Portal

THE PATIENT IS WAITING

Accelerating the delivery of innovative, new medicines VOLUME 5 NUMBER 2 | SPRING 2015

www.sanfordburnham.org | PORTAL

1

FOUNDERS

Dr. William H. and Lillian Fishman HONORARY TRUSTEES

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

A Message from the CEO 1 The Patient is Waiting 2 A Link Between Alzheimer’s and Down Syndrome 4 Thinking Small: Fighting Osteoarthritis with Nanomedicine 5 How Genes Impact the Benefits of Exercise 6 The Power of Philanthropy: An Extraordinary Gift Establishes International Collaboration 7 New Faculty Join Sanford-Burnham 8

TRUSTEES AND OFFICERS

National Academy of Inventors 8

Gregory T. Lucier

Next Generation Profile: Karthik Bodhinathan, Ph.D. 9 It’s All About the People: Q&A with Trustee Hank Nordhoff 10

CHAIRMAN

Perry Nisen, M.D., Ph.D. CHIEF EXECUTIVE OFFICER DONALD BREN CHIEF EXECUTIVE CHAIR

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

Ask the CEO 12 Past & Upcoming Events 13 City of San Diego Back Cover

A Message from the CEO I can attest with pride that when SanfordBurnham scientists arrive at work each day, a current of enthusiasm, even passion, moves through the doors with them. They’re thinking about a friend struggling with a disabling disease, or a family member facing a shattering illness, but bravely wearing a hopeful smile.

Patients drive what we do at SanfordBurnham. Putting their immediate needs first as we conduct vital medical research ensures that Sanford-Burnham scientists keep pushing closer to cures for those who desperately need them.

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

CHIEF OF STAFF, VP

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

Elizabeth Schwarzbach, Ph.D.

EXECUTIVE VICE PRESIDENT

VICE PRESIDENTS, PHILANTHROPY

CHIEF ADMINISTRATIVE OFFICER TREASURER

Paul Baker Philip Graham, M.B.A.

Knox Bell

VICE PRESIDENT, COMMUNICATIONS EDITOR

CHIEF FINANCIAL OFFICER

Deborah Robison

SECRETARY

Lorenzo M. Berho James C. Blair, Ph.D. David W. Down Daniel J. Epstein 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.

2

PRODUCTION MANAGER & CONTRIBUTOR

THE PATIENT IS WAITING VOLUME 5 NUMBER 2 | SPRING 2015

Rhiannon Bruni ART DIRECTION

James Short WRITER

“Patients drive what we do at Sanford-Burnham.

Putting their needs first as we conduct medical research ensures SanfordBurnham scientists keep pushing closer to cures.”

Diane Wilson CONTRIBUTORS

ON THE COVER The patient is waiting for new, innovative medicines. SanfordBurnham scientists are mobilizing to address their unmet needs and accelerate the delivery of new medicines.

www.sanfordburnham.org

Karolyn Baker Patrick Bartosch Susan Gammon, Ph.D. Marissa Igartua DESIGN

Creative Fusion

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

Sanford-Burnham Medical Research Institute is a 501(c)3 nonprofit organization.

PORTAL | www.sanfordburnham.org

In this issue of Portal, you’re going to read about our research leaders and how patients and their families motivate their pursuit of new therapeutics and drive them to translate their discoveries into targeted leads for pharmaceutical development. You’ll also read about how a number of collaborations and initiatives, such as those with UC San Diego’s Moores Cancer Center and the Mayo Clinic, are accelerating the pace of our research in a variety of disease areas.

As Dr. Michael Jackson, senior vice president of Drug Discovery and Development at the Conrad Prebys Center

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

for Chemical Genomics, says, “It’s a heck of a responsibility.” One we take to heart.

Finally, you’ll learn about the impact that generous donors like you have on our research— including the recently announced $1 million gift from Karen and Stuart Tanz to establish an international collaboration between Sanford-Burnham and the Tanz Centre for Research in Neurodegenerative Disease at the University of Toronto. The Tanz family are concerned, impassioned citizens enacting change with their own dollars to improve human health. And that means a lot to our scientists and, ultimately, to patients.

The articles in this issue not only make for impactful reading, they underscore the tremendous talent within the Institute’s workforce. I consider it a privilege to have the opportunity to lead this group.

