summer 2009
DESCRIPTION
Volume 6 | Number 2TRANSCRIPT
VOLUME 6 NUMBER 2 | SUMMER 2009
From Research, The Power to Cure
InsIde >BURNhaM NEwS >FLORIDa UPDaTES >PhIL aNThROPy
Meeting The Stem Cell Challenge
B U r n h a m R E P O R T
I N T h I S I S S U E
B U R N h a M R E S E a R c h
Meeting the Stem Cell Challenge 1
What is a Stem Cell? 3
The Stem Cell Community 5
B U R N h a M N E w S
Science News 6
Florida News 8
P h I L a N T h R O P y
Updates 10
Bring It! 11
A Tribute to Malin Burnham 12
a R O U N D B U R N h a M
Fulfilling the Promise of Stem Cells: President’s Message 13
Partners in Science 14
BL aIR BLUM Senior Vice President External Relations
ELIzaBETh GIaNINI Vice President External Relations
EDGaR GILLENwaTERS Vice President External Relations
chRIS LEE Vice President External Relations
aNDREa MOSER Vice President Communications
O N T h E c O V E R
Vincent Chen, M.D.,
Ph.D., and Mark Mercola,
Ph.D., investigate stem
cells for new ways to treat
heart disease—but come at
the problem from different
angles. Dr. Chen works
with embryonic stem
cells to create pacemaker
cells and hopes to make
a biological pacemaker. Dr. Mercola wants his stem cells to
become cardiomyocytes or beating heart cells. Their collabora-
tion is bearing fruit, as both cell types come from the same
progenitors and failure in one lab can lead to success in the
other.
JOSh BaxT Editor, Burnham Report
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Please address inquiries to: [email protected]
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Founders
wILLIaM h. F IShMaN, Ph.D. L ILLIaN FIShMaN
honorary Trustees
JOE LEwIS cONRaD T. PREByS T. DENNy SaNFORD
Trustees and Officers
MaLIN BURNhaM Chairman
JOhN c. REED, M.D. , Ph.D. President & Chief Executive Officer Professor and Donald Bren Presidential Chair
GaRy F. RaISL, ED.D. Chief Administrative Officer Treasurer
MaRGaRET M. DUNBaR Secretary
Trustees
Linden S. BlueMary F. BradleyBrigitte M. BrenArthur BrodyHoward I. CohenShehan DissanayakeM. Wainwright Fishburn, Jr.
Trustees, continued
Jeannie M. Fontana, M.D., Ph.D.Alan A. GleicherW.D. GrantDavid F. HaleJeanne Herberger, Ph.D.Brent JacobsJames E. Jardon IIDaniel P. Kelly, M.D.Robert J. LauerSheila B. LipinskyGregory T. LucierPapa Doug ManchesterRobert A. MandellNicolas C. NierenbergDouglas H. ObenshainPeter PreussStuart TanzJan Tuttleman, Ph.D., MBAAndrew J. Viterbi, Ph.D.Kristiina Vuori, M.D., Ph.D.Bobbi WarrenAllen R. WeissJudy WhiteGayle E. WilsonDiane WinokurKenneth J. Woolcott
Ex-Officio
Raymond L. White, Ph.D.
B U r n h a m S T E M c E L L R E S E a R c h
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A few months ago my uncle was diagnosed with ALS (amyotrophic lateral sclerosis). It started with some speech problems but since then his condition is constantly worsening. It is very painful for me to know that he is in such a difficult position and that there is no cure for it. However, with the latest stem cell research, there might be some hope. He is very willing to participate in any kind of experiments. So my question is - is there any way that you can take him into your research program and see if there is any potential to cure ALS or at least to improve his condi-tion? If not - can you recommend any programs? Any help or suggestion would be very much appreciated.
— Bellevue, Washington
Meeting theStem Cell Challenge
Smooth muscles cells derived from embryonic stem cells
Burnham scientists
frequently receive letters like
this. They remind everyone at
the Institute how important
the research is, as well as how
much work needs to be done.
In recent years, there has been
intense focus on embryonic
stem cells because they can
form more than 200 different
tissue types. This flexibility,
or plasticity, could make them
ideal to treat diabetes, neuro-
degenerative diseases, heart
problems, cancer and many
other conditions. But where
does the promise of stem cell
therapies meet the reality? And
what will those therapies ulti-
mately look like?
