Bone Disease and Obesity VOLUME 4 NUMBER 2 | SPRING 2014

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FOUNDERS

Dr. William H. and Lillian Fishman HONORARY TRUSTEES

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

Gregory T. Lucier

A Letter from the President and Interim CEO 1 Igniting the Future of Medicine: A Landmark Gift Launches Our 10-Year Vision 2 Hybrid Scientist Bridges Basic Research and Drug Discovery 4 Key Advances in Therapeutics Target Rare Bone Disease and Obesity 6 Venture Philanthropy: Q&A with James M. Seneff Jr. 8 Sanford-Burnham Welcomes Carol Cox, Senior Vice President of External Relations 10

CHAIRMAN

Next Generation: Louis Lapierre, Ph.D. 11

Kristiina Vuori, M.D., Ph.D.

Raising Public Awareness: Dani Grady 12

PPRESIDENT AND INTERIM CEO PAULINE AND STANLEY FOSTER PRESIDENTIAL CHAIR PROFESSOR, NCI-DESIGNATED CANCER CENTER

Gary F. Raisl, M.B.A., Ed.D.

Past and Upcoming Events 13 Multiple Sclerosis Society Visits La Jolla Campus Back Cover

EXECUTIVE VICE PRESIDENT CHIEF ADMINISTRATIVE OFFICER CHIEF FINANCIAL OFFICER TREASURER ACTING SECRETARY

Lorenzo M. Berho James C. Blair, Ph.D. David Down 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., M.S., Ph.D. Henry L. Nordhoff Douglas Obenshain Peter Preuss Ze’ev Ronai, Ph.D. Rasesh Thakkar Andrew J. Viterbi, Ph.D. Allen R. Weiss Luder G. Whitlock Jr. Gayle E. Wilson EX-OFFICIO

Todd Golub, M.D. SCIENTIFIC ADVISORY BOARD CHAIRMAN

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SENIOR VICE PRESIDENT, EXTERNAL RELATIONS

Carol Cox

VICE PRESIDENT, EXTERNAL RELATIONS

Edgar M. Gillenwaters VICE PRESIDENTS, PHILANTHROPY

IGNITING the FUTURE of MEDICINE

Paul Baker Philip Graham, M.B.A.

VICE PRESIDENT, COMMUNICATIONS, EDITOR

Deborah Robison ART DIRECTION

Landmark Gift Launches 10-Year Vision Hybrid Scientist Bridges Basic Research and Drug Discovery Key Advances in Therapeutics Target Rare Bone Disease and Obesity www.sanfordburnham.org | PORTAL

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ON THE COVER

James Short PRODUCTION MANAGER

Rhiannon Bruni LEAD WRITER

Kate Callen CONTRIBUTORS

Our ambitious 10-year vision is poised to ignite the future of medicine.

Karolyn Baker Patrick Bartosch Susan Gammon Marissa Igartua

www.sanfordburnham.org

Toll-free: 1-877-454-5702

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 Letter from the President and Interim CEO January 28, 2014, is a day the SanfordBurnham community will long remember.

Greg Lucier, chairman of the Board of Trustees, and I gathered with Institute colleagues in La Jolla’s Fishman Auditorium (and by videoconference in Lake Nona) to make two landmark announcements: The Board had approved our 10-year strategic vision, and we were setting the vision in motion with support from a $275-million philanthropic pledge, the largest in Sanford-Burnham’s history.

Normally, a gift of such magnitude is celebrated with great festivity. But the donor requested anonymity and asked us to keep the focus where it belongs: on implementing the vision and fulfilling its potential to revolutionize 21st-century life-science research. To honor that request, Greg and I marked the occasion with a campus town hall for the scientists, staff, fellows, and graduate students, who are, as Greg said, “the people who will carry this Institute and this vision forward.”

Just before the meeting, I contacted supporters and partner organizations to deliver the news personally, and I announced a new goal of raising $500 million over the next decade. Sanford-Burnham is destined to ignite the future of medicine. We will need substantial private funding to realize that vision, and we will make a compelling case that investment in the Institute will yield extraordinary returns in conquering diseases and improving human health.

My most vivid memories of the January 28 town hall was looking up at the wonderful portrait of our cofounders Bill and Lillian Fishman that graces their namesake venue and seeing many of our postdoctoral

Sanford-Burnham is destined to ignite the future of medicine.

