Winter 2012

YOUR DOORWAY TO SCIENTIFIC DISCOVERY

Hunting for New Medicines:

A Journey of Discovery Drug Repurposing: A “New” Source of Treatments Exploring the Human Microbiome

VOLUME 3 NUMBER 1 | WINTER 2012

www.sanfordburnham.org | PORTAL

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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

M. Wainwright Fishburn, Jr.

A Message From John Reed, CEO 1 A Journey of Discovery 2 Drug Repurposing: a “New” Source of Treatments 4 Nobel Prize-Winning Technology at Work 5 What is “Disease in a Dish”? 5 Talking With a Donor: Jeanne Jones 6 Talking With a Scientist: Scott Peterson 7 Seeking Medicines in the Natural World 8

Uncharted Territory

CHAIRMAN

Please Join Us in Congratulating Erkki Ruoslahti 9

Duane J. Roth

Recent Events 11

VICE CHAIRMAN

Community Outreach 12

John C. Reed, M.D., Ph.D. CHIEF EXECUTIVE OFFICER PROFESSOR AND DONALD BREN CHIEF EXECUTIVE CHAIR

Upcoming Events 13 Philanthropy Catalyzes Great Science Back Cover

Kristiina Vuori, M.D., Ph.D. PRESIDENT PROFESSOR AND DIRECTOR, NCIDESIGNATED CANCER CENTER PAULINE AND STANLEY FOSTER PRESIDENTIAL CHAIR JEANNE AND GARY HERBERGER LEADERSHIP CHAIR IN CANCER RESEARCH

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

SENIOR VICE PRESIDENT, EXTERNAL RELATIONS

Ann Carollo YOUR DOORWAY TO SCIENTIFIC DISCOVERY

PORTAL EDITOR

Kristina Meek, M.A.

Margaret M. Dunbar, J.D.

CONTRIBUTORS

Patrick Bartosch Heather Buschman, Ph.D. Sam Reed James Short

SECRETARY

Lorenzo Berho Shehan Dissanayake, Ph.D. Daniel J. Epstein Pauline M. Foster Alan A. Gleicher Jeanne L. Herberger, Ph.D. Brent Jacobs James E. Jardon II Stuart Lipton, M.D., Ph.D. J. Bernard Machen, D.D.S., Ph.D. Nicolas C. Nierenberg Hank Nordhoff Douglas Obenshain Peter Preuss Duane J. Roth Andrew J. Viterbi, Ph.D. Allen R. Weiss Gayle E. Wilson EX-OFFICIO

Todd Golub, M.D.

SCIENTIFIC ADVISORY BOARD CHAIRMAN

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VICE PRESIDENT, COMMUNICATIONS

Deborah Robison

EXECUTIVE VICE PRESIDENT CHIEF ADMINISTRATIVE OFFICER CHIEF FINANCIAL OFFICER TREASURER

Hunting for New Medicines:

A Journey of Discovery Drug Repurposing: A “New” Source of Treatments Exploring the Human Microbiome

DESIGN VOLUME 3 NUMBER 1 | WINTER 2012

Creative Fusion

ON THE COVER

Michael Jackson, Ph.D., vice president of drug discovery and development, leads the charge in searching for potential new treatments and cures at Sanford-Burnham.

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 decade ago, we set out on a journey, embarking on a new 10-year plan for the Institute. A major part of that plan was to increase our investments in the technology and talent needed for drug discovery. This emphasis provided us with new tools and greater possibilities for realizing our motto: From Research, the Power to Cure.

In a way, we were setting out into uncharted territory. Technological resources of the caliber found in our drug screening center were new to the nonprofit research community. The human genome had been sequenced only the previous year. We found ourselves with the ability to identify small drug-like chemicals that might turn on or off specific genes (or the proteins they encode) to effect a change in the body. That meant an entirely new world of possibilities for potential medicines.

Every journey of exploration needs funding, and ours was no exception. In 2008, the National Institutes of Health awarded us a six-year, $98 million Molecular Libraries Probe Production Centers Network (MLPCN) grant to support our drug screening center.

