Spring 2010

Introducing the

Volume 7 | Number 1

Spring 2010

Sanford|burnham

In T h i s Iss u e Volume 7 | Number 1

Spri ng 2010

Founders

Trustees, continued

William H. Fi sh man, Ph.D. Lillia n Fi sh man

Shehan Dissanayake, Ph.D. M. Wainwright Fishburn, Jr. Jeannie M. Fontana, M.D., Ph.D. David Hale Jeanne Herberger, Ph.D. Brent Jacobs James E. Jardon II (Florida) Robert J. Lauer Fred Levine, M.D., Ph.D. Sheila B. Lipinsky Papa Doug Manchester Robert A. Mandell (Florida) Douglas H. Obenshain Peter Preuss Stuart Tanz Jan Tuttleman, Ph.D., M.B.A. Andrew J. Viterbi, Ph.D. Kristiina Vuori, M.D., Ph.D. Bobbi Warren Allen R. Weiss (Florida) Gayle E. Wilson Diane Winokur Kenneth J. Woolcott

Honorary Trustees S anford | B urn h a m R e s e arc h

A Vote of Confidence

1

Joe Lew is C o nra d T. P rebys T. De nny Sa nfor d

About Denny Sanford

3

Trustees and Officers

Partners

4

G reg Lucier Chairman

News

7

A l an G leich er Vice Chairman Jo hn C. Ree d, M.D., Ph .D. President & Chief Executive Officer Professor and Donald Bren Presidential Chair

Ph i l a nth r o p y

Honoring Good Friends

11

Bring It! Again

12

Running for Discovery

12

Marg are t M. Du nbar Secretary

F i n a l th o u g hts

President’s Message

13

Partners in Science

14

O n T h e C over On January 26, Denny Sanford made a $50 million transformative gift to support the cutting-edge science at Burnham Institute for Medical Research, which has been renamed Sanford-Burnham Medical Research Institute.

Denny Sanford and Malin Burnham

G ary F. R ai sl, Ed .D. Executive Vice President Chief Administrative Officer Chief Financial Officer Treasurer

Denny Sanford and Malin Burnham share a vision of

Trustees

Ex-Officio

Brigitte Bren Arthur Brody Malin Burnham

Raymond L. White, Ph.D.

B l a i r B lu m Senior Vice President External Relations

Andr e a M o se r Vice President Communications

El i z a b eth Gi a ni ni Vice President External Relations

J o sh Ba x t Editor

Edg a r G i l l enwat e rs Vice President External Relations C hr is L e e Vice President External Relations Stephanie Boumediene, M.P.H. Vice President External Relations

Sanford Gift Takes Institute to Next Level

Chairman, Science Advisory Board

HA N NA H LOtt- S C H WART Z Contributor Gavin & Gavin Advertising Design M a r k Dast ru p Natha n Pe tt y Na di a Bo rows k i S c ott Photography Please address inquiries to: jbaxt@sanfordburnham.org

how science can improve human health. Together, they hope

www.sanfordburnham.org

their support will continue to advance medical research and

Sanford | Burnham Medical Research Institute

alleviate the great suffering caused by disease.

A Vote of Confidence

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

Denny Sanford and Malin Burnham watch the gift announcement video.

On January 26, the excitement at Burnham was everywhere. In La Jolla, television news trucks were double-parked; an intricate stage was set up in the Fishman Auditorium and overflow seating was arranged so that everyone could participate in the upcoming announcement.

At Lake Nona, a video

others, Senior Vice President

tant research and speed the

feed was bringing the news

of External Relations Blair

search for new treatments.

cross-country. Throughout the

Blum announced that South

The gift will also strengthen

Institute, people were eager to

Dakota philanthropist T. Denny

the Institute’s endowment.

learn what all these prepara-

Sanford was making a $50

tions meant. They did not have

million gift to rapidly expand

in our team and this organiza-

to wait long.

biomedical research at

tion, with a landmark $50

Burnham.

million gift, atop the $20

At a little after 10 a.m., surrounded by scientists,

“Your show of confidence

Sanford’s gift will expand

million that you previously

community leaders,

the Institute’s intellectual

directed to build children’s

California Governor Arnold

resources by supporting the

research programs at

Schwarzenegger, San Diego

recruitment and retention of

Burnham, is nothing short of

Mayor Jerry Sanders, Burnham

bright and creative scientists.

amazing,” said John C. Reed,

co-founder Lillian Fishman,

In addition, the gift will help

M.D., Ph.D., President and

Malin Burnham and many

the Institute carry out impor-

CEO, professor and Donald

Sanford | Burnham Medical Research Institute at Santa Barbara 2324 Life Sciences Building, University of California, Santa Barbara Santa Barbara, CA 93106 • 805.453.0259

www.sanfordburnham.org

Sanford|Burnham

1

S a n f o r d | b u r n h a m Ann o u nc e m e nt

S a n f o r d | b u r n h a m Ann o u nc e m e nt

opportunities for scientific research that will have an

Denny Sanford

About

impact on world health.”

