Spring 2008

BURNHAM REPORT

VOLUME 5 | NUMBER 1

W W W. B U R N H A M . O R G

Focus on Pediatric Research

Giving Children the Gift of Good Health

From Research, The Power to Cure

In This Issue

PRESIDENT’S MESSAGE

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BURNHAM REPORT Volume 5, Number 1

FOCUS ON PEDIATRIC RESEARCH

Giving Children the Gift of Good Health

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Research Profiles

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PHILANTHROPY MATTERS

JOHN C. REED, M.D., PH.D.

President and CEO Professor and Donald Bren Presidential Chair KRISTIINA VUORI, M.D., PH.D.

Executive Vice President, Scientific Affairs

A Message from Blair Blum

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The Legacy Society: Effie Apelast-Weinberg

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KARIN EASTHAM

Executive Vice President and COO BLAIR BLUM

Senior Vice President, External Relations

BURNHAM IN THE COMMUNITY

EDGAR GILLENWATERS

A Toast to the Future Gala

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Team Burnham Update

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News from Florida

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Vice President, External Relations

Burnham Board of Trustees

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CHRIS LEE

Vice President, External Relations ELIZABETH GIANINI

Director, External Relations ANDREA MOSER

Vice President, Communications Please address inquiries to: communications@burnham.org ON THE COVER Standing: Burnham Board of Trustees Chair Malin Burnham, Burnham

BURNHAM INSTITUTE FOR MEDICAL RESEARCH, LA JOLLA 10901 NORTH TORREY PINES ROAD,

Trustee Doug Manchester, Betsy Manchester, Denny Sanford, Sanford

LA JOLLA, CA 92037

Health Executive Vice President Dave Link. Seated: Dr. John Reed,

(858) 646-3100

Sanford Health President and CEO Kelby K. Krabbenhoft.

BURNHAM INSTITUTE FOR MEDICAL RESEARCH AT LAKE NONA 8669 COMMODITY CIRCLE, 4TH FLOOR, ORLANDO, FL 32819 (407) 745-2000

www.burnham.org

President’s Message WITH 2007 GONE AND THE NEW YEAR OF 2008 before

us, it gives me great pleasure to report that the state of the Burnham Institute for Medical Research is not only strong—it is very strong!

Last year was an incredible year for the Institute. In addition to our scientific achievements, in 2007 we saw: Continued growth in grant revenue. At a time when other organizations have contracted due to declining NIH budgets, we have grown to become the fourth most highly funded private research institute among the approximately 100 such institutes.

Record growth in philanthropy. Our philanthropic support included $2.5 million to endow our first chair by Donald Bren, a $10 million endowment gift from Joe Lewis and the Tavistock Group, and an amazing $20 million gift from Denny Sanford through Sanford Health to fund pediatric research.

Launch of operations in Orlando. We opened our first laboratory facilities at our temporary location in Florida’s Blood Centers in Orlando. We broke ground on our state-of-art 175,000 square foot laboratory facility at Lake Nona. The project is moving right on schedule for on-time completion in spring 2009.

Record year for faculty recruiting. We added 13 new faculty investigators in 2007, including six talented young Assistant Professors.

Leadership. I am pleased to announce two faculty changes. Dr. Kristiina Vuori, Director of the Cancer Center, has been named the Executive Vice President for Scientific Affairs. In this added role, she will be a partner to our faculty and Center Directors, and our administrative team, in linking scientific objectives with operational capabilities and priorities. Dr. Robert Liddington has been promoted from Acting Director to Director of our Infectious and Inflammatory Disease Center.

The successes of 2007 place Burnham on a trajectory for even greater progress in 2008. They validate that our collaborative, entrepreneurial culture and commitment to team work are producing results.

To highlight the establishment of the new Sanford Children’s Health Research Center at our San Diego campus, this issue of the Burnham Report focuses on Burnham researchers who are working to solve the mysteries of both rare and common childhood diseases.

The Burnham Report would not be complete without stories about the inspiration and generosity of our extended Burnham family—our donors and supporters. From Team Burnham running marathons to raise funds for our medical research, to the elegant touch of our gala chairs organizing black-tie fundraisers, it is truly an honor to be associated with our passionate volunteers. These dedicated individuals are committed to making a difference in the fight against diseases.

Sincerely, JOHN C. REED, M.D., PH.D.

President and Chief Executive Officer Professor and Donald Bren Presidential Chair www.burnham.org 1

focus on pediatric research

Giving Children the Gift of Good Health

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The Sanford Children’s Health Research Center was established at Burnham’s San Diego campus by a $20 million gift from South Dakota philanthropist Denny Sanford through Sanford Health.