With the patient always in our minds and hearts, we’re moving therapeutics from bench to bedside at an ever-increasing speed.

Perry Nisen, M.D., Ph.D. CHIEF EXECUTIVE OFFICER DONALD BREN CHIEF EXECUTIVE CHAIR

www.sanfordburnham.org | PORTAL

1

PUTTING THE PATIENT FIRST

For a person diagnosed with Parkinson’s, cancer, or any of the numerous conditions for which there currently is no cure, the wait for an effective treatment can seem endless.

One question lingers: How long will it take?

At Sanford-Burnham, researchers carry a keen sense of this patient need throughout the workday. It stokes their perseverance and drives discovery. Now, powered by new research and development strategies, patients are part of a research model that will accelerate the delivery of new therapeutics.

Michael Jackson, Ph.D., senior vice president of Drug Discovery and Development.

“We’re in a golden age of biology,” says Michael Jackson, Ph.D., senior vice president of Drug Discovery and Development at the Conrad Prebys Center for Chemical Genomics. “Interdisciplinary partnerships and advanced technology are speeding up cures. The patient is directly in our line of sight and we’re shortening their wait.”

2

PORTAL | www.sanfordburnham.org

The state-of-the-art technology at the Prebys Center is one key to speeding cures.

Under Jackson’s direction, the Center screens chemical compounds by the millions to find the few with the potential to be developed into new medicines.

To maximize benefits for patients, all of the compiled results are added to a public database, PubChem, available to researchers around the world. “We have the capabilities here that you typically only see in giant pharmaceutical companies,” says Jackson. “Now we can marry research with innovative methods for actually starting the process of early drug development.”

That process is not without risk, which is why so many pharmaceutical companies have abandoned in-house drug discovery efforts. But Sanford-Burnham operates under a different model. “That’s what’s inspiring about the Institute,” says Jackson. “Here we have a nonprofit putting the patient first.”

SECOND CHANCE THERAPIES

Putting cancer patients first by providing them a second chance at therapeutics is the cornerstone of a current collaboration between Jackson and Seth Field, M.D., Ph.D., of the UC San Diego Moores Cancer Center.

Field recently identified a signaling pathway that is implicated in a number of cancers— pancreatic, colorectal, prostate, and ovarian— that, together, account for the majority of cancer deaths. Targeting the pathway is likely to provide cancer patients a unique therapy when other treatments have failed.

Susanne Heynen-Genel, Ph.D., director of Sanford-Burnham’s High-Content Screening Systems, is helping accelerate the discovery of a therapeutic targeting this pathway by orchestrating the drug screening process. She’s enthusiastic about the prospects. “There’s now a possibility that cancer patients will still have another avenue of treatment after relapse. It gives patients and their families hope in a situation where they need it the most.”

Searching through the library of compounds takes time, expertise, and money. Thanks to

a grant from Pedal the Cause, a San Diegobased fundraising event, this collaborative project found wings. “Dr. Field’s ideas had tremendous merit,” emphasizes Jackson, “but they weren’t developed far enough to apply for a federal grant. Now, I’m happy to say that the project results have proved so promising, we’ve just applied for a grant to expand this into a full-scale drug discovery effort.”

By pursuing research projects that have

Robert WechslerReya, Ph.D., director of the Tumor Initiation and Maintenance Program.

potential to deliver new drugs— even when risky or unproven— Sanford-Burnham underscores its focus on the patient.

COMBATTING DRUG RESISTANCE

Another collaboration between SanfordBurnham and the Moores Cancer Center is addressing the problem of drug resistance, common in patients with gastrointestinal stromal tumors.

This particular cancer arises from nerve cells in the intestine. Many of these tumors have a high level of a specific protein called KIT that can be targeted to treat the cancer, but for patients whose tumors don’t express KIT, or for those with tumors resistant to anti-KIT therapy, an alternative treatment approach is necessary.

This led Robert Wechsler-Reya, Ph.D., director of the Tumor Initiation and

Cancer research is moving at an accelerated pace, but there are still many unanswered questions. Partnering with elite National Cancer Institute-designated cancer centers like Sanford-Burnham Medical Research Institute provides important additional resources that help us transform scientific discoveries from the bench to the bedside, and to change how we treat cancer for the better. Together, we can save the lives of loved ones—in San Diego and around the world.