Governor Schwarzenegger echoed Mayor Sanders’ sentiments and applauded

T. Denny Sanford, Chairman of the Board of First Premier Bank and Chairman of the Board of Premier Bankcard, Inc., was born in St. Paul, Minnesota, at the height of the Great Depression.

Sanford for his contributions to improving health.

“Today is a terrific example of the kind of public/private partnerships that are so important,” said Governor Schwarzenegger. “I know that everyone here will agree that this research is extremely Cameras ringed Fishman Auditorium while staff prepared for the announcement.

important because it will

Denny Sanford and Malin Burnham show off the new sign.

He started working at age 8, helping in his father’s

ease the suffering for cancer Bren Presidential Chair. “Your

said Mayor Sanders. “He gave

Regenerative Medicine. And

patients, diabetes patients,

the last year, in spite of

had the vision to see what it

clothing distribution company. As a teen, Sanford spent his

investment in this institu-

a $20 million gift to create

today, $50 million to accel-

Alzheimer’s patients and the

the recession, we gained

could do.”

summers selling clothing to retail stores. He attended the

list goes on and on.

2,000 jobs in biotechnology.

tion will catalyze innovative

the Sanford Children’s Health

erate the scientific research

medical research for decades

Research Center at Burnham.

here at Burnham. That’s

With that, Burnham called

“But also, California

Denny’s donation will create

Denny Sanford up on stage

hundreds of additional jobs.”

to join him. Together, they

to come, giving rise to break-

He gave a $30 million gift to

$100 million of support

biotech firms employ more

through discoveries and

build the research facility for

for new jobs and research

than 280,000 workers

helping to translate those

the Sanford Consortium for

infrastructure and many more

in high-paying jobs. Over

great discoveries into real progress for improving human health worldwide.”

H ono r ed Gu ests

B rothe r s Throughout the festivities,

degree in psychology.

Sanford was recruited by Armstrong Cork Company

announced that the Institute

to work in sales and marketing management. Later, he

had been renamed Sanford-

founded a company that promoted technical products

Burnham Medical Research

through architects and engineers.

it was clear that no one was

Institute, unveiled the new

having a better time than

logo and premiered the

Malin Burnham and Denny

renamed First Premier Bank. Under his leadership, First

Sanford-Burnham video.

Premier Bank has grown its assets to almost $1 billion.

Sanford. In the two plus years

As the event continued,

University of Minnesota, Twin Cities, and graduated with a

“I am truly honored to have

In 1986, he purchased United National Bank, which was

Another business, Premier Bankcard Inc., has become a

since they met, Burnham

Mayor Sanders and Governor

my name associated and right

and Sanford have developed

Schwarzenegger noted how

next to this guy right here,”

a bond of friendship and

Denny Sanford’s gifts to

said Denny Sanford, putting a

mutual respect.

charitable giving. By 2005, he ranked 14 on The Chronicle

hand on Burnham’s shoulder.

of Philanthropy list of America’s Most Generous Donors,

“This is an outstanding organi-

with donations that year alone of more than $70.5 million.

Burnham and the Sanford

“Denny has been telling

national leader in the credit card industry.

In 2001, Sanford established the Sanford Foundation for

Consortium for Regenerative

people that he and I are

Medicine are having a remark-

zation that has at its core the

brothers, and he even calls

In 2006, he was named to Business Week’s Top 50 most

able impact on science, as

development of the very best

me ‘bro’ from time to time,”

generous philanthropists in the United States.

well as the economy, in San

research, the hiring of the very

said Burnham. “So yes, we

Diego and beyond.

best people and the success

are brothers, and we are part-

based Sioux Valley Hospitals & Health System to create

that can come from this.”

Sanford Health. Later that year, he donated $20 million

“Over the past two years,

ners. When Denny and I first

In 2007, Sanford donated $400 million to South Dakota-

through Sanford Health to Burnham Institute for Medical

Mr. Sanford has established

started talking about what

an impressive legacy of philan-

To view the Sanford-Burnham

more he could do here at the

Research to create the Sanford Children’s Health Research

thropy on Torrey Pines Mesa,

announcement video, visit:

Burnham Institute, I really

Center. The following year, he donated $30 million to the

helping our life sciences

www.sanfordburnham.org/video.

didn’t know exactly where it

San Diego Consortium for Regenerative Medicine, which

community stay in the fore-

might go, but I knew that he

front of medical research,”

2

Sanford|Burnham www.sanfordburnham.org

was subsequently renamed the Sanford Consortium for Regenerative Medicine.

Malin Burnham and Governor Arnold Schwarzenegger

www.sanfordburnham.org

Sanford|Burnham

3

S a n f o r d | b u r n h a m R e s e a r ch

S a n f o r d | b u r n h a m R e s e a r ch

Partners

cable to a variety of solid

properties to consider. Does

proteins that bind to them.