The gift serves as the foundation for collaboration between Sanford Health of Sioux Falls, South Dakota and the Burnham Institute for Medical Research.

The collaboration combines world-class scientific talent with state-of-the art technology to conquer childhood diseases. In addition to the new center in La Jolla, Sanford Health will develop a Children’s Health Research Center in Sioux Falls. The collaboration between the two locations will establish the basis of an integrated, world class, academic pediatric research network.

“I have the utmost confidence that this collaboration will promote solutions to some of the most troubling health issues that affect children,” said Denny Sanford when the gift was announced. “Nothing gives me greater pleasure than to encourage efforts and activities that

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I know will help children enjoy the gift of good health.”

As part of the collaboration, Burnham will advise Sanford Health in recruiting talented research scientists to work at Sanford Health in Sioux Falls. In addition, Burnham will dedicate nine senior faculty to be supported by approximately 40 scientists and related staff in approximately 25,000 square feet of research and support space in La Jolla, California.

Sanford Health serves an 80,000 square mile, four-state region including parts of South Dakota, Iowa, Minnesota and Nebraska.

Their comprehensive, integrated system includes more than 360 physicians in 115 clinics, 23 hospitals, 13 nursing homes, 17 assisted living facilities and congregate living locations, 27 home health services and 19 pharmacies. With approximately 12,000 employees, Sanford Health is the largest employer in the region. Its primary 500-bed nonprofit tertiary care hos-

pital serves an average of more than 30,000 inpatients annually. With more than a million outpatient visits each year, Sanford Health is the largest healthcare system between Mayo Clinic in Rochester, Minnesota and Denver, Colorado.

Its divisions include Sanford University of South Dakota Medical Center, Sanford Clinic, Sanford Health Network, Sanford Health Plan and Sanford Health Foundation. Originally known as Sioux Valley Health System, the organization changed its name to Sanford Health to recognize a transformational gift of $400 million made by Mr. Sanford in early 2007. The gift is the largest single donation ever made to a hospital or health system.

“The opportunity to collaborate with Sanford Health expands our ability to combine Burnham’s worldclass laboratory research with a powerful clinical partner dedicated to advancing medicine,” says John Reed, M.D., Ph.D., Burnham’s President & CEO, Professor and

Donald Bren Presidential Chair.

“Denny Sanford’s gift and his commitment to improving child health will enable us to consolidate and expand our present pediatric medical research activities and to speed our progress towards solving the unmet medical needs of children. We are honored for the opportunity to work with Mr. Sanford and the Sanford Health organization towards this noble goal.”

Important milestones are already being met by the new Sanford Children’s Health Research Center.

Dr. Fred Levine has been named director of the new center. In a few short months, the excitement of the gift announcement has been transformed into new project funding that advances ongoing pediatric disease research at Burnham, and sets the stage for adding new investigators working to solve the mysteries of childhood diseases and devising new therapeutic strategies for eventual cures. The Burnham Report will continue to provide updates on the progress of the Sanford Children’s Health Research Center.

“This collaboration will promote solutions to some of the most troubling health issues that affect children.”

—Denny Sanford

focus on pediatric research

news Drs. Fred Levine and Pamela Itkin-Ansari Type 1, or “juvenile,” diabetes is a disease in which a person’s immune system attacks insulinproducing beta cells in the pancreas. Most often it is diagnosed in children, and it continues to affect them into adulthood, with debilitating effects on the nervous system, kidneys, and cardiovascular system.

There is currently no cure for type 1 diabetes.

Patients who have it must live their lives in almost constant awareness of blood sugar levels, with regular injections of insulin to manage their body’s energy needs. But Dr. Fred Levine and Dr. Pamela Itkin-Ansari, who collaborate at the Burnham Institute, are working on several options for better treatment, and they are very close to testing these in human clinical trials.

THIS FOCUS ON CHILDREN’S HEALTH WILL ALLOW A FORMAL COLLABORATIVE STRUCTURE FOR OUR INVESTIGATORS WHO ARE ALREADY INVOLVED IN CHILDHOOD DISEASE RESEARCH.

Newly appointed Sanford Children’s Health Research Center Director Dr. Fred Levine.

Dr. Fred Levine with Dr. Pamela Itkin-Ansari.

Dr. Itkin-Ansari was the first speaker for the Burnham Research Briefing and Community Forum in Orlando. Her topic was “The Future of Cell Replacement Therapies for Diabetes.”

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One of their strategies involves understanding the development of beta cells in the pancreas.

“We’ve already shown that there is an endocrine progenitor, or stem cells in the pancreas that become beta cells,” explains Levine. “Now we’re trying to understand what genes are involved in that process of differentiation and what is required to maintain the beta cell function once they are up and running.”