Additional support from philanthropist Conrad Prebys and other generous donors has helped build the Conrad Prebys Center for Chemical Genomics, the most advanced drug discovery center in the nonprofit world.

In the following pages you will learn some of the ways we’ve made progress toward inventing new medicines thanks, in large part, to the capabilities of the Prebys Center.

You’ll read about researchers who embark upon adventures to discover scientific truths and treasures. From far beneath the ocean to inside the human microbiome, SanfordBurnham researchers pursue potential treatments and cures. Other “treks” featured in this issue apply advanced technologies to uncover new applications for existing medicines or accelerate the concept of personalized medicine.

As we focus on the next 10-year plan, our journey continues, bolstered by success and primed for even greater discoveries.

As a donor, you walk this path beside us and we are ever grateful. I look forward to continuing to chart the future of scientific discovery together.

Sincerely, John C. Reed, M.D., Ph.D. CHIEF EXECUTIVE OFFICER DONALD BREN CHIEF EXECUTIVE CHAIR

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A Journey of Discovery

Intrepid researchers and investors travel a long, challenging road to uncover new treatments and cures.

At Sanford-Burnham, the process starts in the Conrad Prebys Center for Chemical Genomics, our state-of-the-art drug discovery facility. The numbers below represent new medicines currently under investigation at Sanford-Burnham. Successfully completing the journey of discovery requires dedication, financial resources, the willingness to take risks, and— IN PROGRESS above all—a commitment to helping people.

Screens

Hits

Leads

IN PROGRESS 32 Screens IN PROGRESS

IN PROGRESS 12 Hits IN PROGRESS

IN PROGRESS 12 Leads IN PROGRESS

IN PROGRESS 12 Hits IN PROGRESS

IN PROGRESS 12 Leads IN PROGRESS

A scientist identifies a biological “target”—a gene or protein that malfunctions in a particular disease. Drug discovery experts screen hundreds of thousands of chemical compounds to find some that might alter the target’s behavior.

32 Screens

A robotic arm in the Prebys Center holds a plate capable of testing 1,536 samples at a time

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32PROGRESS Screens IN 32 Screens

From their chemical compound screening efforts, the team IN PROGRESSa few “hits”—chemicals identifies 32PROGRESS Screens IN that act on the target.

32 Screens

12 Hits

Medicinal chemists take the most effective hits and optimize IN PROGRESS them by making closely related 12PROGRESS Hits with improved IN molecules 12 Hits qualities. The most drug-like promising chemicals, or “leads” are then tested to see if they are effective in a model organism.

12 Leads

IN PROGRESS 3IN Prototype PROGRESS Drugs

12 Hits Prototype Drugs

12 Leads Clinical Trials

3 Prototype Drugs Approval

IN PROGRESS 3IN Prototype PROGRESS Drugs

IN PROGRESS Phase I 19; IN PROGRESS

3 Prototype Drugs

Phase 16; Phase III 8 Phase II I 19; Phase II 16; Phase III 8

SUCCESSES 1 Cancer Drug, SUCCESSES

IN PROGRESS Phase I 19; IN PROGRESS

SUCCESSES 1 Cancer Drug, SUCCESSES

After further optimization one or more prototype drugs are selected to advance to the clinic.

IN PROGRESS At this point the team typically 12PROGRESS Leadswith a pharmaceutical IN partners 12 Leads to conduct precompany clinical safety testing.

There are three phases of clinical trials a drug must pass before it is approved to go to market.

IN PROGRESS These trials test a drug’s safety 3 Prototype IN PROGRESS and efficacy inDrugs humans.

3 Prototype Drugs

If it makes it past all of these hurdles, a drug is reviewed by the FDA. If approved, it goes IN PROGRESS to market, Phase I 19;where it can benefit IN PROGRESS patients.

Sanford-Burnham Phase 16; Phase III 8 Phase II I 19; research Phase II has 16; contributed Phase III 8 to three drugs currently on the market.