We will need substantial private funding to realize that vision, and we will make a compelling case that investment in the Institute will yield extraordinary returns in conquering diseases and improving human health. fellows in the standing-room-only crowd.

This new course will extend the Fishmans’ legacy of high-impact science to future generations of research pioneers. I know they would feel gratified and proud.

Sincerely,

Kristiina Vuori, M.D., Ph.D. President and Interim CEO Pauline and Stanley Foster Presidential Chair

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Igniting the Future of Medicine:

A Landmark Gift Launches OUR 1o-YEAR Vision It wasn’t just the size of the gift that captured nationwide attention, it was also the scope of the vision the gift was launching.

Sanford-Burnham’s announcement that an anonymous $275-million pledge would help initiate its new 10-year strategic vision made front-page headlines on both coasts. As the news spread, and as leaders of partner organizations praised the donor’s generosity (see sidebar), the Institute was mapping out next steps to implement the vision and sustain this incredible momentum. A first priority was to set the fundraising bar even higher to $500 million to match the cost of the decade-long strategy.

President and interim CEO Kristiina Vuori, M.D., Ph.D., told supporters that the ambitious goal was intended “to increase the size of our institutional endowment and strengthen future support for faculty and scientific programs. Growing our endowment will ensure our long-term financial stability and generate funding to bolster our leading-edge technological capabilities and expand our partnerships, both of which we believe will ultimately contribute to discovering more personalized therapeutic approaches to fighting disease. And it will advance promising research to the point where our scientists can attract significant support through NIH grants and commercial partnerships.”

With expanded private support, Sanford-Burnham can realize the vision’s overarching goal of ascending into the very top echelon of global research powerhouses.

“Over the past 10 years, we have been extremely successful,”

Tributes from Partner Organizations

—Gary Firestein, M.D.

“This magisterial gift […] is a great example of how private philanthropy is playing a crucial role in supporting science, which is especially important at this time when federal funding is in decline.”

—Marsha Chandler Ph.D.

Director, UC San Diego’s Clinical and Translational Research Institute

Executive Vice President, Salk Institute for Biological Studies

“This gift provides resources that allow basic and translational researchers to be more creative and approach problems from a new perspective.”

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“We have to figure out how to take our great science and move it closer to the patient. That will help us attract supporters and ambassadors who want to come along with us on this mission to change the world.”

—Greg Lucier Chairman, Sanford-Burham Board of Trustees Vuori told colleagues at a January 28 town hall. “Over the next 10 years, we will become even more significant and relevant, in large part by increasing the human-disease focus of our research.”

To that end, team-based science will gain visibility. Two exploratory research concepts, Integrated Disease Teams and Diversified Translational Laboratories, will bring together basic research and clinical investigators at health care organizations to rapidly gauge the relevance of laboratory findings for curing or preventing disease.

A Grateful Community Response:

“A community message to the anonymous donor who is giving $275 million to Sanford-Burnham Medical Research Institute based atop Torrey Pines Mesa: Thank you. It marks the most generous act of philanthropy ever in San Diego, a city that is becoming known for such gifts. San Diego is lucky to have so many people willing to give so much back to their community.”

—U-T San Diego Editorial Board “Sanford-Burnham Medical Research Institute has received a $275-million grant from an anonymous donor to further biomedical research and attract more scientists […]. The pledge will be important to Central Florida, where Sanford-Burnham will continue to raise the visibility of Medical City and the region with its biomedical research.”

—Orlando Sentinel

Continued on Page 12

“This gift to Sanford-Burnham further strengthens one of the great biomedicalresearch centers in the world. We’re excited about the positive impact that this will have in years to come.”

—Thad Seymour Ph.D.

“It’s the most exciting time to advance medicine, and terrific that Dr. Vuori and her colleagues have been able to not only craft an ambitious new strategic vision, but also get the funding to execute on it.”

— Eric Topol, M.D.

Senior Vice President, Tavistock Development Company, LLC

Director, Scripps Translational Science Institute

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“At heart, I still feel like a basic researcher… but there’s a point where you want to be part of a team that will work on something with a patient impact.”