Pasquale, Ph.D., has studied

tumors that over-express the

the molecule stay in the body

If successful, their work will

a family of receptors called

Eph receptor, including pros-

long enough to be effective?

create compounds that further

Eph. These receptors play a

tate, lung, breast and ovarian

Is it safe? Does it attach only

help Dr. Pasquale unravel

major role in early develop-

cancers.”

to the target protein or is it

the mysteries behind Eph/

binding to other proteins,

ephrin interactions. Also, since

which could lead to unwanted

protein/protein interactions

side effects?

are abundant in cells, finding

For many years, Elena

ment but are also implicated in the uncontrolled cell growth associated with cancer. Dr. Pasquale’s laboratory was among the first to identify several Eph receptors and their ligands (called ephrins), describe how they function and develop ways to control them.

“Because these receptors are so prevalent in cancer cells, they could be an excellent target for treatDrs. Nicholas Cosford and Elena Pasquale

ment,” says Dr. Pasquale.

“For example, we could use a

Denny Sanford and Malin Burnham are the latest in a long line of partnerships to advance the Institute’s important work, starting with co-founders Dr. William and Lillian Fishman.

aberrant proteins that may

research tools to learn how a

be too active or not active

protein’s function affects an

enough. Much of the work

organism or the progress of

being done at Sanford-

a disease. And sometimes, a

Burnham seeks to identify

small molecule will be devel-

these problem proteins and

oped into a new medicine.

With the creation of the

In fact, collaboration is one of Sanford-Burnham’s most cherished values and has led

These over-expressed receptors could become a doorway

thousands of small molecule compounds against biological material to find a handful of hits—molecules that can alter a protein’s function. But that’s only the beginning of the story. Medicinal chemists, like Nicholas Cosford, Ph.D., take those molecules and improve (optimize) them. Small molecules work by binding to a specific part of a protein, so making the bind tighter could improve the molecule’s effectiveness. There are other

Dr. Cosford optimizes a

them might open doors in

adding atoms to the original

other disease research.

molecule, a process that combines science and experi-

how to use small molecules

ence. Once a molecule has

to disrupt protein/protein

been altered, it must be

interactions, then we have the

retested against biological

opportunity to treat diseases in

material to determine if its

new ways,” says Dr. Cosford.

properties have improved.

This can be slow work, but

hope that at least one of the

Dr. Cosford is pioneering

compounds they are testing

a new technology, called

will have strong drug-like prop-

microfluidics, that accelerates

erties that would merit further

molecular redesign.

testing, possibly leading to

“We have compressed

process,” says

the road.

Making Cell s B eh ave

Pellecchia, Ph.D., to attach

Dr. Cosford.

The outside surfaces of

Eph ligands to anti-cancer

“What used to

drugs, like Taxol.

take a couple of

to bring in medication.”

Dr. Pasquale is working

days, we can now

“We believe this could

do in about 20 minutes. This is

“This technology is likely applicable to a variety

of solid tumors that over-express the Eph receptor, including prostate, lung, breast and ovarian cancers.”

engineers and others routinely cross disciplinary boundaries

a powerful way to accelerate drug discovery.”

Dr. Cosford

—Dr. Maurizio Pellecchia

to find solutions to pressing

and Dr. Pasquale

medical problems.

As Sanford-Burnham enters a new era of acceler-

Center), scientists were given

binding with other molecules

a new tool to find chemical

called ligands. When a ligand

be a very effective strategy,” says Dr. Pellecchia. “Our data

attaches, the receptor is acti-

strongly suggest that these

vated and sets off a cascade

drug conjugates effectively

ated research, one area

compounds (also called small

where this collaborative spirit

molecules) that could benefi-

will lead to new successes is

cially alter the behavior of a

of activity within the cell. This

deliver the chemotherapeutic

drug discovery. Quite often,

protein. Sometimes, these

is one of the fundamental

agent to Eph cancer cells.

diseases can be linked to

small molecules become

ways the body talks to cells.

This technology is likely appli-

4

Sanford|Burnham www.sanfordburnham.org

are collaborating to improve molecules that interfere with the interaction between Eph proteins Dr. Maurizio Pellecchia

and the ephrin

ical development, many of which went on to clinical trials.

Dr. Jackson recently joined Sanford-Burnham as Vice President for Drug Discovery and Development.

In this role, Dr. Jackson leads Sanford-Burnham’s

preclinical and clinical trials.

closely with Maurizio

Chemical Genomics (Prebys

Computer model of small molecule compound (top of image) binding to Eph protein.

Drs. Pasquale and Cosford

new cancer treatments down

receptors, which work by

pharmacologists, physicists,

“If we can understand

down to a single

cells contain proteins called

ratory. Biologists, chemists,

ways to obstruct (or enhance)

compound by strategically

Their efforts could result in

Conrad Prebys Center for

to many successes in the labo-

to deliver a therapeutic drug.

