Winter 2014

Our new CEO Perry Nisen, M.D., Ph.D., on the evolution of modern medical research

VO L U M E 5 N U M B E R 1

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WINTER 2014

FOUNDERS

Dr. William H. and Lillian Fishman

A Message from the Chairman of the Board and the President 1 From Molecules to Medicine: Our New CEO Perry Nisen M.D., Ph.D. 2

HONORARY TRUSTEES

Influential Science: Randal Kaufman, Ph.D. 4

Roberta and Malin Burnham Joseph C. Lewis Conrad T. Prebys T. Denny Sanford

Developing A Better Test for Prostate Cancer 5 Carl Ware, Ph.D., on the Benefits of Partnering 6 Institute News: 2014 Pedal the Cause Success 7

TRUSTEES AND OFFICERS

Institute News: Titan Krios Microscope Inauguration Symposium and Reception 8

Gregory T. Lucier

2014 “Top Hat” Gala Highlights 9

CHAIRMAN

Staying Competitive: Q&A with Trustee David Down 10

Perry Nisen, M.D., Ph.D.

Past Events Highlights 12

CHIEF EXECUTIVE OFFICER DONALD BREN CHIEF EXECUTIVE CHAIR

Partners in Science: The Honorable Mel Martinez Back Cover

Kristiina Vuori, M.D., Ph.D. PRESIDENT PAULINE AND STANLEY FOSTER PRESIDENTIAL CHAIR

VICE PRESIDENTS, PHILANTHROPY

Paul Baker Philip Graham, M.B.A.

PROFESSOR, NCI-DESIGNATED CANCER CENTER

Gary F. Raisl, M.B.A., Ed.D. EXECUTIVE VICE PRESIDENT

VICE PRESIDENT, COMMUNICATIONS EDITOR

CHIEF ADMINISTRATIVE OFFICER

Deborah Robison

CHIEF FINANCIAL OFFICER

PRODUCTION MANAGER & CONTRIBUTOR

TREASURER

Rhiannon Bruni

Lorenzo M. Berho James C. Blair, Ph.D.

ART DIRECTION

James Short

David W. Down Daniel J. Epstein

Our new CEO Perry Nisen, M.D., Ph.D., on the evolution of modern medical research

M. Wainwright Fishburn Jr. Pauline M. Foster Patrick J. Geraghty William Gerhart Alan A. Gleicher Jeanne L. Herberger, Ph.D. Donald L. Jernigan, Ph.D. J. Bernard Machen, D.D.S., M.S., Ph.D. Papa Doug Manchester Henry L. Nordhoff Douglas H. Obenshain Edward R. Schulak Rasesh Thakkar Luder G. Whitlock Jr.

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WINTER 2014

ON THE COVER Perry Nisen, M.D., Ph.D., joined as our Chief Executive Officer, and holder of the Donald Bren Chief Executive Chair, in August of 2014.

WRITER

Kate Callen CONTRIBUTORS

Karolyn Baker Patrick Bartosch Susan Gammon, Ph.D., M.B.A. Marissa Igartua Diane Wilson DESIGN

Creative Fusion

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

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From L to R: Institute President Kristiina Vuori, M.D., Ph.D., CEO Perry Nisen, M.D., Ph.D., and Chairman of the Board Gregory Lucier at the August 2014 press conference announcing Dr. Nisen’s appointment.

A Message from the Chairman of the Board and the President By any measure, Sanford-Burnham Medical Research Institute has had a very big year.

We began 2014 with the announcement of two milestones: first, the launch of our 10-year strategic vision to translate basic research discoveries into novel therapeutics, and then, a $275-million pledge, the largest philanthropic gift in our 38-year history, to help implement the vision.

As the news spread, an Institute Trustee committee intensified its global search for a chief executive.

The Trustees set high selection criteria. To fully realize the vision, the new CEO would need expertise in laboratory science, health care, and drug development. Given our aspirations, we wanted someone with a dynamic leadership style, a passion for innovation, and a commitment to accelerating research breakthroughs into new medicines.

That person is Perry Nisen, M.D., Ph.D. He arrived on September 15 for his first day as our new CEO, and he has hit the ground running.