— Scott Lippman, M.D., UC San Diego Moores Cancer Center Director and UC San Diego Senior Associate Dean and Associate Vice Chancellor for Cancer Research and Care

Maintenance Program, to partner with Jason Sicklick, M.D., at the Moores Cancer Center to pursue a novel therapeutic. “There’s a great synergy here,” says WechslerReya. “Dr. Sicklick is a scientist and a surgeon. He understands tumors and has access to patient samples. I have extensive knowledge of how cancer cells grow and I know ways to target them.”

The pair is now using the Prebys Center’s screening technology to identify drugs that will specifically target KIT-resistant cancer cells, thus improving the outcome for patients who do not respond, or have become resistant, to current therapies.

Both researchers understand the need for speed in this endeavor. “We are including drugs in the screening process that have already been approved by the FDA for other diseases,” says Wechsler-Reya. “If we are lucky enough to find one of these, we can really shorten the regulatory process and rapidly move the drug to these patients.”

FACE-TO-FACE ON THE FRONT LINE

While all of Sanford-Burnham’s researchers work tirelessly in their labs, there are Continued on Page 13

www.sanfordburnham.org | PORTAL

3

Huaxi Xu, Ph.D., professor in the Degenerative Diseases Program at Sanford-Burnham.

A Link between Alzheimer’s and Down Syndrome The human brain is the single most sophisticated and enigmatic achievement of evolution.

Unlocking the mysteries of how it functions— or malfunctions, in some cases—is a personal mission for Sanford-Burnham’s Huaxi Xu, Ph.D., professor in the Degenerative Diseases Program. Xu leads a team of researchers whose goal it is to discover the precise mechanisms leading to aging-related diseases, such as Alzheimer’s, dementia, Parkinson’s, and other neurodevelopmental and neurodegenerative disorders.

Xu and his team have made significant strides toward this goal by unraveling a specific connection between Down syndrome and Alzheimer’s disease—a link that scientists had suspected for some time, but not proven.

As life expectancy for individuals with Down syndrome continues to grow—from 25 years in 1983 to 60 today—research

4

PORTAL | www.sanfordburnham.org

into the age-related challenges they face is Interested increasingly important. in Dr. Xu’s Xu’s recent research progress? explains how the extra Check out our copy of chromosome blog Beaker for 21 in individuals with updates on Dr. Down syndrome creates Xu and his team’s an increased risk of drug-screening developing dementia. efforts.

“We knew there was similar pathology related to the cognitive decline in both aging Down syndrome individuals and Alzheimer’s patients,” Xu explains. “We suspected that if there was a similar progression of symptoms, there could be a similar cause at work in both cases.”

The team was right. The study revealed that a protein called SNX27 regulates the generation of beta-amyloid, the main component of detrimental amyloid plaques found in the brains of people with Down syndrome and Alzheimer’s. By the age of 40, nearly 100 percent of all individuals with Down syndrome develop changes in the brain associated with Alzheimer’s disease.

“We are all living longer thanks to many factors, but it’s not just about living longer,” Xu explains. “It’s also about preserving a quality of life that makes it worth living.

By knowing that the same changes that cause dementia in Alzheimer’s patients are present in older Down syndrome individuals, doctors can prescribe mental exercises to strengthen memory earlier, as well as employ current medications to manage or alleviate symptoms before they manifest.”

But Xu and his team aren’t satisfied with only identifying the connection between Alzheimer’s and Down syndrome. They are determined to take their discovery and translate it into new, targeted therapeutics that could benefit both Alzheimer’s patients and individuals with Down syndrome.

The team is now working with Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics to screen and identify molecules to turn this goal into reality.

Alzheimer’s awareness

THINKING SMALL

Fighting Osteoarthritis with Nanomedicine The U.S. population is aging. At last count, one in eight citizens is over age 65. As the populace continues to grow older than ever before, more and more people are becoming uncomfortably familiar with the term “osteoarthritis” (OA).

This degenerative disease occurs over time as the protective cartilage on the ends of bones wears down. Joints become stiff and tender, and everyday tasks, even walking or bending, become difficult and extremely painful.

At Sanford-Burnham, Massimo Bottini, Ph.D., adjunct assistant professor in the Bioinformatics and Structural Biology Program, is searching for novel ways to defeat the degenerative condition.