The Prebys Center screens

several steps

understand how they contribute to disease.

molecule that binds to them

B u i l d ing a B ett e r Mo l ec u l e

efforts to identify drug candidates, develop promising chemical compounds into new medicines and create partnerships for preclinical

B r i ng i ng Med i c in e s C los e r to Pat i ents

and clinical drug development.

Michael Jackson, Ph.D.,

In essence, his job is to take

knows drug discovery. Dr.

the basic science discoveries

Jackson spent 15 years with

at Sanford-Burnham and

Johnson & Johnson in a

help translate them into new

number of positions, including

medicines.

Senior Vice President of

The role that Dr. Jackson

Drug Discovery at Johnson

plays at Sanford-Burnham

& Johnson Pharmaceutical

is unique among non-profit

Research and Development

biomedical research institu-

(J&J PRD), where he estab-

tions. To streamline their

lished their state-of-the-art

business model, pharma-

drug discovery site in San

ceutical companies are

Diego. Under his leadership,

devoting fewer resources to

J&J PRD delivered numerous

basic biomedical research.

drug candidates into preclin-

Their efforts are now more focused on developing proven

www.sanfordburnham.org

Sanford|Burnham

5

S a n f o r d | b u r n h a m R e s e a r ch

S a n f o r d | b u r n h a m R e s e a r ch

to advance a project to a

studying apoptosis

tion of that disease in a dish.

this shift raises an important

Dr. Pasquale’s

point where licensing makes

(programmed cell

The Institute’s capabilities

question: Where will these

work, different

sense. But there are even

death) for years. In

in neuroscience, synaptic

companies find active, drug-

types of Eph recep-

more sophisticated options.

cancer research, the

damage, cell biology, embry-

like compounds if they don’t

tors are found on

“We are looking more

goal is to understand

onic and induced pluripotent

develop them in-house?

cells throughout

thematically at what we’re

why cancer cells don’t

stem cell biology and drug

compounds into drugs. But

Returning to

the body, so

doing in the laboratory,” says

die and design ways

discovery technology puts

companies downsize their own

understanding

Dr. Laikind. “The critical mass

to reverse that. With

Sanford-Burnham at the

research and development

the mechanisms

of talented scientists and

neurodegenerative

leading edge of research on

departments, they are looking

that influence

cutting-edge technology we

diseases, researchers

neurodegenerative disorders.

to outsource that work,” says

them can impact

bring to bear on a project,

and clinicians want

Dr. Jackson. “Ultimately, it will

other conditions

combined with our collab-

to keep cells alive.

collaborations with industry

be up to academic institutions

beyond cancer.

orative, multi-disciplinary

So learning the basic

will help us translate our

like Sanford-Burnham to pick

Recent collabora-

approach, can be very

mechanisms that

hard work into tomorrow’s

up some of that slack and

tions between

appealing to a drug company.”

determine whether a

bring new compounds to the

Dr. Pasquale and

drug pipeline.”

the Max Planck

“As pharmaceutical

Drs. Paul Laikind and Michael Jackson work closely to translate Sanford-Burnham advances into new medicines.

As an example, Dr. Laikind to follow up on this ancillary

has extensive experience

Institute have shown that Eph

in business development

corporate and non-profit

finding may well depend on

receptors play a key role in

whether the company even

and has cofounded three

research organizations

regulating glutamate levels

biotech companies—Gensia

are beginning to develop a

has a neurology division. Our

in the brain. Glutamate helps

academic freedom allows us

Pharmaceuticals, Viagene

complementary relationship:

relay electrical signals between

to pursue numerous research

and Metabasis Therapeutics.

academic research uncovers

neurons, but if not removed

avenues and then seek out

His goal is to find good

the root causes of disease

properly, it becomes toxic and

the appropriate company to

corporate homes for Sanford-

Dr. Jackson notes that

cell dies or survives

“Establishing long-term

cures,” says Dr. Laikind. “Our The high-throughput robots at the Prebys Center screen for chemical compounds that beneficially alter protein function.

job is to communicate the

cites Sanford-Burnham’s

can impact the study

approach to neurodegenerative

of numerous conditions.

induced pluripotent stem

tise to industry and create

disorders. Talented neurosci-

Sanford-Burnham’s world-class

cells (mature cells that are

collaborations that advance

entists collaborate closely

stem cell biologists also play

transformed into stem cells)

these discoveries as rapidly

with cancer researchers and

a role, devising new ways to

from patients with a specific

as possible.”

other scientists who have been

replace lost tissue or create

disease and study the evolu-

Institute’s far-reaching exper-

Burnham discoveries.

“The critical mass of talented scientists and

cutting-edge technology we bring to bear on a project, combined with our collaborative, multi-disciplinary approach, can be very appealing to a drug company.”

—Dr. Paul Laikind

“My job is to help SanfordBurnham achieve its mission of transforming cutting-edge science into important medical advances that benefit patients and society as a whole,” says Dr. Laikind.