As the bio-industry publication FierceBiotech reported, “Nisen’s plan is to encourage unfettered, collaborative research that is already suited for professional drug discovery, crafting an institution able to think more creatively than big pharma while focusing more on value creation than nonprofits tend to do.”

But Nisen’s most profound impact may come from his desire to connect patients with scientists in a translational research enterprise. As you will read in this issue, he wants Sanford-Burnham to have a tangible impact on people’s lives, and he is moving ahead on all fronts to make that happen.

We begin 2015 with a new leader, new momentum, and new discoveries within reach. All of us can take pride in Sanford-Burnham’s ascension to date. Together, we will drive that trajectory ever higher in the years ahead.

Sincerely,

Gregory T. Lucier

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

Chairman, Sanford-Burnham Board of Trustees

President Pauline and Stanley Foster Presidential Chair Professor, NCI-Designated Cancer Center

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from Molecules to Medicine Our new CEO Perry Nisen, M.D., Ph.D.

“Sanford-Burnham is a hybrid place where basic research and therapeutics discovery intersect and things happen in a unique and different way. The pieces are here to do something really grand.”

— Perry Nisen, M.D., Ph.D. 2

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Perry Nisen, M.D., Ph.D., has an inner compass that points the way to finding cures for diseases.

Assay plates, like the one above, are used in the Conrad Prebys Center for Chemical Genomics to screen for small molecules that could be the building blocks for new medicines.

It has guided him through an extraordinary 35-year career to pivotal junctures in basic research, clinical care, and drug discovery.

As Sanford-Burnham’s fourth chief executive officer in nearly four decades, Nisen can now chart a bold new course in 21st-century bio­ medical science. With his vast experience in translational medicine and his broad knowledge of therapeutics development, he is uniquely qualified to implement our 10-year strategic vision, and he is eager to propel it forward.

Shepherding Scientific Discoveries

At an August town hall where his appointment was announced, Nisen told his new colleagues, “I’ve lived my professional career at the inter­­face of basic science and clinical medicine, and I’ve always had an absolute passion for having an impact on patients.

“I’ve had the privilege of helping shepherd scientific discoveries through the clinic and to patients,” he said. “It is an indescribable experience. It is also incredibly hard.”

“What I’m all about is exactly what the Institute’s tag line says: ‘From Research, the Power to Cure,’” he continued. “Sanford-Burnham is a hybrid place where basic research and therapeutics discovery intersect and things happen in a unique and different way. The pieces are here to do something really grand.”

Nisen, who also holds the Donald Bren Chief Executive Chair, came to Sanford-Burnham from the global pharmaceutical giant GlaxoSmithKline (GSK), where he was senior vice president for Science and Innovation. Before he rose to prom­inence in drug develop­ment, he was a distinguished research oncologist who explored the molecular structures of pediatric brain tumors to identify potential gene therapy pathways.

Nisen met that challenge with ever greater success as a clinical researcher until a fateful encounter altered his life. He was the Lowe Foundation Professor of Neuro-Oncology at the University of Texas Southwestern Medical Center when he met with the family of a young patient. The girl’s brain tumor was malignant, and Nisen had to tell her parents that no available treatments could save her.

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ASK THE CEO

Do you have questions for Dr. Nisen?

We’d love to hear from you! Email your name and any questions to asktheceo@sanfordburnham.org. We’ll include the most popular questions, and Dr. Nisen’s answers, in the 2015 spring issue of Portal.

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Influential Science:

Randal Kaufman, Ph.D. Productive scientists make discoveries that yield new knowledge.

Influential scientists make discoveries that enable legions of other researchers to generate new knowledge.

The 2014 list of “The World’s Most Influential Scientific Minds” from the multinational media giant Thomson Reuters includes SanfordBurnham’s own Randal Kaufman, Ph.D., director of the Degenerative Disease Program.

Kaufman was recognized as a global leader in the highly competitive category of “Biology and Biochemistry.”

Thomson Reuters compiled the list using citation data from scientific papers to determine which investigators are most frequently acknowledged by other investigators. “The identification of these individuals is rooted in the collective, objective opinions of the scientific community,” the company announcement said. “They are the people who are on the cutting edge of their fields. They are performing and publishing work that their peers recognize as vital to the advancement of their science.”