He, along with his brother, Nunzio Bottini, Ph.D., associate professor in the division of cellular biology at the La Jolla Institute for Allergy & Immunology, are teaming up to use their complementary skills and studies to produce an innovative way to treat osteoarthritis.

The brothers are using nanomedicine— manipulating matter at the molecular level— to deliver anti-osteoarthritis drugs precisely to diseased cells, leaving neighboring cells unaffected. The pair’s groundbreaking work appears so promising that the Arthritis National Research Foundation has provided funding for their studies.

The brothers’ motivation: everyday contact with colleagues and patients who suffer from arthritis.

Dr. Massimo Bottini is a medical engineer while his brother, Dr. Nunzio Bottini, is a rheumatologist. They’ve been

collaborating for over 10 years. “If you want to succeed in science,” says Massimo, “you have to join forces. My brother and I have complementary expertise.”

Drs. Massimo and Nunzio Bottini at Sanford-Burnham’s La Jolla campus.

Their current advance comes in the form of a graphite nanoparticle 100,000 times thinner than a human hair. It is built to deliver drugs right into the nucleus of osteoarthritic chondrocytes, the cells found in cartilage. To date, their work on mice is showing exciting results. “Our method not only delivers the drug effectively, but stays in the joint for a prolonged time without causing side effects,” says Massimo.

“This is a significant improvement over previous attempts to deliver antiosteoarthritis drugs to affected joints.”

Clinical studies in humans lie in the near future, pending additional funding.

Until then, the Drs. Bottini will continue to hone their nanoparticle delivery system.

“The patient cannot wait too long,” says Massimo. “We are focused on improving the lives of those who suffer from arthritis.” www.sanfordburnham.org | PORTAL

5

Lauren Sparks, Ph.D., adjunct professor at Sanford-Burnham.

Did you know?

According to the U.S. Centers for Disease Control and Prevention, about 40 percent of Americans will develop diabetes within their lifetime.

6

How Genes Impact the Benefits of Exercise A crucial element of Sanford-Burnham’s 10-year strategic vision is to quickly move basic research discoveries to the patient. The Florida Hospital–Sanford-Burnham Translational Research Institute for Metabolism and Diabetes (TRI-MD) in Orlando, Fla., is helping to achieve that goal. At the TRIMD, scientists from both Sanford-Burnham and Florida Hospital have access to human tissue samples in order to test hypotheses and close the loop of the bench-to-bedside and back-to-bench research continuum.

One of the TRI-MD scientists is Lauren Sparks, Ph.D., adjunct professor at Sanford-Burnham. She and her team recently published a study in the Journal of Clinical Endocrinology & Metabolism that was widely discussed in national and international media. The paper describes how exercise does not have a positive impact on blood-sugar levels in up to 20 percent of type 2 diabetics. The research indicates that the effect of exercise may be linked to genetic factors.

“Since obesity and lack of physical activity are two key risk factors for type 2 diabetes, physicians frequently recommend exercise to prevent or manage the disease,” said Sparks. “Most people benefit from an exercise regimen, but our research indicates that a significant minority of individuals with type 2 diabetes do not experience the same improvements in metabolism due to their genes.”

Sparks’ team analyzed studies in which people with type 2 diabetes participated in exercise regimens, and also examined animal and genetic studies. They found that around 15 to 20 percent of type 2 diabetics did not see any improvement in their blood-sugar control, insulin sensitivity, or fat-burning capabilities. This resistance to exercise is encoded in DNA and can be handed down through generations.

“More research is needed to determine which people with, or at risk of developing, type 2 diabetes will respond to an exercise program and which will not,” Sparks said. “Genetic and epigenetic patterns could hold the key to differentiating between the two groups. With that information in hand, we can target specific interventions and treatments to the individuals who will benefit most and identify novel treatment approaches to help those who do not respond to exercise.”

Of course this doesn’t mean you should stop exercising if you have type 2 diabetes. Even if exercise does not improve the management of diabetes in some, the benefits of physical activity, such as improved cardiovascular health, still apply.

PORTAL | www.sanfordburnham.org

The Power of Philanthropy: An extraordinary gift establishes international collaboration

By any calculation, the 2014 “Top Hat” gala was a fantastic evening filled with great entertainment and the remarkable generosity of Sanford-Burnham supporters.

Honorary gala co-chairs Stuart and Karen Tanz “stole the show” with the announcement of their extraordinary $1 million gift aimed at treating neurodegenerative disease.