Transplantation, where new beta cells from a human adult, an animal such as a pig, or an embryonic stem cell source are used, is one experimental treatment for type 1 diabetes patients.

Unfor-tunately, despite having these three sources, beta cells are difficult to acquire for the transplant. Also, once the cells are transplanted, patients must take immunosuppressant drugs— which carry a host of dangerous side effects— to prevent their bodies from attacking the new beta cells, just as they did when the disease began.

“But, if we can learn how to grow new beta cells, either by taking progenitors in a culture and then transplanting them into patients, or by giving someone a drug so that progenitors in their own pancreas are stimulated to become new beta cells, then we can solve this problem,” says Levine, who is also a clinical geneticist treating children with rare energy metabolism disorders.

In Itkin-Ansari’s lab, researchers are looking at a revolutionary technique called encapsulation, where transplanted cells are “housed” in a mesh envelope that protects them from the immune system so that patients who receive the transplant would not have to take immunosuppressants.

Furthermore, the envelope can be placed under the skin almost anywhere on the body. The technique has already worked in animal studies and clinical trials are around the corner.

Levine and Itkin-Ansari worked together at UC San Diego before coming to the Burnham Institute as adjunct faculty. “It was really serendipitous that we came here when we did,” ItkinAnsari explains. “The resources here at Burnham, particularly our state-of-the-art drug screening center, have really sped this research forward.

And with the addition of the new Sanford Children’s Health Research Center, we will be that much closer to the infrastructure needed for clinical trials that can bring new treatment options to patients.”

Dr. Lorenzo Puri In the late 1990s, Dr. Lorenzo Puri was treating patients at a hospital in Rome while completing his medical residency in internal medicine. He enjoyed the work, but at the end of the day he had lingering questions about the molecular and genetic causes of his patients’ diseases.

“I asked myself, would I rather apply something that someone else has discovered as a physician, or would I prefer to discover these things myself?” he explains. “That’s when research became a passion of mine, fueled by curiosity.”

Now at the Burnham Institute, his team is studying how muscle stem cells are guided, or misguided, in their growth and maturation. What they are learning about this process will someday help children and young adults who have muscular dystrophy, as well

Puri’s team is studying how muscle stem cells are guided, or misguided, in their growth and maturation. www.burnham.org 5

focus on pediatric research

as those who develop a muscle-cell based cancer called rhabdomyosarcoma.

In mice, for example, he found that deacetylase inhibitors, a class of drugs that are used to treat cancer, can cause muscle cells to grow bigger and stronger due to increased expression of follistatin protein. The mice that received the drug could move as easily as if they had no disease at all.

His work is also helping clinicians understand what may be the cause of rhabdomyosarcoma.

“During embryonic development, it appears that muscle stem cells may become misplaced in the body,” he explains of the tumors. “We speculate that these cells are triggered to become tumors by misreading the normal signals in this inappropriate

A COMMUNITY WORKING T O G E T H E R T O T R E AT CHILDHOOD DISEASE May 22, 2008 7:00 p.m.

Lawrence Family Jewish Community Center La Jolla, CA Free and open to the public Prominent local clinicians and researchers will discuss issues surrounding children’s health.

Presented by the Fishman Fund.

For more information contact Chelsea Jones at cjones@burnham.org or (858) 795-5239.

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setting, but our research will soon show if this is truly the case.”

Much of Puri’s research, which he also conducts at the Dulbecco Telethon Institute in Rome, is supported by muscular dystrophy charities.

Through his relationship with these groups and consultation with physicians, he still sees children who have the disease. “I think about these children as I’m doing my work,” he says, “and now that I’ve gotten to know some of them. It’s not just about satisfying my curiosity, it’s about not wanting to lose them.”

Dr. Rolf Bodmer Approximately eight out of every 1,000 babies that are born each year have heart defects. Usually these defects are minor and can be managed with routine checkups as the child grows, or in more serious cases, with early surgery. But the initial cause of the defects is usually unknown.

Dr. Rolf Bodmer and his staff at the Burnham Institute are working to change that. Already they’ve discovered a handful of genes that are directly responsible for proper heart formation and function, and their list is growing. But you will not easily find these genes in humans.

These are genes that Bodmer’s team has first found in fruit flies.

“At first glance, this might seem strange,” he says, “but the fruit fly is a very useful system and we’ve proven that there are sufficient genetic parallels between their bodies and ours, from the way that things are assembled in the embryo, to events that occur after birth.”