When Kaufman joined Sanford-Burnham in 2011, he already was a pioneer in elucidating the mechanisms by which cells identify the presence of misfolded proteins, and he had helped delineate the signaling pathways that cells employ to prevent further protein

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misfolding. His fundamental discovery was that protein misfolding is key in the etiology of metabolic, inflammatory, and neurodegenerative diseases, including diabetes, metabolic syndrome, and inflammatory bowel disease.

Kaufman also has applied his work on protein misfolding to explore how the process plays a profound role in cancer development. In the August 22, 2014 journal, Nature Reviews Cancer, he and his Institute colleague Miao Wang, Ph.D., demonstrated that as tumors grow, their nutrient requirements increase and eventually exceed what’s available. Lack of nutrients compromises protein folding and triggers signals that have both tumor-supporting and tumor-suppressive roles. Deciphering these signals enables strategies to target the signaling molecules for cancer treatment.

“Basic research in the field of protein folding is central not only to understanding some of the most debilitating diseases,” said Kaufman, “but it is also fundamental to understanding why we age. Research in this area, conducted with an open mind for possible applications, is contributing to information that has the potential to help us live longer and healthier lives.”

Developing a Better Test for Prostate Cancer Prostate cancer is the most common cancer in American men.

The sheer number of diagnoses is staggering: More than 230,000 Americans will have received a prostate cancer diagnosis by the end of 2014, according to the American Cancer Society. But the diagnosis is often a com­­­plicated, invasive, and unreliable journey for affected patients.

Since its introduction in the 1980s, the prostate-specific antigen (PSA) test has helped diagnose prostate cancer in millions of men worldwide. But the test cannot detect cancer with absolute certainty. If PSA levels are too high, doctors usually recommend invasive, and often unnecessary, biopsies. A team of scientists led

by Ranjan Perera, Ph.D., professor and scientific director of Analytical Genomics and Bioinformatics at our Lake Nona campus, has recently published a study that could revolutionize the diagnosis of the disease.

Perera and his team have found that certain segments of RNA (which are molecules that encode DNA information) called longnoncoding RNAs play a role in the development of prostate cancer and could potentially be used as bio­­­­markers for the disease. “We have identified a set of long noncoding RNAs that appears to have an important role in prostate cancer diagnostics,” said Perera. “The findings broaden the opportunity to use long noncoding RNAs as bio­markers to detect prostate cancer.”

The research could be the first step toward developing a test that would identify prostate cancer with certainty using blood and urine samples. The presence of these long noncoding RNAs in the blood or urine could not only help diagnose a cancerous tumor, but the aggressiveness of the tumor as well. A treatment regimen could then be developed accordingly. Unnecessary biopsies could be avoided, saving health-care systems money and bringing peace of mind to patients and their families, as well as more-reliable diagnoses at an earlier stage.

Did you know?

The PSA test is based on a technology that was invented by Eva Engvall, Ph.D., adjunct professor at our La Jolla campus.

Perera’s study of these markers is now being validated in a larger number of patient samples to pave the way for their incorporation into clinical diagnostics. Make sure to follow the progress of this potentially life-saving research in future issues of Portal and by reading our Sanford-Burnham blog Beaker at beaker.sanfordburnham.org.

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Carl Ware, Ph.D., on the

Benefits of Partnering

Carl Ware, Ph.D., professor and director of the Infectious and Inflammatory Disease Center, conducts research that spans from the herpes virus and HIV to cancer and the development of new drugs to treat autoimmune disorders, such as lupus and psoriasis.

With such a broad area of inquiry, Ware’s efforts have garnered significant interest from biotech and pharmaceutical companies that are keen to partner in the development of new drugs. These partnerships are designed to support research in areas of mutual interest.

We sat down with him recently to learn more about the benefits of these partnerships.

The Journal of Immunology recently nominated you as a “Pillar of Immunology”—meaning that your published research is seminal within the field. Tell us about the focus of your work.