— Anne Bang, Ph.D., director, Cell Biology

Hybrid Scientist Bridges Basic Research and Drug Discovery When Anne Bang, Ph.D., arrived at Sanford-Burnham in 2010, she was a new kind of hybrid scientist, a proficient developmental biologist who had directed an industrial group working on embryonic stem-cell therapies for diabetes.

Today, Bang leads Institute efforts to create a “disease in a dish” drug-discovery platform using induced pluripotent stem cells (iPSCs) derived from adult cells. As director of Cell Biology at the Conrad Prebys Center for Chemical Genomics, she is applying new advances in iPSC technology to pinpoint compounds with vast therapeutic potential.

“At heart, I still feel like a basic researcher,” she said. “But there’s a point where you want to be part of a team that will work on something with a patient impact.

Drug discovery does that on a large scale.

With patient-derived stem-cell technology, we can pursue drug discovery in ways that weren’t possible even a few years ago.”

“We also have a new opportunity to be less

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dependent on federal funding,” she added.

“That was Michael Jackson’s goal when he came in to direct the Prebys Center in 2009, and we are well on our way to achieving it.”

Bang’s research portfolio reflects the 10-year strategic vision’s emphasis on aligning basic biomedical research, translational research, and drug discovery and development. And in line with the vision’s focus on expanding partnerships, she has teamed up with collaborators in a range of organizations to capitalize on what she calls “unprecedented” opportunities for treatment breakthroughs.

In November, Bang, Jackson, and their colleagues Siobhan Malany and Layton Smith of the Lake Nona campus received a three-year grant from the U.S. Air Force to find ways of assessing the potential toxicity of large collections of chemicals in a rapid, cost-effective manner.

“The current approach to assessing the health risks of chemical exposure relies extensively on data from animal

models,” said Bang. “But humans may react very differently to chemicals than animals. To help solve this problem, we have developed a technology platform that relies on high-throughput, human-cellbased assays to analyze processes in a cell when it is exposed to a certain chemical.”

In one of her earliest projects at the Institute, Bang joined the Neuroscience Research Initiative, launched in 2012 with a $1 million gift from philanthropists Andrew and Erna Viterbi, to seek out compounds for treating neurological disorders. Using iPSC neurons, her team screened more than 5,000 bioactive compounds to find targets that influence the formation of neurites, projections from neurons that wire up the nervous system.

“The iPSC technology has the potential to identify new molecules that affect neurite differentiation,” she said.

“However, the major goal of the project was to develop a technology platform that can be used to perform drug screens on iPSC-derived neurons from patients with neurological disorders.”

In another effort, Bang has performed a drug screen using cells from patients with a rare form of muscular dystrophy called limb-girdle muscular dystrophy 2i (LGMD2i). “One of the benefits of this project was the opportunity it gave us to

collaborate with patient advocates and disease foundations,” Bang added. “We were very grateful to receive support from the LGMD2i Research Fund, and help from Cure CMD, who connected us with patients who have congenital muscular dystrophy.”

And at the nearby Sanford Consortium for Regenerative Medicine, Bang collaborates with its scientific director, Larry Goldstein, who also leads the Sanford Stem Cell Clinical Center at the UC San Diego School of Medicine. Goldstein is studying iPSC lines with known mutations that trigger hereditary Alzheimer’s disease. His project is funded by the California Institute for Regenerative Medicine, and Bang’s group has been supporting a drug screen for compounds that will improve different physical traits caused by the disease.

The ultimate aim of this and Bang’s other research is, she explained, “to show that iPSC-derived disease models will add a new dimension to drug discovery.”

“In current drug trials, some patients respond and some don’t, but patient populations aren’t well stratified,” Bang added. “With patient-specific stem-cell biology, we can stratify patients into subpopulations and create more relevant human disease models as platforms for a more targeted drug discovery that wasn’t possible in the past.”

Anne Bang, Ph.D., director, Cell Biology, pictured in the Conrad Prebys Center for Chemical Genomics in La Jolla, California.

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Key Advances in Therapeutics Target Rare Bone Disease and Obesity Sanford-Burnham scientists have made extraordinary progress in fulfilling the Institute’s quest, “From Research, the Power to Cure.” The 10-year strategic vision will accelerate that progress by building on the two pillars that distinguish the Institute from other top-tier research organizations: research breakthroughs and therapeutics discoveries. Here are two examples: Rare Bone Disease: José Louis Millán, Ph.D.