Bodmer began his career in Switzerland and San Francisco studying how genes are responsible

Bodmer and his staff discovered a handful of genes that are directly responsible for proper heart formation and function. for nervous system development in the fruit fly. By accident, he discovered a gene that was expressed exclusively in the flies’ hearts. Then he made a dramatic discovery. Flies with this gene mutated weakly, had irregular heartbeats and electrical activity, but flies with strong mutations had no heart at all.

He artfully named this gene tinman. Now, using weak tinman mutants as a bait, his lab “knocks out” additional sections of the fly genome, and if the corresponding flies have additional heart defects, then he narrows his search to genes in the segment that was deleted.

In addition to cardiac development, his lab uses this technique with fruit flies to study cardiac function and aging.

Bodmer’s research made headlines in 2004 when the Wall Street Journal published a story about an American family that suffered from a hereditary congenital heart defect, one that is caused by a gene mutation equivalent to Bodmer’s tinman gene. “We’ve found a number of genes since then that interact in heart development, and we’re actively testing these with clinical collabora-

tors to see if they are involved in human heart disease,” says Bodmer.

By understanding the earliest fundamental factors in heart development, he may soon provide the genetic map that clinicians need to detect heart defects earlier and provide more effective treatments for their patients.

Dr. Dorit Hanein On Dr. Dorit Hanein’s desk, a stack of black, threeinch square films show minute portions of a human cell from a series of angles, building a 3-D map of its inner structure. Using the data, she will work together with her collaborators to determine what is happening with the molecules that form the skeleton machinery of that cell as it creeps forward.

This truly is basic science. It involves using an electron microscope that fills an entire room to analyze objects that are ten thousand times smaller than the “o” on this page. And yet, Hanein’s research has tremendous implications for medicine. She is providing structural and mechanist background that will one

Hanein is working to determine what is happening with the molecules that form the skeleton machinery of that cell as it creeps forward. www.burnham.org 7

focus on pediatric research

day assist in answering questions about how cancer cells break away from the original tumor and spread to other parts of the body; how chronic inflammatory responses affect malignant processes; how people develop problems with bone density; and how it is that young athletes can suddenly die from a disease known as familial hypertrophic cardiomyopathy (FHC).

A striking, passionate person, Hanein talks with full-arm gestures and looks you straight in the eye, almost leaning forward, as she speaks.

“When I am tasked with something, even by myself, I want to do it to perfection,” she says.

Hanein studied chemical engineering as an undergraduate in Israel and worked in industry for a while to improve manufacturing practices.

For fun, she attended a summer course at the Weizmann Institute in Science, where an inspiring teacher introduced her to the tool of her craft, the electron microscope.

Now, Hanein’s lab is using this tool to answer questions about a molecule called Arp2/3, which promotes the formation of new actin filaments and serves as the engine that “pushes” the cell forward—much like straightening your elbow to push out your hand. She is also interested in determining how an assembly of molecules (vinculin, talin, and integrin) anchors the cell’s skeleton to its tracks, and thus halts cell movement.

“I would struggle with the responsibility of being a physician,” Hanein says. “But I really want to know everything there is to know about these molecules. And hopefully, this will allow others to develop a more targeted approach to medicine with fewer negative side effects.”

Freeze is studying congenital disorders or glycosylation (CDG)—a disease that prevents cells from attaching sugars to proteins, a biochemical process that has important consequences for nearly all of the body’s functions.

Dr. Hudson Freeze On the shelf next to Dr. Hudson Freeze’s desk is a photo of Rocket, a little boy with dark wavy hair, blue eyes, and a beatific smile. Sadly, he was born with a disease that prevents his cells from attaching sugars to proteins, a biochemical process that has important consequences for nearly all of his body functions.

“That photo was taken about a year ago, when Rocket was about 9 months old,” Freeze says.

“But he’s gotten worse since then. Now he has to be fed with a tube.”

Rocket’s illness is one of many that fall into a category known as congenital disorders of glycosylation (CDG), a focus of Freeze’s research. In his lab there are photos of other children who have CDG, some of whom have passed away, but others who are waiting for the answer to what is causing their specific form of CDG and how it might be cured.

Originally from Indiana, Freeze came to UC San Diego as a graduate student studying slime molds. As his interests began shifting toward human disease, he applied for a research grant to study enzymes in these molds that were thought to be similar in humans. During this time, a physician sent him a sample from a patient who had CDG, which at that time was still an unclassified illness.

“I looked at those human cells and saw that they had some of the same properties as my slime molds!” Freeze says. Now, his lab at Burnham is the center of research on CDG in the United States. Physicians who suspect that their ailing patients may have a form of CDG send genetic samples to his team for diagnosis. So far, they and others have found 30 genes that cause it, and every day they move closer to finding others.