My lab investigates cytokines—proteins that serve as the communication network for the immune system. Think of them as the AT&T for immunity. Our discovery of the cytokine that controls the formation and function of immune organs, like lymph nodes, is the basis of my nomination. It was a much unexpected discovery, which grew from my graduate research on cytokines that kill cancer cells.

What motivates you every day?

At the beginning of my career, the motivation to be a scientist came from the thrill of discovery—but by mid-career it was to support my family. Nowadays, it’s the satisfaction of giving the opportunity to young scientists to discover new knowledge.

What are the top priorities in your research?

Maintaining the funding to support the scientists in our Center is always top priority. On the science side, about half of our efforts focus on designing and testing drugs directed at cytokines to rewire the immune system. We design drugs that suppress inflammation in patients with autoimmune disease, or enhance immunity to kill cancer cells. Interestingly, understanding how viruses trick the immune system—the other half of our research program—contributes to our success in designing new drugs.

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Infection with Human Cytomegalovirus (CMV) In people with a compromised immune system, CMV can result in devastating disease in many organs, including the eye.

Despite the immune system’s best efforts, CMV persists for the lifetime of the infected person. One of the genes in CMV restricts inflammation caused by the immune response. Ware’s research has revealed a new way to dampen inflammation for potential use in treating patients with autoimmune diseases, such as Sjogren’s disease (dry-eye syndrome).

Left: Ocular view of CMV-infected retina; electron micrograph of human CMV.

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INSTITUTE NEWS

Team Sanford-Burnham (from L to R) Brian James, Steve Williams, Ryon Graf, Kimberly McKewon, Wainwright Fishburn, and Philip Graham.

2014 Pedal the Cause Success Enthusiastic cheers swept Team SanfordBurnham, along with more than 750 riders, across the finish line on September 21 as the second annual Pedal the Cause San Diego bike event came to a successful end.

Pedal the Cause San Diego, the only multiday cycling fundraiser to support cancer research in San Diego, provides critical funding to the community’s National Cancer Institutedesignated cancer centers.

As home to three such centers, San Diego has long been a powerhouse for cancer research, prevention, and treatment. Sanford-Burnham, UC San Diego Moores Cancer Center, and the Salk Institute for Biological Studies form the novel collaboration known as San Diego’s National Cancer Institute-Designated Cancer Centers Council, or C3, and work together to leverage their distinct resources to accelerate innovative approaches for the treatment and prevention of cancer.

The second annual ride featured 10-, 25-, and 50-mile courses, as well as a two-day cycling route from La Jolla to Temecula, Calif.

Two-day riders gathered at UC San Diego early on the morning of September 20 and embarked on an epic cycling adventure through the late-summer terrain on their way to the Temecula Rider Camp, located at the Tucalota Creek Ranch, for the evening.

Once at the camp, informally known as “Tent City,” road-weary riders relaxed while enjoying barbeque, live entertainment, sport massages, and the camaraderie of fellow riders.

The next morning, they cycled back to La Jolla and joined the 10-, 25-, and 50-mile riders for finish-line celebrations.

The event was an incredible success—raising $1 million for critical cancer research in San Diego. Sanford-Burnham was delighted to once again be chosen as a beneficiary for Pedal the Cause San Diego. Thank you to everyone— staff, volunteers, generous benefactors, and riders—who helped to make this year’s event a phenomenal success.

We look forward to next year’s ride!

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INSTITUTE NEWS

TITAN KRIOS MICROSCOPE Inauguration Symposium and Reception Dorit Hanein, Ph.D., professor in the Bioinformatics and Structural Biology Program, hosted the Titan Microscope Inauguration Symposium and Reception on August 21 at our La Jolla campus.

Dorit Hanein, Ph.D., (L) professor in the Bioinformatics and Structural Biology Program, and Nina Fishman (R), daughter of the Institute’s founders, in front of the new Titan Krios transmission microscope.

The event celebrated the arrival of the new Titan Krios Transmission Electron Microscope and honored our founders, Dr. William and Lillian Fishman, who acquired the Institute’s first micro­scope more than 35 years ago, thereby establishing a legacy of providing Sanford-Burnham’s scientists with cutting-edge technology.

will help the Institute’s scientists—and their collaborators—tackle unanswered questions in structural biology by creating 3-D images of cells, proteins, pathogens, and even single particles. By visualizing these tiny details, we move closer to understanding the molecular origins of disease.