Nobel Laureates

Anchor Lake Nona’s

Inaugural Symposium

Dr. Laikind’s Business and develops compounds

can cause epilepsy and other

Development and Intellectual

with therapeutic potential,

develop what we find. This

conditions.

should be a highly efficient

Property team are looking

model for drug discovery.”

for the best ways to protect,

and industry applies its vast

“Academic institutions

resources to optimize these

have a great advantage in that

drug candidates and shepherd

we can follow the research

them through clinical trials.

wherever it takes us,” says Dr.

One of the advantages of

Jackson. “It’s not uncommon

advance and commercialize

Ind ustry C oll aborations To bring new drugs closer

Sanford-Burnham technology.

Sometimes, the method can be as simple as a licensing

this model is that it can maxi-

that a scientist will be investi-

to market, Dr. Jackson is

agreement, in which a scien-

mize the serendipity that so

gating cancer biology and find a

working closely with Paul

tist has developed technology

often occurs when scientists

relationship to other conditions,

Laikind, Ph.D., Senior

that a company wishes to

are trying to answer a thorny

such as neurodegenerative

Vice President, Business

use. In other cases, busi-

question about biology.

diseases. In a corporate

Development and Chief

ness development might help

setting, the researcher’s ability

Business Officer. Dr. Laikind

secure corporate funding

6

Sanford|Burnham www.sanfordburnham.org

On February 12, Sanford-Burnham at Lake Nona hosted its inaugural scientific symposium, which attracted 200 scientists from across the country.

The symposium was described by one participant as the scientific equivalent of Woodstock, with a program that featured

during informal exchanges and in poster sessions, during which SanfordBurnham postdoctoral scientists presented their research.

Drs. Michael Brown, Tim Osborne, Robert Lefkowitz and Joseph Goldstein

“This symposium helps put Orlando on the map as a desti-

two Nobel laureates, Drs. Michael Brown and Joseph Goldstein, as

nation for medical research,” said John C. Reed, M.D., Ph.D.,

well as Drs. Robert Lefkowitz, Lewis Cantley, Cynthia Kenyon, Eric

Sanford-Burnham President and CEO, professor and Donald Bren

Olson and Ronald Evans.

Presidential Chair. “If we can continue to attract some of the

Disseminating scientific information is one of the most important

world’s greatest scientists to meetings such as this, Orlando will

activities researchers do after concluding their investigations. At the

become known world-wide as an area from which great research

symposium, ideas were exchanged at the formal presentations,

emanates.”

www.sanfordburnham.org

Sanford|Burnham

7

S a n f o r d | b u r n h a m N e ws

S a n f o r d | b u r n h a m N e ws

Delivering Medicine

Directly into a Tumor Drs. Kazuki Sugahara, Tambet Teesalu, and other researchers in the Ruoslahti laboratory of SanfordBurnham at the University of California, Santa Barbara, have identified a peptide (a piece of a protein) that recognizes and penetrates cancerous tumors but not normal tissues.

published

The iRGD

December 8

peptide incor-

in the journal

porates two

Cancer Cell.

elements: the

“This peptide has extraor-

Secrets of Immunologic Memory

RGD cell attach-

dinary tumor-penetrating

ment sequence

properties, and I hope that it

discovered by

will make possible substantial

Dr. Ruoslahti in

improvements in cancer treat-

the 1980s and

ment,” says Dr. Ruoslahti.

a tissue penetra-

“In our animal studies, the

tion element his

against diseases and, as memory cells, provide immunity

iRGD peptide has increased

laboratory identi-

to subsequent infections. Linda Bradley, Ph.D., Bas Baaten,

the efficacy of a number of

fied recently. The

Ph.D., and colleagues determined that without CD44, Th1 cells

anti-cancer drugs without

RGD element

increasing their side effects.

directs the

If these animal experiments

peptide to tumor

translate into human cancers,

vessels. An enzyme then

shown to deliver diagnostic

we would be able to treat

cuts the peptide, activating

particles and medicines into

cancer more effectively than

the penetration element,

the tumor. This new peptide,

before while greatly reducing

which takes the peptide out

called iRGD, could dramatically

the side effects the patient

of the tumor vessels and

enhance both cancer detection

would suffer.”

into the tumor cells. Previous

and treatment. The work was

peptides have been shown to

The peptide was also

Investigators in the Bradley laboratory have discovered a new way the cell surface protein CD44 helps Th1 cells (a type of white blood cell) develop immunologic memory.

Th1 cells are important components in the body’s defense

died off during their initial immune response and thus were Dr. Erkki Ruoslahti

recognize and bind to tumors but were unable to go beyond the tumor blood vessels. The researchers later attached

Drs. Bas Baaten and Linda Bradley

“In various infections and autoimmune conditions, Th1 cells

unable to generate immunologic memory. This is the first time

are often the bad guys,” says Dr. Bradley. “They can contribute

scientists have identified this unique CD44 function on Th1

to disease by overproducing cytokines (immune system

cells, making the protein a potential target to treat a variety of

signaling molecules) and are often responsible for disease

diseases. The study was published online on January 14 in the

pathology. Our findings reveal an opportunity to harness CD44

journal Immunity.

to control this pathogenesis.”

drugs to the iRGD peptide, which were also carried to the tumor cells.