Through a pharmaceutical collaborator, these genes will soon be assembled on a panel that will help screen patients for CGD more quickly.

His research has led to cures for some patients who have CGD, including a drink that contains mannose, a type of sugar, for those with one type of genetic defect. He has found other promising treatments for a related disorder, protein losing enteropa-

thy (PLE), which affects CDG patients and children born with heart defects that require a Fontan surgery. People with other diseases, including colitis, Crohn’s disease, and inflammation-induced cancer, could soon see experimental treatments that stem from this work.

“It’s been such a thrill for us to do this research,” says Freeze, “and we’ve had wonderful support from the families of some of these children— fundraisers, fashion shows and 10K runs, for example—to help further our work. Their perseverance is really a big part of our inspiration.”

José Luis Millán, Ph.D. José Luis Millán, Ph.D. has been a valued member of the Burnham family for more than 30 years. In fact, only Mrs. Lillian Fishman, the co-founder of the Institute, has been here longer.

Dr. Millán joined La Jolla Cancer Research Foundation, the predecessor of the Burnham Institute for Medical

Millán’s team developed a mouse model of a condition resembling rickets, which they used to demonstrate the first successful enzyme replacement therapy in mice to prevent hypophosphatasia. www.burnham.org 9

focus on pediatric research

Research, in 1977 as a graduate student trainee in clinical enzymology. After receiving his Ph.D. from University of Umeå, Sweden, he was again drawn to La Jolla Cancer Research Foundation to complete his postdoctoral training, during which he was awarded the first ever Fishman Award, an advisory board-reviewed grant from the Fishman Fund for Postdoctoral Career Development. Dr. Millán subsequently received progressive faculty appointments and has held the title of Professor since 1989.

One of Dr. Millán’s passions is investigating childhood skeletal and bone diseases. By understanding the mechanisms that control normal skeletal mineralization, Dr. Millán tries to explain abnormalities that lead to diseases such as hypophosphatasia (HPP)—a rare, heritable form of rickets, where the bones soften as a result of lack of vitamin D, calcium, or insufficient exposure to sunlight.

“While the biochemical mechanism that leads to skeletal and dental defects of HPP is now generally understood,” said Dr. Millán, “there is currently no established medical treatment.”

Recently, Millán’s team developed a mouse model of a condition resembling rickets, which they used to demonstrate the first successful

enzyme replacement therapy in mice to prevent hypophosphatasia, thereby laying the groundwork for future clinical trials for HPP patients.

Dr. Millán, along with other members of Burnham’s faculty who are involved in childhood disease research, is pleased that Burnham is now establishing a major center for childhood diseases.

This focus on children’s health will allow a formal collaborative structure for our investigators who are already involved in childhood disease research.

This new center will also garner much needed attention to children’s health-related disease research from our community supporters. The Fishman Fund and its group of advisors are paving the roadway in terms of fostering support for children’s health.

Burnham Scientist on NIH Pathway to Independence Lars Bode, Ph.D., received the prestigious NIH Career Development Award to study most common disorder in newborns.

Necrotizing enterocolitis (NEC) is the most frequent and life-threatening disease in premature infants. More than ten percent of premature infants with very-low-birth-weight develop NEC; one quarter of them dies. However, breast-fed infants are at a six-fold lower risk to develop NEC compared to formula-fed infants. How does mother’s milk protect the infant from this devastating disease? Dr. Lars Bode, Staff Scientist at the Burnham Institute, set out to answer this question. He now receives substantial support from the National Institute of Health (NIH).

“During my Ph.D. thesis work in Germany and London, we found the first evidence that

“We must invest in the future of our new scientists today if we expect to meet the nation’s health challenges of tomorrow.”

—NIH Director Elias A. Zerhouni, M.D. complex sugars, present at very high concentrations in human milk but not in formula, inhibit key events in NEC development.” explains Dr. Bode.

“We are now aimed at elucidating whether these complex milk sugars indeed lower the risk to develop NEC and, if so, which ones are most effective. If mom invests so much energy in making these complex sugars, there must be a good reason for it.”

NIH reviewers strongly endorsed Dr. Bode’s research, calling his scientific approach “very significant and innovative,” and awarding him with the prestigious Pathway to Independence Award, which will support his research with almost $1 million over the next five years. “We must invest in the future of our new scientists today if we expect to meet the nation’s health challenges of tomorrow,” said NIH Director Elias A. Zerhouni, M.D., when he first announced the program in 2006. “This award program is a major step toward fostering the early independence of new

investigators, a key to innovation and creativity.”