For nearly four decades, José Luis Millán, Ph.D., has investigated how irregularities in the protein alkaline phosphatase (ALP) can trigger an array of medical ailments. His study of the microbiology of this single protein has paid off in two therapeutics discoveries: a drug that is successfully treating rickets, and another in clinical development for arteriosclerosis.

Millán began his Institute career in 1977, studying why some lung-cancer patients had an uncommon form of ALP. Over time, his exploration of one disease anomaly led him down different pathways. He eventually zeroed in on diseases related to ALP gene mutations that produce defects in bone mineralization and vascular calcification.

One such disease is hypophosphatasia, an inherited form of rickets that causes defective bone calcification. Millán and his research team at the Sanford Children’s Health

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Research Center, working in a clinical partnership with the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospitals for The top image shows a hypophosphatasia patient Children in before treatment. The bottom reflects the same patient 24 weeks after treatment.

St. Louis, Mo., found that an ALP mutation caused a buildup of a molecule that suppressed skeletal calcification. Their next step was to see if replacing the mutated ALP enzyme could correct the malady.

Through a partnership with Enobia (now Alexion Pharmaceuticals), ALP was engineered by adding parts to the molecule that would deliver it to the bone matrix. Now in the hands of Alexion and named “Asfotase alfa,” the drug is in phase 2 clinical trials. Last year, the FDA designated it a “breakthrough” drug that demonstrates a substantial improvement over existing therapy and will undergo expedited review. To date, more than 50 patients have been successfully treated.

Millán is repeating his success in breakthrough ALP therapies with the recent development of a drug to treat medial vascular calcification, a form of arteriosclerosis where the muscular layer of

arteries become calcified due to increased levels of ALP. The drug was discovered through a sophisticated screening process in Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics and is ready for clinical trials.

Obesity: Steven R. Smith, Ph.D., and Sheila Collins, Ph.D.

Most weight-loss compounds work in the central nervous system to suppress appetite, but such drugs too often produce unwanted side effects. A new therapeutic approach developed at the Florida Hospital – Sanford-Burnham Translational Research Institute for Metabolism and Disease (TRIMD) focuses on a hormone that helps the body burn sugar and fat, and the treatment is progressing from laboratory research to clinical evaluation. Steven R. Smith, M.D., scientific director of the TRI-MD, is overseeing the human research phase.

Orexin is a hormone produced in the brain that was originally discovered and recognized as having a role in sleep disorders and obesity. More recently, Sanford-Burnham researchers examined orexin at a cellular level to assess whether there are direct effects on fat metabolism. They discovered that orexin works by activating calorie-burning brown fat, or “good” fat, that burns sugar and fat instead of storing the energy. In laboratory studies, mice that were orexindeficient weighed more than their normal counterparts, but they actually consumed less food, and their brown fat was not activated.

Sheila Collins, Ph.D., a professor in the Metabolic Disease Program at Sanford-Burnham, has worked on fat cells and energy expenditure for many years, and she sums up why brown fat may hold a clue for treating obesity. “Brown fat is an ancient mechanism that allows mammals to generate heat in order to keep warm during times of cold exposure,” she said. “Special proteins allow brown cells to burn calories where the net result is heat instead of conserving caloric energy, as white fat cells do.” The ability of brown fat cells to consume substantial quantities of glucose and fatty acids helps the body dissipate caloric energy and reduce blood levels of sugars and fats linked to diabetes and heart disease.

“Advancing orexin to the TRI-MD is a crucial step in translating fundamental research at Sanford-Burnham to the clinical phase,” said Smith, who also serves as codirector of translational research for the Sanford-Burnham Diabetes and Obesity Research Center. “At TRI-MD, we conduct proof-of-concept experiments to validate new drug targets and test them for safety and efficacy. Orexin is progressing to human clinical studies at the Orlando-based institute.”

For TRI-MD and Sanford-Burnham scientists, the next step is determining if and how orexin changes metabolism in humans.