The gift provides funding to launch a collaboration between Sanford-Burnham and the Tanz Centre for Research in Neurodegenerative Diseases at the University of Toronto.

It will bring together the two organizations’ unique and complementary strengths.

On January 8, 2015, the collaboration officially kicked off with scientists from both organizations meeting at Sanford-Burnham’s La Jolla, Calif., campus to outline their upcoming joint research projects.

“Creating new medicines that tackle challenging diseases is a daunting task that demands a cross-disciplinary, teambased approach,” said Perry Nisen, M.D., Ph.D., CEO of Sanford-Burnham. “The collaboration between Sanford-Burnham and the Tanz Centre is an exceptional opportunity to accelerate the development of new drugs by capitalizing on key strengths from each organization.”

The collaboration will bring together the cutting-edge resources of SanfordBurnham’s drug discovery platform with the Tanz Centre’s expertise in neurobiology, preclinical testing of prototype therapeutics, and diagnostics for identifying and monitoring individuals at risk.

“The Tanz Centre is an international leader with a track record of outstanding discoveries in neurodegenerative disease

Karen and Stuart Tanz at the November 2014 “Top Hat” gala.

research. Our expertise in the discovery and validation of novel therapeutic targets and diagnostic biomarkers offers deep insight and knowledge into the molecular understanding of these diseases,” said Peter St. GeorgeHyslop M.D., director of the Tanz Centre.

Historically, universities, hospitals, and research institutes have identified and validated new disease targets for drug discovery. Building on their progress, pharmaceutical companies have used this information to mount drug development programs to take drug candidates into clinical development.

However, pharma R&D success rates have declined while costs have risen, resulting in a major reduction of new drug candidates on the horizon.

“A new approach and fresh thinking are necessary to tackle the problems of neurodegenerative diseases, which are attributes my family believes these two organizations share,” explained Stuart Tanz, an advocate for establishing the collaboration and the architect of the family gift. “Together, the Tanz Centre and SanfordBurnham have a complementary set of capabilities to go from disease target to the production of drug candidates ready for clinical testing.” www.sanfordburnham.org | PORTAL

7

INSTITUTE NEWS

INSTITUTE NEWS

New Faculty Join Sanford-Burnham Four new faculty members have joined Sanford-Burnham Medical Research Institute.

With a wide range of scientific expertise, they are a welcome addition to the campuses.

Cosimo Commisso, Ph.D., is an assistant professor in the Cell Death and Survival Networks Program. He completed his Ph.D. in molecular genetics and developmental biology at the University of Toronto, Canada.

Commisso is investigating the regulatory mechanisms that control Kras-stimulated macropinocytosis and their role in cancer development and progression. His research could lead to therapies to treat Kras-mutated pancreatic cancer. Kras mutations are present in 80 to 95 percent of all pancreatic cancers.

Peter A. Crawford, M.D., Ph.D., is an assistant professor and associate director of the Cardiovascular Pathobiology Program.

Crawford is a physician scientist trained at Washington University in St. Louis. He is a cardiologist and a National Institutes of Health-funded expert in metabolic sciences.

His main research focus lies in examining how fuel metabolism can be modified in situations of cardiometabolic stress.

Maximiliano D’Angelo, Ph.D., is an assistant professor in the Development, Aging, and Regeneration Program.

He received his Ph.D. in molecular biology from the University of Buenos Aires, Argentina. In 2012, he was one of 22 young researchers named a Pew Scholar in Biomedical Sciences by the Pew Charitable Trusts.

D’Angelo is exploring the basic biology of nuclear pore complexes—the protein channels that connect the nucleus with the cytoplasm—and their relationship to diseases associated with aging, such as cancer.

8

PORTAL | www.sanfordburnham.org

Aniruddha (Ani) Deshpande, Ph.D., is an assistant professor in the Tumor Initiation and Maintenance Program.

Deshpande completed his Ph.D. in human biology at the Ludwig-Maximilians University of Munich, Germany. His research interests lie in how DNA modifications drive normal and malignant stem cell development.

Cancer stem cells are cells that persist in tumors and cause relapse and metastasis by giving rise to new tumors.

The Institute is pleased to welcome these distinguished scientists to the faculty.