21 Cancer Heart Disease Drug, Drugs 2 Heart Disease Drugs

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Drug Repurposing:

A “New” Source of Treatments

Layton Smith , Ph.D.

Discovering a new compound that, in turn, becomes a new drug is like finding a rare treasure. It takes vast amounts of time, money, and dedication. The bold explorers leading the charge leave no stone unturned, no territory uncharted. Sometimes, the treasure is right under our feet, but a resourceful scientist with creativity, motivation, and sophisticated tools is required to unearth it.

Researchers are turning increasingly to the practice of drug repurposing—reexamining existing medicines for potential new uses. “The results can be startling,” says Michael Jackson, Ph.D., vice president of drug discovery and development. “For example, we discovered that a drug that was approved to treat cancer might be a valuable new treatment for patients suffering from muscular dystrophy.”

Repurposing a drug is safer, faster, and less expensive than starting from scratch.

More than 90 percent of new drugs fail during development, contributing to the high cost of pharmaceutical research and development.

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Since an existing drug has already passed a significant number of important tests, it is already way ahead of the game. The Conrad Prebys Center for Chemical Genomics, which spans both Sanford-Burnham locations—in La Jolla and Lake Nona—can screen these existing drugs quickly and efficiently for new uses. Dr. Jackson adds, “A new therapy based on a repurposed drug could benefit patients sooner—ultimately saving and improving more lives.”

One challenge to this process is that the current drug repurposing library (collection) available to scientists is incomplete. Fortunately, a comprehensive listing of clinically approved drugs was recently made available by the National Institutes of Health. Now, in order to make the promise of drug repurposing a reality, Sanford-Burnham researchers are building the world’s most complete drug repurposing library, which can be leveraged against our strengths in stem cell biology, phenotypic cell-based screening, and high-throughput drug screening. Moreover, this library will be made available to other nonprofit research

“A new therapy based on a repurposed drug could benefit patients sooner— ultimately saving and improving more lives.” institutions to advance translational medicine.

“Philanthropic support for building our drug re-purposing library is critical since federal funding is becoming more restrictive.

This is an area of great promise—where philanthropy can truly benefit patients,” says Layton Smith, Ph.D., director of drug discovery at Sanford-Burnham at Lake Nona.

The more complete this library, the greater the chance we will find a drug that can potentially be repurposed to treat patients. You can help build the library and give our “explorers” greater odds of making that next important discovery. Just contact us at (877) 454-5702 to learn how.

Nobel Prize-Winning Technology at Work Among the invaluable tools in SanfordBurnham’s quest for new medicines are induced pluripotent stem (iPS) cells. The technology behind this special type of laboratory-made stem cell received worldwide attention in October when Drs. John B. Gurdon and Shinya Yamanaka, the scientists who helped make it possible, won the 2012 Nobel Prize in Physiology or Medicine.

Like other types of stem cells, iPS cells have the potential to become any other type of cell in the body. This makes them especially useful for studying cell types that are hard to access in a living person—brain cells, for example.

Sanford-Burnham has been at the forefront of iPS cell research almost since the technique’s inception. We are using

iPS cells to study the root causes of disease and to test chemicals for their potential to work as medicines. (See below for more details.) In 2011, we launched a new iPS cell program within our Stem Cell Research Center, reaffirming our commitment to this technology and making it available to our scientists, as well as other researchers in the greater scientific community. We are now poised to become the largest notfor-profit source of human iPS cells.

Here’s the best part about iPS cells: Because they are made from existing cells in your body, usually skin cells, they exhibit your unique genetic makeup. This ability marks an important step toward personalized medicine.

What Is “Disease in a Dish”?

Sanford-Burnham researchers use iPS cells derived from individual patients to create a model of a person’s unique disease in a laboratory dish. The advantage of disease-in-a-dish modeling is that it generates large numbers of otherwise hard-to-access cell types—such as brain cells—complete with a person’s unique genetic and epigenetic make-up. These characteristics make disease

in a dish a valuable tool for discovering and developing therapies that are more personalized to the individual.