The collaborators hope that their “bench to bedside” research will also contribute to the development of more-personalized treatments for a variety of metabolic diseases. www.sanfordburnham.org | PORTAL

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Venture Philanthropy:

Q&A James M. Seneff Jr. Photo credit: Doug Scaletta

with

James M. Seneff Jr., executive chairman of CNL Financial Group, is a lifelong student of history. The pre-eminent financier and philanthropist sees the 21st century as a “conceptual age” in which human endeavor moves its focus from gathering facts to generating ideas. He believes that Sanford-Burnham, with its 10-year strategic vision, has gotten a head start on the new era—and that’s why he supports the Institute and has great confidence in its future.

What is your personal approach to philanthropy?

My primary goal as a philanthropist is to figure out ways to create human flourishing and thriving cities. One way is through better health and the other is science.

We’re looking to support institutions with strong leadership and a strong sense of vision, and Sanford-Burnham clearly qualifies. Sanford-Burnham is doing things that are going to change the way people live in the future. That’s

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pretty spectacular. And there’s a personal motivation for supporting the Institute; my dad had Parkinson’s disease, and I know your scientists are working on that.

You’ve spoken about the importance of “getting to ‘next’” in this rapidly changing global society.

How will the Institute’s 10-year strategic vision achieve that?

The 10-year vision exemplifies principles of CNL’s da Vinci Center, our laboratory

for envisioning the future, which are “Scan. Focus. Act.” Scanning comes first because it’s how you find new discoveries and connect things that don’t necessarily relate. Sanford-Burnham is scanning the underlying pieces of information about medical science in a way that will lead the field to focus and act in new directions. Your vision also puts you on the cutting edge of technological innovation, and it emphasizes the need to recruit the best people, which is how you create great organizations.

Do you share the view that philanthropy is really about investing in the common good?

I agree. People who approach philanthropy as an investment tend to be much more proactive about the organizations they support. My overall philosophy is that, for every 10 ideas, there is one idea that will be a breakthrough idea and make a real difference. That’s the way we run our business, and that’s the way we think about philanthropy. Supporting SanfordBurnham has been a great investment in Central Florida—you are the “one out of 10” idea, and you’ve been the right

institution for our community. Our former Governor Jeb Bush has said that bringing Sanford-Burnham to Florida is one of the things he is most proud of.

How can an organization like SanfordBurnham stay true to the strengths that have given it a competitive advantage yet evolve for the future?

There are two types of mindsets: fixed and growth. The fixed mindset thinks, “I know a lot of things, and I need to prove myself.” This type of mindset is not open to new ideas. The growth mindset thinks, “I’m curious about everything, and I’m always trying to improve myself.” Sanford-Burnham is fostering the growth mindset, and it does that with genuine passion for its mission.

Researchers, like entrepreneurs, have tremendous passion for their pursuits, and that gives them energy and creativity.

They are constantly thinking, “What can I do differently?” I know that the scientists and the leaders at SanfordBurnham are very passionate about what they’re doing, and that’s why they’re making a huge difference in society.

“Sanford-Burnham

is doing things that are going to change the way people live in the future.

That’s pretty spectacular.”

— James M. Seneff Jr., executive chairman, CNL Financial Group

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INSTITUTE NEWS

Sanford-Burnham Welcomes Carol Cox as Senior Vice President of External Relations Carol Cox joined Sanford-Burnham March 4 as senior vice president of External Relations to oversee the Institute’s communications, philanthropy, marketing, and government relations functions. She came to SanfordBurnham from Life Technologies, where she served as senior vice president of External Affairs and Corporate Communications.

“Carol is an outstanding addition to our executive team at a most opportune time,” said Kristiina Vuori, M.D., Ph.D., president and interim CEO. “As Sanford-Burnham builds up health care, pharmaceutical industry, and other private-sector partnerships to advance our translational initiatives, Carol’s business experience will foster that growth, and her marketing savvy will help convey the unique value of the Institute’s achievements.”

Carol will take a leadership role in communicating and leveraging the Institute’s 10-year strategic vision to stakeholders,

Carol Cox, senior vice president of External Relations, at last year’s Pedal the Cause, San Diego, benefitting NCI-designated Cancer Centers.

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as well as implementing a $500-million fundraising campaign in support of the vision. She will draw on expertise and business skills developed over more than 20 years as a communications and investor relations leader in life-science industries.

“Carol is a dynamic and respected communications professional, and I am pleased that she will lead Sanford-Burnham’s External Relations team,” said Greg Lucier, chairman of the Board of Trustees. “I’ve worked with her for a number of years and value her strategic thinking and expertise in elevating global brand awareness. She will be critical to elevating the Institute’s recognition and attaining our ambitious fundraising goal.”