Institute News: National Academy of Inventors Two outstanding SanfordBurnham scientists, Erkki Ruoslahti, M.D., Ph.D., distinguished professor and former president of the Institute, and Kristiina Vuori, M.D., Ph.D., current president, have been named Charter Fellows of the National Academy of Inventors (NAI).

Ruoslahti and Vuori are now part of a group of only 414 NAI Fellows from more than 150 prestigious research universities, government, and nonprofit research institutions. Election to the NAI Fellows is a significant professional distinction accorded to academic inventors who have demonstrated a highly prolific spirit of innovation in creating, or facilitating, outstanding inventions that have made a tangible impact on quality of life, economic development, and the welfare of society.

Academic inventors are elected by their peers for innovative contributions in areas such as patenting and licensing, innovative discovery and technology, significant impact on society, and support and enhancement of innovation.

Congratulations to Drs. Ruoslahti and Vuori on this prestigious honor.

“To be a principal investigator in the 21st century, you must be a hybrid scientist—capable of leading a laboratory with one foot firmly rooted in the lab, with the other in the meeting room among drug developers.”

Next Generation: Karthik Bodhinathan, Ph.D. When asked to describe his favorite way to unwind, Karthik Bodhinathan, Ph.D., a postdoctoral fellow in the Degenerative Diseases Program, admits to indulging in autobiographies from world leaders. Heavy reading before bed, but no doubt worth the extra effort for the dedicated young scientist.

Last autumn, he was selected to receive the prestigious Fishman Fund Award, an honor reserved for the Institute’s most gifted and promising postdoctoral researchers. Bodhinathan, who received his Ph.D. in neuroscience and M.S. in business management from the University of Florida, joined Sanford-Burnham in 2014 with one goal in his sights—discover and develop drugs to treat neurodegenerative diseases.

He’s already making significant progress.

Bodhinathan’s research focuses on molecules that regulate communication between neurons in the brain. Specifically, he studies the role of ion channels in the pathology of neurodegenerative diseases.

“I want to be more than a neuroscientist,” Bodhinathan says. “I want to lead a team of researchers, manage a lab, secure partnerships, and shepherd new drugs through the discovery process. To

Cross-section of a mouse brain depicting the expression of alphasynuclein, a protein being studied for its role in Parkinson’s disease (PD). Raw traces of abnormal brain signals (in red) observed in PD. The signals were recorded from a single neuron, located in the brain’s “memory encoding” region called the hippocampus (black arrow).

Dr. Karthik Bodhinathan, postdoctoral fellow in the Degenerative Diseases Program.

achieve that, one has to understand drug development and possess business acumen to foster successful partnerships.”

Bodhinathan is definitely on to something.

With shrinking federal funding and more competition for philanthropy dollars, he is part of the next generation of scientists who must work harder than ever to fund their research, which helps to explain those autobiographies he enjoys. They help him to uncover the characteristics and skills needed to be a leader despite any obstacle.

“Those autobiographies are proof that anyone can conquer insurmountable challenges,” he explains. “To be a principal investigator in the 21st century, you must be a hybrid scientist—capable of leading a laboratory with one foot firmly rooted in the lab, with the other in the meeting room among drug developers—that’s a major challenge.

But it’s also why I picked Sanford-Burnham for my postdoctoral training. The Institute is a pioneer of translational research. There is no better place to refine the skills to successfully develop new medicines for the future.” www.sanfordburnham.org | PORTAL

9

IT’S

ALL

Sanford-Burnham grow its income to expand opportunities and increase the pace of developing products that help people.

ABOUT THE PEOPLE Q&A with Henry L. “Hank” Nordhoff

Hank Nordhoff is a master when it comes to directing the strategic growth of biotech companies. He says it all starts with having the right people. Currently executive chairman of Banyan Biomarkers, Inc., and Cypher Genomics, he previously headed Gen-Probe for 15 years, leading the company to phenomenal growth. An energetic member of Sanford-Burnham’s Board of Trustees, Nordhoff shares his philosophy for getting the most out of people and companies.

You contribute your talents to a number of boards in the San Diego area. What drew you to Sanford-Burnham?

I joined in 2012 because of Malin Burnham and John Reed. For me, it’s all about the people and I’ve always had a lot of admiration for both gentlemen. Of course, I’d heard about Sanford-Burnham’s incredible productivity and ability to make meaningful medical discoveries, and transition those discoveries into treatments and diagnostics that improve human health.