Scientists in the Conrad Prebys Center for Chemical Genomics use these diseasein-a-dish models to search for chemicals that correct the cellular defects behind conditions such as Parkinson’s disease or muscular dystrophy.

These chemicals might then

be further developed into the precursors for new medicines.

Potential new medicines can then be tested to find out how that specific patient responds, including what side-effects that person might experience.

Prebys Center scientists also use the technique to look for new applications for existing medicines, an approach known as drug repurposing (see page 4).

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TALKING WITH A DONOR

TALKING WITH A SCIENTIST

Jeanne Jones:

Exploring New Frontiers

Jeanne Jones and her husband, Don Breitenberg, at the 2010 Sanford-Burnham gala

A warm, enthusiastic cookbook author who calls La Jolla home, Jeanne Jones has always been one to explore new frontiers. SanfordBurnham’s entrepreneurial spirit resonates with Jeanne and her forward-thinking approach to cooking. She was venturing into new territory in light cooking in the 1960s, decades before “The Biggest Loser” or The South Beach Diet. When she wrote her second book, focused on eating for heart health, she recalls with a smile, “Every editor asked me what saturated fat was.” Today, Jeanne is the acknowledged leader in the field of light cuisine. In addition to more than 30 books, she writes a syndicated column, “Cook It Light,” which reaches 30 million readers a week. Numerous world-class hotels, spas, and restaurants have sought her counsel.

Jeanne admires Sanford-Burnham’s commitment to collaboration, something she values in her own work. “I’ve cooked with a lot of different people, of all different

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skill levels. No matter who they are, experienced or inexperienced, I always learn something. Sometimes you even learn things you don’t want to,” she laughs. She likens this type of interaction to the way that scientists share information, sometimes finding new ways of seeing a problem after discussing it with a colleague.

When asked what first drew her attention to Sanford-Burnham, Jeanne says, “It was the scientists, their willingness to share with one another, and their compassion.” Her family began supporting cancer research here when her sister was diagnosed with ovarian cancer. “Once you start giving to SanfordBurnham, you find yourself involved,” she says, “and you get to know everybody.”

Her relationship with Sanford-Burnham really blossomed when she agreed to cochair our fundraising gala in 2007. She brought the same innovative approach she takes with cooking to the Institute’s biggest annual event—and set a new bar.

She and co-chair Cathryn Ramirez planned a seven-course wine and food pairing, something that had never been done at a major San Diego fundraising event. The two women meticulously designed a memorable, high-class experience. Detractors feared that not enough people would purchase tickets, which were priced at a premium, yet the event sold out well in advance. Of course, the real value in a gala ticket is the funding it provides for medical research. That gala raised more money than any previous Sanford-Burnham event. Some of the strategic choices Jeanne made have influenced the course of the event year after year.

Jeanne’s success has allowed her to give generously to a number of carefully selected institutions. What they all have in common, she says, is “an entirely different approach. They take risks… they desire to be a leader.” Clearly, she has found a kindred spirit in Sanford-Burnham.

We don’t always have to think about treating disease with drugs. We should also be mining the human microbiome for therapeutic compounds–things akin to vitamins and herbs–that can keep us from getting sick in the first place.

Scott Peterson:

Microbe Hunter

Trillions of microbes—bacteria, mostly—live on our skin, in our mouths, and in our guts.

Together they make up the microbiome, which might seem like an unusual source of health information, but that’s exactly where Scott Peterson. Ph.D., is searching. While scientists in Sanford-Burnham’s Conrad Prebys Center for Chemical Genomics are hunting down new medicines that could help us when we get sick, Dr. Peterson is seeking molecules that keep us healthy.

“In my view, we don’t always have to think about treating disease with drugs. We should also be mining the human microbiome for therapeutic compounds—things akin to vitamins and herbs—that can keep us from getting sick in the first place,” says Dr. Peterson, who joined Sanford-Burnham’s faculty in August, as a professor in our Infectious and Inflammatory Disease Center.