Ze’ev Ronai, Ph.D., scientific director of our La Jolla campus, addresses the crowd at the Titan Krios microscope inauguration.

The symposium attracted distinguished presenters from peer research institutes, including UC San Diego, The Scripps Research Institute, Caltech, Stanford, and the National University of Singapore.

Attendees were given an exclusive tour of the electron microscopes housed at our La Jolla campus.

Enthusiastic guests gather in Chairmen’s Hall for the Titan Krios inauguration.

The new Titan Krios embodies that legacy. The state-of-the-art electron microscope will help our scientists visualize cells, viruses, and bacteria at the atomic level. With fewer than 45 instruments available worldwide, and only a handful located in the U.S., it is the most advanced and powerful electron microscope on the planet. The instru­ment

Immediately following the symposium, guests and donors gathered in Chairmen’s Hall for a cocktail reception and tour of the new Titan Krios suite. Nina Fishman, Dr. William and Lillian Fishman’s daughter, joined the reception and praised the Institute’s continued commit­ ment to her parent’s vision for the Institute.

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SANFORD-BURNHAM’S 2014 ANNUAL GALA HIGHLIGHTS Premier entertainment, great food, and good friends hit high notes at the Sanford-Burnham “Top Hat” gala, held Saturday, November 1, 2014, at the Estancia La Jolla Hotel & Spa. The evening raised a grand total of $2.1 million for medical research.

Honorary gala chairs Lisa & Steven Cassidy and Karen & Stuart Tanz orchestrated a top-notch evening that showcased the exuberance and charm of Irving Berlin’s classic musical, “Top Hat.” Thank you to our generous benefactors and supporters for continuing to show your dedication to our shared quest, From Research, the Power to Cure.

(L-R) Bill Gerhart, Melissa Seipel, and David Malcom

(L-R) Steven & Lisa Cassidy and Karen & Stuart Tanz

(L-R) Malin Burnham, Jeanne & Gary Herberger, Diane & Tom Might, Matt & Cristina Might

(L-R) Gary & Jeanne Herberger, Malin & Roberta Burnham, Denny Sanford & Elizabeth Dewberry

(L-R) Doug Obenshain, Clare McGowen & Michael Jackson, Jeanne & Gary Herberger

(L-R) Steven & Lisa Cassidy, Perry Nisen, M.D., Ph.D., Kristiina Vuori, M.D., Ph.D., Stuart & Karen Tanz

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Staying Competitive: Q & A with Trustee David Down “My hope is that 10 years from now, we are celebrating important drug discoveries that came directly from research breakthroughs at the Institute and are transforming people’s lives.”

— David Down

Shortly after joining SanfordBurnham’s Board of Trustees at its fall 2014 meeting, David Down retired from the global accounting giant KPMG LLP after 20 years as managing partner and launched a new chapter in his life and career.

Down has served with distinction on numerous nonprofit boards, but he is especially enthusiastic about his new role as Institute Trustee. As he explains in this interview, he is committed to helping the Institute remain competitive in a rapidly changing biomedical research arena.

What drew you to Sanford-Burnham?

I’ve worked with life-science companies in San Diego for many years, and I became friends with Duane Roth through my involvement with CONNECT. When Duane asked me to be of service to SanfordBurnham, I did my research, and I saw that this Institute is the Cadillac of medical research institutions. The talent here is just overwhelming. I was honored to be invited on the Board. I’m truly excited about the Institute’s future, particularly with regard to its research on childhood diseases, cancer, and obesity. Those are three key areas in health care today, and the ability to have an impact in any one of them would be incredible. I plan to spend a lot of time looking for ways that I can contribute to Sanford-Burnham’s success.

What are your most important duties as a Trustee?

I would say stewardship to all constituents who rely on Sanford-Burnham, including clinicians and patients, and also fiduciary responsibility to all who provide funding.

We need to make sure our scientists have adequate resources to do their work, and we need to be connected to the health-care community. And above all else, Trustees must strive to protect the Institute’s brand and reputation.

What were your early impressions of Sanford-Burnham?