New Form of Stem How

Flu Succeeds

Investigators in the Chanda laboratory, in collaboration with Mount Sinai School Drs. Sumit Chanda and Renate Koenig of Medicine, the Salk Institute and the Genomics Institute of the Novartis Research Foundation, have identified 295 human proteins and other cell factors that influenza A strains, including swine-origin H1N1, must harness to infect a cell.

“Because influenza mutates so readily, it has become a moving target for therapeutic intervention, making it difficult to treat circulating strains,” says Sumit Chanda, Ph.D. “As a result, there is now widespread resistance to two classes of antiviral drugs. However, by targeting more stable human host factors, we may be able to develop therapies that prevent or treat a variety of influenza A strains and are more likely to maintain their effectiveness.”

Influenza A virus contains only enough genetic information to produce 11 proteins and must co-opt host cellular machinery to complete its life cycle. Drs. Sumit Chanda, Renate Koenig and others used RNAi screening technology to selectively turn off more

The team also identified small molecule compounds that act

than 19,000 human genes to determine which human factors

on several of these factors and inhibit viral replication, pointing to

facilitate viral entry, uncoating, nuclear import, viral replication and

new ways to treat flu. These findings were published on December

other necessary functions.

21 in the journal Nature.

8

Sanford|Burnham www.sanfordburnham.org

differentiate into the specific type of neuron to provide this

Cell Communication Rescues

therapeutic effect.

Diseased Neurons

“We showed a while ago that stem cells may exert a therapeutic effect on damaged

cells, when in direct contact with diseased neurons, send Dr. Evan Y. Snyder signals through specialized An international team of channels that rescue the investigators, co-led by Evan neurons from death.

Y. Snyder, M.D., Ph.D., and including researchers from These direct cell-to-cell the Karolinska Institutet, connections may also play Beth Israel Deaconess, a role in normal developHarvard Medical School ment by creating the and the Universite Libre blueprint for more mature de Bruxelles, have demonelectrical connections strated in mouse models between neurons and other that transplanted stem

cells. The research was

or diseased host systems by

published in the journal

secreting therapeutic factors

Proceedings of the National

and ‘bathing’ the dying cells,”

Academy of Sciences.

says Dr. Snyder, director of the

While it was already

Stem Cell and Regenerative

known that stem cells will

Biology program. “However,

seek out diseased cells in

we did not know that stem

the brain, the team showed

cells can also exert their

that stem cells bring

action through direct cell-to-cell

diseased neurons back

contact. This may be a newly

from the brink via cross-talk

recognized way in which stem

through gap junctions, the

cells communicate with the

connections between cells

cells around them, not only

that allow signals to pass

under diseased conditions but

back and forth. Significantly,

during normal development.”

the stem cells do not need to

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Sanford|Burnham

9

S a n f o r d | b u r n h a m NEWS

S a n f o r d | b u r n h a m Ph i l a nt H r o p y

Natural Compound Inhibits Cancer Cell Migration Investigators in the Vuori laboratory have discovered that the natural compound sceptrin, which is found in marine sponges, reduces cancer cell motility (movement) and has very low toxicity in normal cells.

Metastasis, the process in which malignant cells migrate to other parts of the body, is one of the deadliest aspects of cancer, so restricting aberrant cell movement is an important step toward advancing treatments. The research was published in ACS Chemical Biology in collaboration with Phil S. Baran, Ph.D., of The Scripps Research Institute.

The team tested sceptrin in multiple tumor cell types, including cervical, breast and lung cancers, and found that sceptrin restricts

Honoring Good Friends

motility in all cell lines.

illness. Many of us have had

Mandell recently. “One of the

our own personal battles with

only ways to change medicine

illness. And it is through our

is through research. This

support that we give Burnham

institution has an incredible

scientists the tools to fight

reputation for collaborative

tion for cell motility. The

these diseases. That is why

research. The way they go

researchers also found

we have created this plaza, to

about their work, I don’t think

that sceptrin synthesized

encourage people to support

it gets any better than that.”

in the laboratory was just

this wonderful place.”

Further tests showed that the compound works by limiting the cells’ ability to contract, a critical func-

as effective as the natu-

are focal points for the

A B i - C oasta l M essag e

rally derived compound.

“Given the recently

Dr. Kristiina Vuori

Mandell Innovation Plaza

scientists. Designed to

sceptrin in multi-gram quantities by the Baran laboratory, sceptrin

was dedicated as part of the

resemble DNA helices, the

could prove to be an attractive lead molecule for further preclinical

ceremonies to open Sanford-

sites provide an artistic touch

testing and development for therapeutic purposes,” says Kristiina

Burnham’s facility at Lake

to each campus, as well as

Vuori, M.D., Ph.D. “It may also prove to be a useful research tool in

Nona. Like Mary

order to elucidate the mechanisms involved in cell motility.”