Only a fraction of the roughly 100 trillion cells in your body are actually human—the rest are bacteria and other microorganisms. We and our microbiomes live in a mutually beneficial balance. We provide our microbes with a home and food while they help protect us from other more menacing bacteria and viruses.

With the recent realization that our microbial makeup might impact our health in many additional ways, scientists have undertaken the challenge of sequencing the human microbiome. From these microbiome sequencing projects, we’ve found that, in terms of bacterial species, different people have different microbiomes. We’ve also learned that a person’s unique microbiome can fluctuate with health, disease, and time.

But the field is rapidly changing. “The

Scott Peterson, Ph.D.

characterization phase of the microbiome— where we sequenced and cataloged bacterial species to put order to complexity—is coming to an end,” Dr. Peterson explains.

“We now know for the most part which species are where. The next phase will have more to do with how they interact and communicate with one another and with us, what goes wrong when disease develops, and what’s needed to maintain health.”

That’s what brings him to SanfordBurnham. Until recently, Dr. Peterson was part of a team known for its sophisticated genome sequencing techniques and innovative genomics projects. Now, to take his microbiome work to the next level, Dr. Peterson needs the people and the resources that can help him do it—and he finds all that at Sanford-Burnham.

“I’m a microbiologist, so I look at things from the bacterium’s point of view. But I also need to understand that in the context of what’s going on in the host. There are many immunologists, cancer biologists, and obesity experts here at Sanford-Burnham who are studying the host perspective.

I’ll be teaming up with many of them to complement what I do,” he says.

Dr. Peterson’s work is likely to impact our understanding of a number of human conditions, including body weight, heart health, and cancer.

Learn about some of the research projects Dr. Peterson is currently involved with on our blog, Beaker, at beaker.sanfordburnham.org. www.sanfordburnham.org | PORTAL

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Photo by Harbor Branch Oceanographic Institute

A submersible used to plumb the depths of the ocean for natural compounds that might become medicines.

Medical researchers will leave no stone unturned in their search for the next “treasure.”

One source Sanford-Burnham’s treasure hunters explore are the chemical compounds found in nature. According to the National Cancer Institute, more than half of the drugs approved to treat cancer today come from a natural product such as a microbe, plant, invertebrate, or a natural product prototype.

As we’ve increased our commitment to drug discovery, Sanford-Burnham scientists have joined the hunt for these natural compounds. The resources and expertise available in our Conrad Prebys Center for Chemical Genomics make it possible to screen thousands of these samples at a time. They painstakingly test millions of chemical compounds derived from natural products found in deep ocean waters or dense rain forests.

“At Sanford-Burnham, we sift through numerous plant, marine, and insect extracts, testing each to find those with the potential to fight human disease,” says Gregory Roth, Ph.D., associate professor and director of medicinal chemistry at Sanford-Burnham at Lake Nona. “We’re looking for a class of novel chemical compounds that will act on a biological target—for example, a natural product that is active against a specific protein that’s overproduced in tumor cells, where it helps keep the cancer cells alive and replicating.”

Dr. Roth has partnered with Amy Wright, Ph.D., of Harbor Branch Oceanographic Institute at Florida Atlantic University, to pursue natural marine products with medicinal potential. Together they explore products found in sea sponges for their

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potential to treat pancreatic and breast cancer.

Recently, Dr. Roth was invited to join a trip to Guyana in the spring of 2013 where he will experience the challenges of finding sources of new chemical compounds in a tropical rainforest.

The compounds Drs. Roth and Wright find will augment Sanford-Burnham’s growing collection. “We have been expanding our natural product and extract libraries over the past two years now, and many of the screens being run in the Prebys Center include one or more of these specialty collections,” says Christian Hassig, Ph.D., director for drug discovery.