Back TO BaSIcS
While stem cells (See box
on page 3) offer great potential
as future therapies, they are not
always obedient. Sometimes
they differentiate into other cell
types when researchers want
them to remain stem cells—or
vice versa. They can become
glial cells (non-neuronal brain
cells) when the goal is to create
neurons. They can form tumors,
called teratomas. To solve these
problems, researchers must deci-
pher the signaling mechanisms
that direct stem cells towards a
specific fate. By understanding
the developmental biology, scien-
tists can potentially replicate it
and assert control over stem cells.
Mark Mercola, Ph.D.,
associate director of the Del
E. Webb Neuroscience, Aging
and Stem Cell Research
Center, has discovered signals
that make embryonic stem
cells differentiate into cardio-
myocytes (beating heart cells),
which could be used to repair
damaged heart muscle.
Early on, Dr. Mercola and
other stem cell researchers were
frustrated by their inability to
direct stem cells to become
cardiomyocytes, or other cell
types, any more efficiently than
they would without human
intervention.
“We just didn’t know enough
about embryology,” says Dr.
Mercola. “We needed to under-
stand where the heart actually
comes from and what natural
signals in the early embryo
stimulate undifferentiated cells
to form a heart.” Studying how
embryos make hearts was a
good strategy. Dr. Mercola and
others succeeded in unraveling
many of the signals that tell
cells to form heart tissue.
Dr. Mercola then returned to
embryonic stem cells to find
the right chemical compounds,
proteins or microRNAs (ribonu-
cleic acids) to take pluripotent
stem cells through the multiple
stages they must travel to
become cardiomyocytes.
“Now we know quite a
bit about the early steps of
stem cell differentiation from
embryology,” says Dr. Mercola.
“But the major prize is to find
small molecules that influence
the latter steps. With those,
we could potentially create
drugs that help damaged hearts
rebuild themselves.”
To discover these molecules,
his laboratory uses high-
throughput screening in the
Conrad Prebys Center for
Chemical Genomics.
“We already have several
promising new compounds,
as well as a couple of existing
drugs, that are candidates to
stimulate a patient’s own heart
to regenerate cardiomyocytes,”
says Dr. Mercola.
BUILDING a BIOLOGIcaL PacEMakER
“Everything Mark Mercola
wants to do, I want to do in
mirror image,” says Vincent
Chen, M.D., Ph.D., who works
closely with Dr. Mercola.
Dr. Chen’s goal is to create
cardiac pacemaker cells to
treat arrhythmias and other
dangerous heart rhythm disor-
ders. The research being done
by each laboratory informs the
other’s work, as both cardiomyo-
cytes and pacemaker cells come
from the same precursor cells
but require different signals to
become the two types of heart
tissue. A practicing electro-
physiologist, Dr. Chen routinely
implants electronic pacemakers
and recognizes how that tech-
nology has evolved over the past
35 years.
“Current pacemakers have
very sophisticated algorithms
to make them pace the heart
appropriately and make them
failsafe,” says Dr. Chen. “They
need to respond to exercise,
and they need to recognize
when you are trying to sleep. If
we want to create a biological
pacemaker, we need to match
or exceed the standard of elec-
tronic pacemakers.”
The cellular pacemakers
Dr. Chen envisions would
fully integrate into the heart.
In other words, they would
respond to the nervous system
like the original cells they
must replace. Using human
embryonic stem cells, he has
developed a system to monitor
the formation of pacemaker
cells — a critical step towards
discovering molecules to build a
biological pacemaker.
B U r n h a m S T E M c E L L R E S E a R c h
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Theresearchbeingdonebyeachlaboratory
informs the other’s work,asboth
cardiomyocytesandpacemakercellscomefromthe
sameprecursor cells
Brandon Nelson, manager of Burnham’s Stem Cell Core, removes cells from cryogenic storage.
The core provides expertise to grow, differentiate and analyze embryonic stem cells.
MOTIVaTING ENDOGENOUS cELLS
A major goal of Burnham
researchers is to develop drugs
that can coax endogenous
somatic stem cells (progenitor
cells within our bodies) to
rebuild damaged tissue. In
collaboration with other labs,
Pilar Ruiz-Lozano, Ph.D.,
is working to pinpoint the
heart cells that cause normal
regeneration.
“It appears that endogenous
stem or progenitor cells can
heal very small traumas,” says
Dr. Ruiz-Lozano. “In nature, if
it happens at all, then it should
be possible to stimulate those
natural abilities to do more.
We need to understand how
various cells contribute to repair
processes to develop therapies.”
The Ruiz-Lozano lab has
discovered important cells in the
epicardium, the protective layer
surrounding the heart. These
epicardial cells provide signals
that minimize the impact of
heart disease on muscle cells.