Carol holds a bachelor of arts degree in International Relations from the University of Virginia. An avid cyclist, she participated in last year’s inaugural Pedal the Cause, San Diego, in honor of her mother, who was an ovarian cancer survivor, to raise money for research at Sanford-Burnham’s NCI-designated Cancer Center and other area cancer centers.

Next Generation

Postdoc Louis Lapierre Shares Why He Joined Sanford-Burnham Louis Lapierre, Ph.D., is coming off a big year. Last June, the postdoctoral fellow received a prestigious Pathway to Independence Award from the National Institute on Aging designed to fast-track the careers of research prodigies. Four months later, he was named one of five 2013 Fishman Fund Award winners.

But the 34-year-old biochemist has little time to savor his laurels. He is intent on developing a breakthrough Alzheimer’s treatment that will catch the world’s attention, and he believes that SanfordBurnham is the place to make that happen.

“This Institute gives you every chance to succeed, but it also sets high expectations,” said Lapierre.

“Anybody can do good science. To do great science, you have to be able to gauge projects that have the potential for sizable impact.

That’s what differentiates

Sanford-Burnham, and that’s why I’m here.

“I’m not just interested in research projects,” he added. “I’m interested in meaningful research stories that will appeal to reviewers, as well as donors and collaborators.

By broadening the network of people who follow and support our work, we can advance our science that much faster.”

The hero of Lapierre’s research story is C. elegans, the tiny worm that revolutionized the field of research on aging. Upon arrival at Sanford-Burnham in 2008, Lapierre joined the laboratory of Malene Hansen, Ph.D., in the

C. Elegans, a type of nematode, frequently used in biomedical research.

Image courtesy of Louis Lapierre, Ph.D.

Development, Aging, and Regeneration Program.

His initial work showed that improving the recycling of fat via autophagy, the main mechanism for cellular material turnover and regeneration, has benefits that could extend to the prevention of Alzheimer’s disease.

Extending his research into mouse models, Lapierre has identified a promising therapeutic target that stimulates autophagy as a means of preventing the toxic aggregation linked to Alzheimer’s. His ultimate objective is discovering drug candidates for clinical trials, and he is racing to reach it.

“The U.S. government’s National Plan to Address Alzheimer’s Disease has set a goal of developing effective treatments by 2025,” said Lapierre. “If we can find compounds that mediate substantial cognitive benefits in mouse models of the disease in the next three years, we can reach that goal—and we can knock it out of the park.”

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Raising Public Awareness: Dani Grady Cancer Survivor and Research Activist Dani Grady Joins Sanford-Burnham’s Cancer Center Community Advisory Board When asked what sets Sanford-Burnham’s NCI-designated Cancer Center apart from peer organizations, Dani Grady— ardent cancer research campaigner, community educator, patient advocate, and cancer survivor—replied candidly, “The prestigious NCI-designation certainly caught my attention, but it is the intense focus of the scientists to understand every aspect of cancer that sets them apart. That level of dedication is what makes the difference.”

Grady, who was diagnosed with advanced breast cancer at the age of 29, has been in remission for the last 23 years. She, along with other cancer survivors and research advocates, are members of Sanford-Burnham’s

Cancer Center Community Advisory Board—a group that meets periodically with the Center’s scientists to bridge the gap between the research community and cancer patients.

“From a cancer survivor’s perspective, time is of the essence,” Grady shares.

“Major breakthroughs often come from collaboration, so we encourage the cancer research community to speak openly with each other about their discoveries, as well as consider the patient’s needs in all they do.” In addition to encouraging collaboration among peer research organizations, the Advisory Board also provides fundraising and public education support the Cancer Center.

It’s a labor of love for

Grady, who understands the realities of life for cancer survivors intimately. “Many have not been so fortunate, and for me, a positive outlook means taking positive action. For all of us, there exists a real possibility that it will come back,” she explains. “Chemo resistance is a significant obstacle to survival. Sanford-Burnham’s scientists are facing it head on through pioneering drugdiscovery strategies and world-class research. Their dedication and tireless efforts fill me with hope that the day is coming when cancer can be controlled and ultimately eradicated.”