Back in the lab, several Sanford-Burnham scientists are already putting natural compounds to the test. Anne Bang, Ph.D., director of cell-based disease modeling and screening, tested some of these samples and uncovered a novel natural product with interesting bioactivity in a cell-based assay of muscular dystrophy. In addition, Institute CEO John Reed, M.D., Ph.D., collaborated with Prebys Center staff to screen a large collection of extracts for compounds that induce cancer cell death. Stuart Lipton, M.D., Ph.D., director of the Del E. Webb Neuroscience, Aging and Stem Cell Research Center, identified a component of rosemary— carnosic acid—that fights off free radical damage in the brain and eye, protecting against certain degenerative diseases.

As promising compounds are found, our collaborative team further investigates and advances these natural products with the goal of developing them into the medicines of tomorrow—treatments and cures that start out hiding somewhere in nature.

Please Join Us in Congratulating Erkki Ruoslahti Thomson Reuters recently selected Erkki Ruoslahti, M.D., Ph.D., cancer researcher and distinguished professor at Sanford-Burnham, as one of its 2012 Citation Laureates. Citation Laureates are scientists that Thomson Reuters predicts will one day win the Nobel Prize.

Dr. Ruoslahti is among the 50 most-cited researchers of the last 20 years and an influential figure in cell biology and cancer research. His work has spawned many drug-discovery programs around the world—some of which have resulted in FDA-approved therapies. Learn more about him by visiting our website at sanfordburnham.org.

Thomson Reuters has made many correct Nobel Prize predictions, so keep an eye on Dr. Ruoslahti in the coming years!

Remembering Jan Tuttleman

Jan Tuttleman and her husband, Craig Lambert, in 2010

Jan Tuttleman, Ph.D., a staunch advocate for the life sciences community and, in particular, for young and aspiring scientists, passed away in October. SanfordBurnham’s faculty, staff, trustees, and supporters were deeply saddened to lose this vibrant woman.

Jan had served as a trustee of the Institute since 2007. She was also an Advisory Board Member to The Fishman Fund and a proponent of our Graduate School of Biomedical Sciences. Guy Salvesen, Ph.D., professor and dean of the graduate school,

said, “Jan had been with us since the beginning of the program, and always championed bettering the education of our students, which we all greatly appreciated. Everyone involved in the program will miss her dearly.”

During her tenure as a trustee, Jan provided experienced leadership and service on a variety of committees, from helping to shape the Institute’s approach to commercializing discoveries to fundraising. She was also a generous supporter and active participant in many of the Institute’s special events.

“Jan was not only a devoted trustee, she was also a good friend to many at the Institute,” said CEO John Reed, M.D., Ph.D.

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Recent Events RECENT EVENTS

Padres Game

On September 7, a group of valued donors gathered to enjoy the San Diego Padres game from the Owner’s Box. Longtime Institute supporter Jeanne Herberger threw out the first pitch (Above left). Above right: Thomas Sudberry, Jessie Knight, and Charles Wax.

Fishman Fund Awards

Five postdoctoral fellows received Fishman Fund Awards at an award ceremony and reception at Sanford-Burnham’s La Jolla location. The awards are given each year to help young researchers pursue their career goals. L to R: Drs. Jun Wang, Soda Diop, Tufan Aydogdu, Zhi-Gang She, and Mihee Kim.

Golf Outing

A group of friends and donors gathered for a day of golf at The Farms Golf Club in Rancho Santa Fe, Calif., in August. At dinner that evening, Sanford-Burnham CEO John Reed gave a brief update on the exciting research taking place at the Institute. Pictured here are Dr. Reed with Institute friends Harold Sadler and Kristopher Dickinson.

Thank You Research Seminar Series Event

On September 6, Sanford-Burnham at Lake Nona kicked off its research seminar series with a reception that featured speakers from the Institute and Florida Rep. Stephen Precourt. The event was sponsored by the Edyth Bush Charitable Foundation.

Pictured at right is event sponsor David Odahowski, president and CEO of the Edyth Bush Charitable Foundation.

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At Sanford-Burnham, we are grateful to all of the companies that support our endeavors — and never forget that those companies are made up of individuals. We wish to thank the people of Life Technologies, lead sponsor for our annual gala. Life Technologies has made a significant cumulative contribution to the Institute by underwriting the gala and other events. The charge was certainly led by Greg Lucier, Life Technologies CEO, who served on our Board of Trustees for six years, and as chair for two of those years. However, many people at the company, including Heather Virdo, helped make their support possible. We appreciate that these individuals believe in our work, believe in our events, and believe in a future where millions of lives are saved through medical breakthroughs.