Dr. Bode will receive additional training in glycobiology and pediatric gastroenterology during the program. He will be supported by his postdoctoral mentor and glycobiology expert Hudson Freeze, Ph.D., at the Burnham Institute, as well as by gastroenterology specialists Drs. Kim Barrett and Jae Kim at the University of California San Diego and Dr. Henri Ford at the Keck School of Medicine, University of Southern California. Milady Ninonuevo, a chemist and mass spectrometry expert from UC Davis, who also worked on milk sugars during her Ph.D. thesis, will join Dr. Bode’s team at Burnham as a postdoctoral associate this spring.

“This award is a great honor and opportunity,” says Dr. Bode. “Almost one third of all infants are not or cannot be breast-fed and receive formula instead. Our goal is to one day provide these infants with the same benefits that breast-fed infants receive with their mother’s milk. The award is a fantastic boost for our research.”

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philanthropy matters

Dear Burnham Institute for Medical Research Friends WE HAVE HAD A FRUITFUL AND PRODUCTIVE YEAR in philanthropy, thanks to all of our great

supporters, friends and community partners. Our year had many highlights that you will learn about in this issue of the Burnham Report.

Last October, a fashion show featuring the designer clothing of Yigal Azrouel was co-chaired by Muffy Walker and Miriam Smotrich. A crowd of more than 150 people gathered in the beautiful home of Joyce and Craig Grosvenor to view the upcoming Yigal spring fashions. The event proceeds supported women’s cancer research at Burnham.

Our November Gala was created and co-chaired by well known author Jeanne Jones and Cathryn Ramirez, Regional Vice President, Tiffany & Co. This extraordinary evening, featuring a tasting menu and musical interludes by the San Diego Opera was held at The Grand Del Mar Resort. Betsy and Papa Doug Manchester were the Presenting Sponsors, Tiffany & Co. was the Design Sponsor and Invitrogen was the Title Sponsor. Table patrons, our sponsors, a successful auction and the generosity of the attendees netted just over $1 million for medical research. This is the first time we have achieved this goal. Thank you!

The Gala ended with the announcement of the Sanford Health gift to the Burnham creating the Sanford Children’s Health Research Center. Denny Sanford, who was introduced to the Institute through Papa Doug and Betsy Manchester, made the gift possible. He is an energetic man who is committing his resources to curing at least one childhood disease during his lifetime. We are excited about the possibilities to make a difference in children’s lives with this new collaboration.

As you will see in this issue, Team Burnham was successful again this year. We had runners in Phoenix and added Orlando, as a second city, where Team Burnham was running to cure disease. Many of our new friends and partners in Orlando joined the race: BE&K Construction, Florida’s Blood Centers and Disney to name a few. Dr. John Reed, our President & CEO, inspired the Orlando runners this year with a second place finish among our team members there.

Finally, as a result of your generosity and interest in our research, our donor base has increased by 23 percent from July through December, 2007. We are all grateful for your support. I hope you enjoy this Burnham Report and will choose to stay connected to us over the years; with your help, we will live up to our motto . . . from research, the power to cure.

BLAIR BLUM

Senior Vice President, External Relations bblum@burnham.org

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T H E L E G A C Y S O C I E T Y: P R O F I L E O F A D O N O R

Effie Apelast-Weinberg When she’s not singing opera, she’s talking.

Speech, no less than song, pours out of Effie Apelast-Weinberg like a passionate orator.

This late bloomer, who subscribes to the philosophy that It’s Never Too Late, made her singing debut at age 69 in New York City.

Effie started voice lessons at the age of 65 and after four years realized that a lyric soprano her age had no place to perform. Determined to achieve her goals, she increased the number of lessons. A year later, just before her seventieth birthday, Effie debuted across the street from Carnegie Hall in her first of many one-woman recitals entitled, “The Impossible Dream.”

Since that time, Effie has produced programs that blend arias with operetta and show tunes. She just completed a performance at Great Neck House in New York.

“I pushed myself to the limit this time,” says Effie.

“I sang opera in eight languages. I think I’m ready to take a break and get back to tennis.”

This 79 year old “Senior Diva” has a history of taking on challenges. At age 50, Effie became intrigued with alternative medicine and went back to Hunter College in NYC to get a Master’s Degree in Nutrition. Effie opened up a small private practice at the time and continues to spread the word today.

In the last five years, Effie’s passion for finding alternative therapies has been put to good use. “I had a stroke five years ago, which affected my voice,” says Effie. “My husband, Norbert, said I was singing Jeanette MacDonald songs on my deathbed in the hospital. My voice is back, but now it’s my turn to help Norbert through his lung cancer.”