Bradley, Robert

supporter and Mary Bradley (1920-2010) touring Bradley Innovation Plaza with daughter Ellen Hunt.

Identify Mechanism for Frank-Ter Haar Syndrome Researchers

The Courtneidge group, together with SanfordBurnham researchers José Luis Millán, Ph.D., Pilar Ruiz-Lozano, Ph.D., and researchers at the University

FTHS suffer from

determined that the mice

skeletal, cardio-

without TKS4 showed virtu-

vascular and

ally identical traits to FTHS

eye abnormali-

patients, confirming the

ties and usually

results of the gene mapping.

Sanford-Burnham recently built donor recognition plazas at our La Jolla and Lake Nona campuses. The installations were designed to honor our friends and evoke the science they so generously support.

who make it possible. During the dedication, surrounded by family and friends, Bradley made an eloquent appeal for the science at Sanford-Burnham.

“Over the years, I have seen the kind of success this organization generates

In particular, the two sites

and have made my own

commemorate the commit-

investments to encourage

ment of trustees Robert

that success,” said Bradley.

die in infancy

“This research illus-

or early child-

trates the importance of

Mandell and Mary Bradley,

“Reena Horowitz and I teamed

collaboration in the scientific

whose gifts made these instal-

up to create the Fishman Fund

discovery process,” says Dr.

lations possible.

to honor Burnham’s founders

Bokhoven, Ph.D., at Nijmegen, with assistance from clinical colleagues in several countries, studied 12 families who had been affected by FTHS. The team mapped the condition in seven of these families to a mutated gene that normally

10

of California, San Diego,

Children with

hood. Hans von

Sanford|Burnham www.sanfordburnham.org

and the innovation of our

achieved synthesis of

been a great

An international team of investigators from the Courtneidge laboratory at Sanford-Burnham, Nijmegen Centre for Molecular Life Sciences and other organizations have discovered that TKS4, a protein implicated in cancer metastasis, also plays a significant role in Frank-Ter Haar syndrome (FTHS), a rare, fatal disorder.

The research was published on February 12 in the American Journal of Human Genetics.

generosity of our donors

A few months earlier, the

Mandell has

Both recognition sites

Dr. Sara A. Courtneidge

contains the code to create the TKS4 protein. Dr. von Bokhoven soon discovered

Courtneidge, “and how the

Sadly, Mary Bradley died

hard to advance the science.

As Chairman of Meritage Homes,

he knows what it takes to

a reminder of the important

build a community and make a

work being done by the

difference in people’s lives.

surrounding laboratories.

“I support SanfordTo honor Mary Bradley with

Burnham because I believe it’s something that will benefit

a contribution to the Fishman

my children and many others

Fund, contact Jane Langer at

in a significant way,” said

858-795-5288.

Dr. William and Lillian Fishman

study of one disease—in

of natural causes a few days

and support postdoctoral

this case cancer—can have

after the Bradley Innovation

fellows. It’s their dedication,

that Sara A. Courtneidge,

a profound impact on the

Plaza was dedicated on

and that of their primary inves-

Ph.D., was also studying

understanding of another. In

January 16. Bradley was a

tigators, that will make cures

TKS4—as it related to cancer

the future, we hope to use

smart, dedicated trustee and

a reality.

metastasis and had created

our mouse model to study the

a passionate advocate for

disease in more depth.”

science and the researchers

a mouse lacking the protein.

has worked

“I support Sanford-Burnham

because I believe it’s something that will benefit my children and many others in a significant way.”

—Robert Mandell

“We all have friends and family who have succumbed to

Mandell Innovation Plaza, Sanford-Burnham at Lake Nona

www.sanfordburnham.org

Sanford|Burnham

11

S a n f o r d | b u r n h a m Ph i l a nth r o p y

S a n f o r d | b u r n h a m P r e s i d e nt ’ s m e ss a g e

Join co-chairs Stath and Terry Karras and lead sponsor Life Technologies on April 29 for the most hilarious and over-the-top fundraising event ever. This is your opportunity to meet, greet, challenge and connect with some of San Diego’s most influential people while eating, drinking and singing your way to the winner’s circle—all while supporting cutting-edge stem cell research.

Bring It! Again

Last year, more than 300 people helped raise nearly $170,000 to support the HeadNorth Chronic Spinal Cord Injury

What famous actor founded the La Jolla Playhouse? What is the leading cause of spinal cord injury? Where do you end up if you take 15 South to 52 West to 5 South, exit 10th Street and drive until it dead ends? Why are we asking?

Because you’re the next contestant on Bring It!—the game show-style fundraiser where everyone plays and the buzz goes on for days. With the resounding success of last year’s event, Sanford-

Project at Sanford-Burnham, and this year promises to be even

Accepting the Challenge Denny Sanford’s recent donation of $50 million to support our work has been inspirational to many of us at Sanford-Burnham Medical Research Institute. This gift will provide much-needed resources to accelerate our science as we seek solutions to some of the world’s most difficult medical problems. We thank Denny for his generosity, his vision and his faith in our research.

bigger and better.