Check our next issue of Portal for photos from the 2012 gala! www.sanfordburnham.org | PORTAL

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Upcoming UPCOMING EVENTS

Community Outreach Sanford-Burnham proudly participated, for the second time, in The Atlantic Meets the Pacific, an innovative forum presented by The Atlantic magazine and UC San Diego. We hosted tours of our La Jolla campus, where visitors asked informed questions and reacted with wonder while visiting our Stem Cell Research Center, Conrad Prebys Center for Chemical Genomics, and select laboratories.

Photo by Bob Ross.

The Atlantic Meets the Pacific

Rare Disease Day 2013: Calcification Disorders – from Hardened Arteries to Soft Bones

A rapt audience at The Atlantic Meets the Pacific listens to a panel on funding medical research

Bring It! for Medical Research

More than 100 health care leaders from across the country gathered for the Lake Nona Impact Forum, to exchange ideas on accelerating the impact of health innovation. Sanford-Burnham CEO, John Reed, M.D., Ph.D., moderated a panel that discussed the obstacles and opportunities in accelerating health innovation.

Pack your gear and get ready for a wild adventure because you’re going to Camp Bring It! Co-counselors Terry and Stath Karras want you to mark your calendars for April 25, 2013, for an unforgettable evening supporting medical research!

Rare Disease Research: Saving Lives Now and in the Future Dr. Reed (far right) moderates a panel at the Lake Nona Impact Forum

Come Inside and Discover More!

As a supporter, you are a part of our research success here at Sanford-Burnham. We want to invite you inside, behind the scenes at our La Jolla campus, to see where the discoveries are made. If you would like to arrange a tour, please contact Christopher Ayson at (858) 795-5238 or cayson@sanfordburnham.org.

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On Rare Disease Day, we will again host a symposium featuring updates on research into rare diseases, with an emphasis on disorders that affect calcification. Researchers from our Sanford Children’s Health Research Center, as well as guest speakers from around the world, will present exciting new findings and success stories.

Pictured here is Bertrand Might (in red), a little boy with a rare genetic disease that is currently being studied by Hudson Freeze, Ph.D., and his laboratory. With Bertrand are his parents, Matthew and Cristina, and little sister Victoria.

April 25

Lake Nona Impact Forum

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February 28

Rare diseases provide important clues in the quest for new treatments for all types of human illnesses. People—usually children—with rare genetic mutations allow researchers to observe the functions of the specific genes that are malfunctioning. This information can then become a sort of “Rosetta Stone” for understanding other, more prevalent, diseases.

For example, Sanford-Burnham’s José Luis Millán, Ph.D., has long studied an enzyme called alkaline phosphatase. Children who inherit a defect in the gene that encodes alkaline phosphatase are born with a rare disease called hypophosphatasia,

which makes their bones dangerously fragile.

Working with a pharmaceutical company, Dr. Millán helped develop a new hypophosphatasia therapy that is now showing great promise in clinical trials. Children with hypophosphatasia produce too little alkaline phosphatase. In contrast, too much of the enzyme can lead to atherosclerosis (hardening of the arteries). Not only has Dr. Millán helped save the lives of several children with a rare disease, he is now using that knowledge to develop a therapeutic drug that could benefit both children and adults affected with atherosclerosis, a much more common condition. www.sanfordburnham.org | PORTAL

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

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Sanford-Burnham Medical Research Institute

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Philanthropy Catalyzes Great Science Philanthropy dollars account for a critical 11% of SanfordBurnham’s annual budget. Your gifts support our scientists as they take their research to the stage where the NIH and other entities will invest grant money.

We couldn’t be successful without you!

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Revenue from Investments 1% Licensing 3% State Support 16%

Federal Grant Support 69%

Philanthropy 11%

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