Effie discovered the Burnham Institute for Medical Research while surfing the web in search of new therapies for her husband’s cancer. She called and was put in touch with Mrs. Lillian Fishman, co-founder of the Institute. Lillian, also trained in the Science of Nutrition, spoke with Effie about their shared interest in finding natural therapies to prevent and treat many diseases, including cancer. The relationship grew and Effie recently decided to support the future of the Institute through a bequest in her will.

A woman of action, Effie’s voice will long be heard.

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philanthropy matters BURNHAM IN THE COMMUNITY

GALA EVENT

A Toast to t A Toast to the Future was Burnham’s most elegant and successful gala. Gala co-chairs Jeanne Jones and Cathryn Ramirez wowed guests with their vision and direction for the evening. Tiffany & Co. designed and decorated the ballroom of The Grand Del Mar Resort in luxurious red, blue and gold hues, and the evening’s five course menu and the wonderful wine pairings were accompanied throughout the night with entertainment by the San Diego Opera.

As the dessert course was served, the energy of the room shifted attention from dinner conversation to the main stage and the excitement of the live auction. Due to the generosity of our guests and the range of interesting items up for bid, the gala netted just over $1 million to support discoveries at Burnham. The real show stopper came at the end of the evening when Burnham Board of Trustees Chairman Malin Burnham made a surprise

introduction of South Dakota philanthropist Denny Sanford and announced his $20 million dollar gift to Burnham through Sanford Health, creating the Sanford Children’s Health Research Center.

The gala would not have been possible without the support of our wonderful sponsors, Papa Doug and Betsy Manchester, Invitrogen and Tiffany & Co. Special thanks is owed to each of them for their generous contributions, commitment and support of medical research at Burnham.

S AV E T H E D AT E N OV E M B E R 1 5, 20 0 8 2008 Burnham Gala at The Grand Del Mar Tables will go on sale in the spring.

For more information, please contact Jocelyn Wyndham at 858-795-5216 or email jwyndham@burnham.org.

Clockwise from above: 1. Co-Chairs Cathryn Ramirez of Tiffany & Co. and Jeanne Jones, with Betsy Manchester; 2. Burnham Trustee Stuart Tanz with wife Karen; 3. Greg Lucier of Invitrogen with wife Marilena and Dr. Claude Benchimol. Invitrogen was the event’s Title Sponsor. Greg is Vice Chairman of Burnham’s Board of Trustees; 4. Burnham Trustee Howard Cohen with wife Toby; 5. Standing: Dr. Vincent Chen, Dr. Mark Mercola, Dr. Alexey Terskikh, Dr. Fred Levine, Dr. Robert Liddington, Dr. Evan Snyder; Sitting: Dr. Pamela Itkin-Ansari, Dr. Stuart Lipton, Dr. Kristiina Vuori

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the Future

photo and caption to come?

philanthropy matters TEAM BURNHAM

Members of Team Burnham for Medical Research proved for the second year in a row that there is nothing you can’t do if you put your mind to it. In July of 2007 members of Team Burnham began their training in preparation for one of the most challenging, inspirational and FUN experiences of their lives.

This year’s team of 51 participants completed an intense, yet entertaining program in preparation for this year’s marathon and half marathon races. While training, each member raised significant funds for the groundbreaking discoveries underway at Burnham.

To date, the team has raised approximately $125,000, receiving more than 900 donations from 32 states and 11 countries. These funds will be used to support programs across the entire Institute—including programs in the Cancer Center, Del E. Webb Center for Neuroscience, Aging and Stem Cell Research, Infectious and Inflammatory Disease Center, Burnham’s Stem Cell Research Program and our research efforts in Orlando.

This year’s runners had the unique opportunity to compete in either of two races, set for the weekend of January 12-13, in different cities, on opposite sides of Clockwise, from right: Orlando Full Marathon: Judy Wade; Orlando Full Marathon from left to right: Mari Enoksson, Cecile Loudet; Orlando Full Marathon from left to right: Julien Courreges, Maurizio Pellecchia

the country. Team Burnham runners were joined at the starting line by more than 35,000 racers at the Rock n’ Roll Marathon and Half Marathon in Arizona and more than 29,000 runners at the Walt Disney World Marathon and Half Marathon in Orlando. The races proved to be no match for Team Burnham runners. Every person who began their journey that weekend across 26.2 or 13.1 miles in support of medical research completed their mission and finished their respective races.

For more information or to join next year’s Team Burnham for Medical Research visit www.burnham.org, or contact Chris Lee at chrislee@burnham.org or (858) 795-5232.