I visited the La Jolla campus several times before I became involved, and I visited the Lake Nona campus for the first time last spring. At Lake Nona, we are actively recruiting scientists to our state-of-the-art campus. In La Jolla, scientists are drawn to the outstanding technology platforms and the legacy of great science. I’d like to see enhancements made to the La Jolla campus so we can continue to attract world-class scientists by offering them world-class research facilities. We’re competing for talent, and we have to keep pace with the quality that our competitor institutions have.

You have been a trusted advisor to non-profits for 35-plus years.

How can non-profits adopt best business practices?

They have to focus on accountability and transparency. They have to understand who their constituents are. And they have to foster a healthy work environment where employees feel that they can voice their opinions and where everyone works together as a big family. Sanford-Burnham has done all of that, which has really impressed me. I think it’s a tribute to the leadership team and to the extraordinary quality of the Board.

How will Sanford-Burnham’s 10-year strategic vision drive innovation in the life sciences?

The strategic vision has given the Institute a chance to refocus on assets with the highest potential—basic research and drug discovery expertise, collaborations and partnerships— because our ability to focus will be critical over the next decade. If 1,000 scientists study 1,000 different things, the likelihood of success is small.

Over the next 10 years, how will the Institute deliver on its quest, “From Research, the Power to Cure”?

My hope is that 10 years from now, we are celebrating important drug discoveries that came directly from research breakthroughs at the Institute and are transforming people’s lives. I believe that will happen because our new CEO, Perry Nisen, knows the road map to generating new medicines and he knows how to get there. When you bring in a leader with his talent and know-how, you raise everybody’s game in the organization.

What role will philanthropy play?

It will be the key to the success of the vision.

Denny Sanford has said it best: “There are lots of people who are willing to give and who can give, but you have to ask them correctly. You need to lay out a clear path for each individual donor. While it’s awesome to receive very large gifts, there are thousands of folks out there who can make substantial contributions at a level that will propel our research forward. In the long term, SanfordBurnham will pursue licensing of Institute innovations that will give us financial sustainability. But right now, we need more philanthropic giving to propel us towards our 10-year endpoint: making tangible contributions to developing therapeutics that improve human health.”

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Past Events AUGUST 27

OCTOBER 2

San Diego Padres VIP Event

Annual Fishman Fund Award Three gifted postdoctoral researchers were presented the prestigious Fishman Fund Award at the annual ceremony on Oct. 2. Generous benefactors and past award winners joined the celebration to congratulate the young scientists and encourage them to continue their research careers.

Congratulations to this year’s awardees: Karthik Bodhinathan, Ph.D., Hongbo Pang, Ph.D., and Emily Smith, Ph.D.

(From L to R) Dan Epstein, Bill Gerhart, Stuart Tanz, and Alan and Zachary Gleicher enjoying the game.

On Aug. 27, more than 50 of our most generous benefactors and supporters gathered to enjoy a day in the Owner’s Box at Petco Park. Guests watched the Padres beat the Milwaukee Brewers 3-2.

SEPTEMBER 21

Sanford-Burnham Cheer Tent On Sunday, Sept. 21, more than 40 volunteers came out to support Pedal the Cause San Diego at the Sanford-Burnham cheer tent located at the top of the Torrey Pines Mesa.

(From L to R) Institute CEO Perry Nisen, M.D., Ph.D., Hongbo Pang, Ph.D., Karthik Bodhinathan, Ph.D., Emily Smith, Ph.D., and Institute President Kristiina Vuori, M.D., Ph.D., at the Fishman Fund Awards Ceremony.

NOVEMBER 6

Sanford-Burnham’s NCI-Designated Cancer Center Open House The Cancer Center Community Advisory Board hosted the second Cancer Center Open House, titled, “The Science Behind Melanoma” on Nov. 6, for cancer survivors, their families and friends, and research advocates.

Sanford-Burnham volunteers, employees, and advocates cheer on Pedal the Cause riders.

Employees, their friends and family, along with members of our Cancer Center Community Advisory Board, showed their support for Pedal the Cause by cheering on all riders as they made their way up the steep Torrey Pines Mesa toward the final finish line at UC San Diego’s main campus.