How do you motivate people?

Make it easy for people to motivate themselves! Start with the best people— those with exceptional talent, training, and enthusiasm. Enthusiasm is important because it’s infectious and creates energy.

10

PORTAL | www.sanfordburnham.org

Sanford-Burnham is emphasizing translational research and getting novel medicines to the patient more quickly. In your experience, what is the best way to accelerate this process?

You take those people and put them in an environment with resources and freedom to be heroes. In medical research, promoting success, acknowledging the superstars, and continually supporting their needs are key to motivation. The best support systems fuel the best people to operate at their maximum potential—and that’s when game-changing discoveries are made.

As CEO of other biomedical research firms, you helped the companies grow significantly.

Is bigger better? How do you envision Sanford-Burnham’s future growth?

What I like to do is grow companies organically and help them get better, not necessarily bigger. You can get bigger simply through acquisitions, but not necessarily better. What I like about SanfordBurnham is that you have these pods of entrepreneurship. You have novel ideas and people are committed to them. There’s a sense of urgency here and that makes people productive. So I say, keep getting the best people, let them set aggressive targets, and give them a shot at making them. I also am excited about Sanford-Burnham’s goal of collaborating with industry. That brings in money and provides an accelerated route to commercializing products that impact people’s health. My goal is to help

Partnering with pharma and investing in biomarkers. By using biomarkers—a molecule, protein, or gene that’s unique to a subset of individuals with a specific disease—you accelerate clinical development because you can separate the patients who will respond to a drug (the responders) from those who won’t (the non-responders).

If you know who your responders are, you can use clinical studies to pool knowledge and resources, saving time and money. The future is individualized medicine.

Increasingly, research institutions are collaborating on projects and sharing their resources. Are there any potential pitfalls to this otherwise beneficial process?

When you have collaboration, some organizations communicate very well and some don’t. One of the first things you have to do is to clarify the patent situation. Once you agree on how to share know-how and patents, you establish trust. Respect soon follows.

Sanford-Burnham has recently embarked on a new 10-year vision to accelerate discoveries that impact human health. How do you see this vision affecting research priorities?

I think the four areas of priority— neurological disorders, cancer, immune system diseases, and metabolic diseases—are the right ones. I’m especially big on the importance of the brain and discovering an effective treatment for Alzheimer’s disease.

Biomarkers may play a significant role in the early detection of Alzheimer’s. Drugs that are not particularly effective in established Alzheimer’s may be useful in delaying the onset of Alzheimer’s if given early enough in its progression. I believe the brain is where we are going to have our biggest impact.

“

What I like

about

Sanford-Burnham is that you have these

pods of

entrepreneurship.

You have novel ideas and people are

committed

to them.

There’s

sense of urgency here a

”

and that makes

people productive.

—Hank Nordhoff

www.sanfordburnham.org | PORTAL

11

Past Events Rare Disease Day

Sanford-Burnham hosted the sixth annual Rare Disease Day Symposium February 27-28, 2015. The two-day symposium, chaired by Hudson Freeze, Ph.D., professor and director of the Human Genetics Program, focused on treating rare diseases with simple sugars. The first day featured presentations by the world’s leading experts, while the second provided the opportunity for patients to meet with clinicians and scientists in a doctor-is-in session.

Upcoming Events Pedal the Cause 2015

Pedal the Cause is gearing up for the third annual bike ride on September 18-20, 2015.

The only multi-day cycling fundraiser to support cancer research in San Diego, Pedal the Cause provides critical funding to the community’s three NCI-designated cancer centers.

Early-bird registration is now open. Sign up today at sandiego.pedalthecause.org.

Annual Gala

Sanford-Burnham’s annual gala will be held Saturday, November 7, at the Grand Del Mar. Guests will enjoy a memorable evening of delectable food, top-notch entertainment, and fabulous company in support of vital medical research. Please mark your calendars to attend!

ASK THE CEO Q. What does Sanford-Burnham need to expand or build upon to be successful?

A: We must find ways to better integrate activities of the Prebys Center with the deep biological insight of the fundamental scientists at Sanford-Burnham. We also need to secure financial sustainability at a time when the ability to obtain National Institutes of Health support is increasingly difficult.

Financial sustainability will primarily have to come from expanded relationships with pharma and other partners, philanthropy, and value creation through discovering and developing medicines.