With Dr. Mercola, Dr. Ruiz-
Lozano is working to discover
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Stem cells are cells that
can both replicate themselves
and differentiate into other
types of cells. In a developing
embryo, pluripotent stem
cells, which can differentiate
into all cell types, are told
through complex signaling to
differentiate into other cells.
Embryonic stem cells go
through several stages before
becoming functional neurons,
cardiomyocytes (beating heart
cells), pancreatic beta cells or
other types of tissue.
Once an embryonic stem
cell starts to differentiate,
it becomes multipotent and
can become many cell types.
These cells are moving
towards a cellular identity
but still have the flexibility
to become different types of
tissue. In other words, the cell
knows its address and will
migrate to the heart, liver,
brain or other part of the body
and differentiate into more
specific cells types. Certain
tissues in adults have their
own multipotent stem cells,
often called somatic stem cells
or “adult stem cells.” However,
the latter term is somewhat
misleading as these cells are
also found in embryos and
children.
Perhaps the most
intriguing types of stem
cells are induced pluripo-
tent stem (iPS) cells. These
cells are created from fully
differentiated cells, often
skin cells, which have been
reprogrammed to become
pluripotent stem cells. Like
their embryonic cousins,
iPS cells can form all cell
types. This approach offers a
number of benefits. For one,
iPS cells may be ideal for
transplantation, as they could
be taken from a patient and
would not face rejection from
the patient’s immune system.
Also, iPS cells could be used
to study diseases on a cellular
level.
“These iPS cells will give
us the ability to study the
molecular underpinnings of
disease,” says Stuart Lipton,
M.D., Ph.D., director, Del E.
Webb Neuroscience, Aging
and Stem Cell Research
Center. “We can set up
models for Parkinson’s and
figure out what is going wrong
with those cells.”
Currently, iPS cells have
their drawbacks. Some of the
methods used to create them
make them unsuitable for
clinical use. They replicate
slowly, making them difficult
to study. Also, scientists don’t
know if iPS cells are identical
to embryonic stem cells. More
study is needed, but break-
throughs are being announced
with great frequency.
“The technology for iPS
cells is changing every day,”
says Dr. Lipton. “They have
great potential, but we need to
learn whether they are iden-
tical to embryonic stem cells,
and we just don’t know yet.”
What is a Stem Cell?
Pilar Ruiz-Lozano, Ph.D.
synthetic compounds that can
enhance or mimic the epicardi-
um’s regenerative effects.
Epicardial cells can also
be isolated from patients
and expanded in culture or
chemically modified and used
for transplantation. “These
cells would be ideal, as they
would come from the patient
and would not cause an
immune response,” says Dr.
Ruiz-Lozano.
Lorenzo Puri, M.D., Ph.D.,
is doing similar work with
skeletal muscle progenitor cells.
“Muscle cells continually turn
over,” says Dr. Puri. “When you
go to the gym for strenuous
exercise, the body perceives this
as damage and the muscle stem
cells work to repair that damage.”
Controlling muscle stem
cells could have important
clinical implications. For
example, muscular dystrophy
(MD) causes debilitating
muscle weakness and eventu-
ally death. Compounds that
encourage muscle progenitors
to form new muscle could be
the next generation of treat-
ments for muscular dystrophy
and other muscle disorders.
But it’s important to know
when intervention can be most
effective.
“We need to understand
whether there is a clock,” says
Dr. Puri. “For a boy with MD,
18 might be too late. But at 3,
we might be able to reset the
cells.” His research has led to
encouraging pre-clinical drugs
that enhance regeneration by
modulating native stem cells in
muscle.
ThE MySTERy OF caNcER STEM cELLS
Stem cells, by definition,
are cells that can either self-
renew to ensure a source of
continued growth or differen-
tiate to become different cell
types. Studying stem cells has
led to an interesting hypothesis
about cancer: Perhaps some
cancers have their roots in
“cancer stem cells” that can
continuously feed malignant
cells to a tumor. This scenario
could be likened to an ant
colony: you can kill workers,
but to wipe it out you must
get the queen. This could
explain why certain tumors
initially respond to treatment
but return with a vengeance.
If this hypothesis is accurate,
clinicians must do more than
shrink the tumor; they must
also treat the source.
Robert Oshima, Ph.D.,
co-director of the Tumor
Development Program in
Burnham’s Cancer Center,
is investigating the role of
the MELK gene in normal
mammary epithelial stem
cells. MELK is often over-
expressed in breast tumors.
The Oshima lab has found
that MELK is also highly
expressed in mammary
epithelial progenitor cells.