N EWS F R O M F LO R I DA

DANIEL P. KELLY, M.D. NAMED SCIENTIFIC DIRECTOR OF BURNHAM FLORIDA Daniel P. Kelly, M.D. has been named as the first Scientific Director of Burnham at Lake Nona in Orlando, Florida. In that capacity he will guide the scientific direction of Burnham’s Orlando facility by forming several research programs related to diabetes, obesity, metabolism and heart disease.

Dr. Kelly will also be responsible for faculty recruitment for the Orlando site.

“I was attracted by the excitement of building a unique world-class research effort focused on the immense problem of diabetes and its complications,” said Dr. Kelly. “The power of interdisciplinary research can be harnessed for the first time within a single institute by bringing together scientists from many different fields. I look forward to assembling a team that capitalizes on the current strengths of the Burnham organization, while

adding talented new scientists that drive our research mission forward. Linking discovery science to health care delivery by working with our clinical partners in the context of the developing medical city will change the future of health care.”

Dr. Kelly will officially join Burnham on July 1, 2008. He is currently the Tobias and Hortense Lewin Professor at Washington University and Chief of the Cardiovascular Division in the Department of Medicine of Washington University School of Medicine in St. Louis, Missouri. He is also the

BURNHAM RESEARCH BRIEFING AND COMMUNITY FORUM June 4, 2008

Director of the Center for Cardiovascular Research at the University.

Dr. Kelly’s scientific interests include fundamental

Dr. Robert Liddington, Professor & Program Director

and translational research on diabetes and its cardio-

Infectious and Inflammatory Disease Center

vascular complications, especially diabetic heart dis-

Burnham Institute for Medical Research

ease. He is the author of more than 100 peer-reviewed

Orange County Agriculture Center

original research papers focusing on these areas.

6021 South Conway Road. Orlando, Fl 32812 6 p.m. in the auditorium For more information, contact Eshma Harry at 407-745-2061 or email eharry@burnham.org

“We are proud that Dr. Kelly is joining the Burnham team to lead expansion of our medical research programs in Florida, and to launch a new research center devoted to diabetes and obesity research,” said John C. Reed, M.D., Ph.D., President and CEO, Professor and Donald Bren Presidential Chair. “Our organization and the collaborators with whom we work will benefit enormously from Dr. Kelly’s superb scientific vision, impressive leadership qualities and strong track record of success in building world-class research programs.”

www.burnham.org 17

BURNHAM INSTITUTE FOR MEDICAL RESEARCH Officers Malin Burnham, Chairman

Alan A. Gleicher

Gregory T. Lucier, Vice Chairman

W.D. Grant

John C. Reed, M.D., Ph.D.,

David F. Hale

President and Chief Executive

Brent Jacobs

Officer, Professor and

Robert C. Kyle

Donald Bren Presidential Chair

Robert J. Lauer

Karin Eastham,

Sheila B. Lipinsky

Executive Vice President

Stuart Lipton, M.D., Ph.D.

and Chief Operating Officer

Gregory T. Lucier

BURNHAM INSTITUTE FOR MEDICAL RESEARCH 30TH ANNUAL SY M P OS I UM Obesity and Diabetes in the 21st Century: From Basic Research to Drug Discovery April 24, 2008

Douglas F. Manchester

Full day program

Executive Vice President

Robert A. Mandell

Hilton La Jolla Torrey Pines

for Scientific Affairs

Nico Nierenberg

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

Douglas H. Obenshain

Hosted by:

Chief Financial Officer

Mark A. Pulido

GEN-SHENG FENG, PH.D.

and Treasurer

John C. Reed, M.D., Ph.D.

Professor, Signal

Scott South

Transduction Program

Stuart Tanz

SEAN OLDHAM, PH.D.

Board of Trustees

Jan Tuttleman, Ph.D.

Assistant Professor,

Linden S. Blue

Andrew J. Viterbi, Ph.D.

Signal Transduction Program

Mary F. Bradley

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

Brigitte M. Bren

Barbara “Bobbi” Warren

Arthur Brody

Judy White

Terrence J. Bruggeman

Gayle E. Wilson

Malin Burnham

Diane Winokur

Symposium sponsored

Howard I. Cohen

Kenneth J. Woolcott

by: Merck; Applied Molecular

Nicole DeBerg, Vice President,

Margaret Dunbar, Secretary

Register online at: www.burnham.org/ AnnualSymposium2008

Evolution, a subsidiary of Eli

Karin Eastham Carl Eibl

Ex-Officio

Jeannie Fontana, M.D., Ph.D.

Ernest Beutler, M.D.

Lilly; Arena Pharmaceuticals

Nonprofit Organization U.S. Postage

PAID The Burnham Institute 10901 North Torrey Pines Road La Jolla, CA 92037 (858) 646-3100 www.burnham.org