Q. In your opinion, what sets Sanford-Burnham apart from other research institutes?

A: The fact that we are home to cutting-edge, fundamental research and professional drug discovery under one roof.

Q: In these first few months at Sanford-Burnham, what has surprised you?

A: I am impressed by the breadth and depth of the incredible science happening here.

Q: If there were one thing at Sanford-Burnham that you could change right now, what would it be?

A: I would like to improve communication internally between scientists and externally with the public.

Do you have questions for Dr. Nisen?

Or do you want to know more about what’s coming up in the Institute’s future? Let us know. We want to hear from you. Please send your questions to asktheceo@sanfordburnham.org and your question may be featured in the next issue of Portal. Or connect with us through our blog, Beaker, or Facebook at facebook.com/sandfordburnham.

12

PORTAL | www.sanfordburnham.org

The Patient Is Waiting Continued from Page 3 some who take their work to the front line, meeting with the patient face-to-face.

Fred Levine, M.D., Ph.D., is a prime example. As well as being director of the Sanford Children’s Health Research Center and an associate professor of pediatrics at UC San Diego, Levine is also a practicing physician. His specialty is treating children with inherited metabolic diseases.

“There’s so much unrealized potential in these children,” he says. “That’s what keeps me motivated. It’s a great joy when we make a diagnosis and start an effective therapy.”

In his lab, Levine delves deeper into how existing cells in the pancreas convert into beta cells destroyed in type 1 diabetes. It’s an intriguing line of discovery, one with the potential to provide a groundbreaking therapeutic for patients with type 1 diabetes.

Clinical researchers and scientists at the Florida Hospital—Sanford-Burnham Translational Research Institute for Metabolism and Diabetes also work directly with patients. There, Sheila Collins, Ph.D.,

professor in the Metabolic Disease Program, works with Richard Pratley, M.D., director of the Florida Hospital Diabetes Institute, to test the effectiveness of a heart hormone on increasing the body’s levels of brown fat, a type of “good fat” that improves metabolism. Initial results from testing this new route to treating obesity are promising.

Not entirely by coincidence, perhaps, Collins is an experienced fitness trainer, which has put her face-to-face with people struggling with metabolic issues.

“Because of all I’ve learned, I have a deeper appreciation for what people need to do to achieve fitness and maintain proper body weight. On a day-to-day basis, that keeps me pushing toward the next discovery.”

POWERING THE CURE

For Sanford-Burnham scientists, it always comes back to the patient and the Institute’s mission: From Research, the Power to Cure. Those words voice the passion felt by every Sanford-Burnham researcher, technician, and staff member. www.sanfordburnham.org | PORTAL

13

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

FPO

PAID

Sanford-Burnham Medical Research Institute

XX%

Cert no. XXX-XXX-XXXX

PARTNERS IN SCIENCE

San Diego mayor Kevin Faulconer visited Sanford-Burnham’s La Jolla campus on February 4 for a very special speaking engagement with the Group of 12 (G12), a community service group that meets every month on campus to hear compelling lectures from experts in academic, political, and professional disciplines.

During his visit, Faulconer, a strong advocate for San Diego’s biomedical research community, spoke frankly about his vision to create new opportunities for San Diegans, as well has his plans for growing and enhancing San Diego’s (From L to R) Doreen Schonbrun, Sanford-Burnham Board member Jeanne biotech cluster. Additionally, Faulconer touched Herberger, Ph.D., Sanford-Burnham CEO Dr. Perry Nisen, Sanford-Burnham President Dr. Kristiina Vuori, Mayor Kevin Faulconer, Reena Horowitz, and on his commitment to the recently announced Hélène Gould at the February 4 event.

Alzheimer’s Part the Clouds project—a citywide initiative designed to bring the high concentration of Alzheimer’s researchers in San Diego together with caregivers, advocates, and patients to search for a cure.

Researchers in the coalition, including Sanford-Burnham’s own senior vice president of Drug Discovery and Development, Michael Jackson, Ph.D., will work collaboratively to expedite the development of new therapeutics to treat Alzheimer’s.

With the support of the city of San Diego, Sanford-Burnham is poised to play an important role in the citywide initiative to beat Alzheimer’s for good.

14

PORTAL | www.sanfordburnham.org

Photo courtesy of Vincent Andrunas.

City of San Diego