Thursday, April 29, 2010, 6 to 9 p.m.

Mission Tower Ballroom at the Del Mar Fairgrounds For more information, contact Chelsea Jones at 858-795-5239 or at cjones@sanfordburnham.org or visit www.burnhamevents. org/bringit.

Since Denny made his first gift to us to create the Sanford Children’s Health Research Center a little more than two years ago, he has impressed many of our scientists, trustees and staff with his indefatigable drive to improve human health and his insatiable thirst for knowledge about medicine and medical research. Though a businessman by profession, Denny has been known to attend our scientific symposia and sit through hours of complex scientific presentations. He has taken his growing scientific knowledge and translated it into

Burnham and HeadNorth Foundation invite you to Bring It! again.

John C. Reed, M.D., Ph.D. President and CEO Professor and Donald Bren Presidential Chair

goals that inspire scientists and physicians. He is a man of great ambition—not ambition directed towards serving self, but ambition to serve humanity through his bold leadership and support of transformative biomedical research. He understands the special role that organizations like Sanford-Burnham play as catalysts for the innovations that produce medical breakthroughs.

Denny Sanford’s support is also a tremendous vote of confidence in our science and our

Running for Discovery

with the endurance required to

scientists. It is their untiring dedication to solving medical problems that has made Sanford-

complete a marathon.”

Burnham the top-ranked organization worldwide, in the fields of biology and biochemistry, over

Congratulations to all team members on another successful run and thank you for your continued dedication

Though the Florida weather was uncommonly frigid, with temperatures in the 20s, the intrepid Team Burnham could not be stopped from completing their appointed races.

to first-class discovery and research.

Also many thanks to the Team Burnham stalwarts (Front) Jeff Decker, Dawn West (Back) Katie McKeever, John Gabrovic, Elizabeth Gianini, Bea Kochheiser, Andy Swart, Peter Lawrie and Justin Simmons.

contributed to helping Team

“I run for discovery because

the nation in our ability to create new intellectual property through patent production.

Though we experience numerous successes, large and small, in our laboratories each day, the medical problems that we face are both large and complex. This gift is indeed a validation of the work that we do. But it is also a challenge to set our sights even higher. For all of us—scientists, trustees, staff and supporters—this is a time to

BE & K Construction, Harmoni Market, CORT Furniture Rental, Florida’s Blood Centers,

$140,000 in its fourth year

find cures for not only cancer

Drs. John Reed, Dan Kelly,

to support Sanford-Burnham

but a multitude of today’s most

Phil Wood and many Sanford-

research in cancer, neurode-

prevalent killer diseases,” said

Burnham staff, braved the cold

generation, diabetes, heart

Peter Lawrie, long-time Team

weather to run the Walt Disney

disease and infectious,

Burnham member and cancer

World half and full marathons

inflammatory and childhood

survivor. “The scientists’ will to

Team Burnham, visit www.

on January 9 and 10. Each

diseases.

succeed has much in common

teamburnham.org.

Sanford|Burnham www.sanfordburnham.org

it is their ingenuity that has made Sanford-Burnham one of the most efficient organizations in

including Baker Hostetler,

Burnham researchers fight to

12

has allowed us to achieve eight consecutive years of double-digit growth in grant revenue. And

Team Burnham sponsors,

Burnham raise more than In all, 60 runners, including

the past decade for the impact of our research publications. It is their resourcefulness that

Orlando Health, Blue Cross

rededicate ourselves to the medical research mission that is embodied in our slogan: From research, the power to cure.

On behalf of everyone at Sanford-Burnham, we are honored that Denny has chosen our

Blue Shield of Florida, Berman

organization to be the beneficiary of such incredibly generous support. We applaud his lead-

Properties, Fleet Feet Sports

ership in supporting outstanding science.

and Brooks.

For more information about

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Sanford|Burnham

13

Nonprofit Organization U.S. Postage

PAID

10901 North Torrey Pines Road

The Burnham Institute

La Jolla, CA 92037

S a n f o r d | b u r n h a m Ph i l a nth r o p y

Partners

in Science: Alex

Martins and Dr. Dan Kelly

As Chief Operating Officer of the NBA’s Orlando Magic—a strong supporter of Sanford-Burnham—Alex Martins can give you chapter and verse on the value of teamwork. He gets no arguments from Dr. Dan Kelly, Sanford-Burnham at Lake Nona’s Scientific Director, who is currently recruiting a

“Sanford-Burnham’s research is invaluable—not just to Central Florida but to the future of human health,” says Martins. “By creating jobs and pioneering disease research, Sanford-Burnham is a win-win for our economy. Sanford-Burnham Medical Research

Institute is an investment in our future.”

premier scientific team to advance our understanding of diabetes and obesity and find new approaches to treating these and other conditions.

Together, the Orlando Magic and SanfordBurnham have teamed up to advance science and encourage economic growth.

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