Guests tour Cancer Center laboratories and take a closer look at the science behind melanoma at the Nov. 6 evening event.

Volunteers handed out ice-cold water and snacks to bikers and used cowbells, pom-poms, and high-fives to help motivate them to keep going.

Guests were invited to speak directly with cancer scientists during an informal reception, and join guided tours to learn about specific avenues of cancer research.

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from Molecules to Medicine “I just couldn’t utter those words anymore,” he recalled. “The work I was doing in my lab was getting recognition and grants, but I couldn’t see it touching patients, so I moved to industry where I felt that I could focus on creating future treatment options.”

Let’s Get Started One of Nisen’s signature industry achievements was the creation of GSK’s groundbreaking Clinical Study Requests database, giving external investigators access to the company’s drug trial results.

A June 2013 Forbes article reported that “GSK has made the bold commitment to be the first to contribute anonymized clinical trial data” and singled out Nisen’s “compelling” public statement in which he said, “In sharing our data with researchers around the world, we hope to further scientific research and increase

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understanding about our medicines. [...] Our belief is that this is the right thing to do for our industry and society—so let’s get started.”

One of Nisen’s priorities at Sanford-Burnham will be to foster alliances with pharmaceutical firms and health-care organizations. “I’ll put everything I have into making connections and establishing additional partnerships,” he said.

He also will reach out to donors and other stakeholders. “I look forward to being the external face of Sanford-Burnham,” he said, “and to helping others connect with the Institute and our outstanding science.”

On both Institute campuses, the new CEO will instill a “line of sight” ethos of scientists interacting with patients. “I want them to be influenced by talking to patients,” he told the San Diego Union-Tribune. “That has had a profound effect on me.”

Carl Ware, Ph.D., on the Benefits of Partnering

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You’ve had significant success establishing partnerships. What makes you an appealing partner to commercial organizations?

Targeting cytokines is a proven therapeutic arena that attracts the attention of the pharmaceutical industry. Drugs directed against the cytokine tumor necrosis factor are already used to treat patients with rheumatoid arthritis, colitis, and psoriasis. However, those drugs are not always effective and don’t work in other autoimmune diseases. Our research focuses on the more than 20 cytokines related to the tumor necrosis factor so that we may rewire the immune system to help patients who fail to respond to known drugs, or have other autoimmune diseases.

Collaborating with pharma is mutually beneficial. What does partnering bring to you that makes it worthwhile?

From my perspective, the partnerships I have with pharma bring industrial-scale power that aids in developing complex drugs made from proteins. By analogy, we design the blueprints and pharma builds the drug.

Why is Sanford-Burnham well positioned to become the partner of choice for the pharmaceutical industry?

I see a bright future for a strong academic and pharma alliance. The reasoning lies in the complementary strengths of the partners. Academia excels in uncovering new knowledge underlying disease processes, and part of that knowledge is identifying molecules, or targets, for which drugs can be made. Pharma brings deep knowledge and resources to drive a new drug through development and finally to the patient.

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

PAID

Sanford-Burnham Medical Research Institute

Partners in Science

The Honorable Mel Martinez Daniel Kelly, M.D., professor and scientific director of the Lake Nona campus, and former Florida Senator Mel Martinez, reviewing research conducted at the Diabetes and Obesity Research Center.

Former Florida Senator Mel Martinez visited Sanford-Burnham at Lake Nona on August 8 to meet with Daniel Kelly, M.D., professor and scientific director of the Lake Nona campus. They toured the Diabetes and Obesity Research Center and discussed some of the latest discoveries originating from the technology-rich Center.

Senator Martinez, who has a long history of championing innovation and economic development in the state of Florida, was instrumental to the effort to bring the Institute to Orlando. As a community leader in Central Florida, he has continued his strong support of medical research.

During his visit, Senator Martinez met with postdoctoral researchers hard at work at the lab bench, as well as senior director of Scientific Resources at Lake Nona and associate professor in the Cardiovascular Pathology Program, Stephen Gardell, Ph.D., who revealed some of the methods and tools that the Institute is employing to drive the translation of basic research discoveries into therapeutics with a tangible impact on people’s lives.