Landmark Gift Launches Our Vision Continued from Page 3 The vision also incorporates a new business model for the Institute that will seek to: • Accelerate discovery research into the underlying mechanisms of disease, • Develop novel therapeutic approaches past the conventional point where nonprofit institutes hand off their scientific discoveries, and • Create a pipeline of drug candidates in the Conrad Prebys Center for Chemical Genomics, the most advanced smallmolecule drug-discovery platform in the nonprofit research world.

Greg Lucier, chairman of the

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Board of Trustees, told the town hall gathering that “a series of milestones” will help measure the Institute’s progress in carrying out the vision.

“It is important that we don’t lose sight of what we have achieved and of where we want to go,” Lucier said. “And we need to embrace the future, because what got us here won’t get us there. We have to figure out how to take our great science and move it closer to the patient. That will help us attract supporters and ambassadors who want to come along with us on this mission to change the world,” he added.

Past Events Metabolic Origins of Disease Symposium Sanford-Burnham at Lake Nona hosted the 2014 Metabolic Origins of Disease Symposium March 3–5, 2014. The symposium featured 24 international scientists presenting their most-recent research into metabolic causes of disease.

More than 250 scientists attended to hear about the latest research and to network. The sold-out symposium was a resounding success and reinforces SanfordBurnham’s position as a world-renowned Institute for collaborative research.

Rare Disease Day Symposium Sanford-Burnham hosted the fifth Annual Rare Disease Day Symposium February 28–March 1, 2014. The two-day symposium, chaired by Hudson Freeze, Ph.D., professor and director of the Human Genetics Program, highlighted a disorder caused by a rare mutation in the NGLY1 gene.

The first day featured presentations by the world’s leading experts, while the second provided the opportunity for affected families to meet with clinicians and scientists in a “doctor is in” session—offering them the chance to meet with specialists in one location.

Upcoming Events Bring It! Playoffs

Sanford-Burnham is hosting the sixth annual Bring It! event at the San Diego Hall of Champions in San Diego, California, on Thursday, May 1, 2014. Join event co-chairs Linde Hotchkiss and Kristoffer Kelly for the opportunity to meet, greet, and compete with San Diego’s business and community leaders in an exciting sports-themed competition supporting medical research.

For information, please contact Karolyn Baker at (858) 795-5239 or kbaker@sanfordburnham.org.

Annual Gala Sanford-Burnham’s annual gala hosts guests for a memorable evening filled with delectable food, exquisite drinks, and top-notch entertainment in support of pioneering medical research. Each year’s theme transports guests to a different time and place, treating them to an unforgettable evening as they help to improve and transform lives.

This year’s gala will be on Saturday, November 1, 2014. Mark your calendars now and plan to attend what is sure to be an extraordinary event. www.sanfordburnham.org | PORTAL

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Multiple Sclerosis Society Visits La Jolla Campus On March 4, more than 40 multiple sclerosis (MS) research advocates came to Sanford-Burnham to see firsthand how our labs are making significant Sanford-Burnham researcher Gülsen Çolakoğlu, Ph.D., (left) and MS Walk fundraisers Nick and Ruth Tsoulos pictured in the Cell Imaging Core. advancements in the fight against this immune-mediated disease.

The advocates were in San Diego in support of the National Multiple Sclerosis Society’s MS Awareness Week, March 3 – 9, 2014, and welcomed the opportunity to visit our La Jolla campus. Their visit began with a presentation by Sanford-Burnham scientist Barbara Ranscht, Ph.D., professor in the Development, Aging, and Regeneration Program. Ranscht and her lab are working on ways to repair the myelin that is damaged in MS. Immediately following the presentation, guests were invited to visit with Ranscht in her laboratory for an informal question-and-answer session, and see the scientific techniques and technologies employed by her team of researchers.

Visitors also toured the Cell Imaging and Histopathology facility, and the Conrad Prebys Center for Chemical Genomics. “It was an exceptional visit—and we were very excited to share our discoveries benefiting MS research with the Multiple Sclerosis Society’s top supporters,” said Ranscht.

Sanford-Burnham researcher Gülsen Çolakoğlu, Ph.D., (left) and MS Walk fundraisers Nick and Ruth Tsoulos pictured in the Cell Imaging Facility

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