“We’re trying to understand
the role of MELK in these
tumors,” says Dr. Oshima. “If
you have a cancer stem cell,
you can screen for chemicals
that will push it to differen-
tiate to a benign state and
those could be less toxic.”
Dr. Oshima is cautiously
optimistic. “The cancer stem
cell idea could be valuable
early on in a cancer’s progres-
sion,” says Dr. Oshima.
“However, tumors evolve and
find ways to grow.”
STEM cELLS FOR cLINIcaL USE
The ultimate goal for stem
cell research is clinical appli-
cation. To get there, scientists
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Robert Oshima, Ph.D.
Ph.D. student Karen Wei at work in the stem cell core.
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Like many in the research community, Stuart A. Lipton, M.D., Ph.D., was very pleased when President Barack Obama announced new federal rules governing embryonic stem cell research.
He is particularly excited that the renewed federal support for basic stem cell research will mean more funding from the California Institute for Regenerative Medicine (CIRM) for disease-focused investigations.
“The landscape is changing,” says Dr. Lipton. “The California money is going to become more about translational research
and finding treatments for disease.”
CIRM was estab-lished by Proposition 71, the California Stem Cell Research and Cures Initiative, which provided $3 billion to fund stem cell research. This stable source of funding has made California a leader in stem cell research, helping inves-tigators find the preliminary data that is so important to getting NIH grants. CIRM plays a unique role by funding the stages between basic research and clinical trials, a critical gap that must be bridged to get treatments to patients.
a UNIqUE cOLL aBORaTION
In 2006, Burnham, The Scripps Research Institute, The Salk Institute and the University of California, San Diego united to create the San Diego Consortium for Regenerative Medicine. This collaboration is now known as the Sanford Consortium after receiving a $30 million gift from T. Denny Sanford in 2008. The consortium was created to unite the expertise of these four world-renowned research institutions to move stem cell research forward more quickly.
“The Sanford Consortium fills gaps,” says Evan Snyder, M.D., Ph.D., Program Director, Stem Cell and Regenerative Biology. “None of us researchers individually are as smart as all of us together. So we fill each other’s gaps. You don’t need to duplicate efforts, or replicate expertise that another member of the consortium has.”
To maximize this efficiency, the Sanford Consortium is building a research facility within walking distance of all four institutions. By putting the collective expertise under one roof, the Consortium hopes to reduce the distance to new treatments.
The Stem Cell Community
must discover methods, or
protocols, to consistently
create the desired cells every
single time.
Alexey Terskikh, Ph.D., and
colleagues have developed a
protocol to rapidly differentiate
human embryonic stem cells
into neural progenitor cells that
may be ideal for transplanta-
tion. Their research could be
adapted to produce committed
neural precursor cells, one
of the key requirements for
clinical applications. When the
neural precursors created using
this protocol were transplanted
into mice, they became active
neurons and integrated into the
cortex and olfactory bulb. No
signs of cellular overgrowth or
tumors were found.
“The uniform conver-
sion of embryonic stem cells
into neural progenitors is the
first step in the development
of cell-based therapies for
neurodegenerative disorders
or traumatic injuries,” said
Dr. Terskikh. “Many of the
methods used to generate
neural precursor cells for
research in the lab would never
work in therapeutic applica-
tions. This protocol is very well
suited for clinical application
because it is controllable,
robust and reproducible.”
Evan Snyder, M.D., Ph.D.
Infantile hypophosphatasia (HPP) is a horrible disease and often fatal. A rare form of rickets, HPP makes bones dangerously fragile.
When Baby Amy was flown from her home in Ireland to Winnipeg, Canada, to be treated for HPP, she was transported in an insulated box to prevent her bones from breaking. However, after receiving an enzyme replace-ment therapy developed by José Luis Millán, Ph.D., and
collaborators, Baby Amy was healthy enough to be held by her mother and make the trip home to Ireland.
“While physicians may be familiar with the feeling of helping other human beings and alleviating their suffering on a day-to-day basis, it is very rare for a basic scientist to experience that,” says Dr. Millán. “Baby Amy has allowed me to experience that satisfaction. It has been very rewarding to see this therapy improve the quality
of life of the first patient ever to receive it.”
The drug, called ENB-0040, progressed from design to orphan drug status in just three years and was the result of a strong collaboration between Dr. Millán’s lab, ENOBIA Pharma (a small Canadian biotech) and Michael P. Whyte, M.D., of Shriner’s Hospital for Children in St. Louis.
Dr. Millán is now working to prove that the treatment can also help patients with
more advanced disease. His lab is developing models of adult hypophosphatasia and assessing if this treatment can improve quality of life for patients with milder, but still debilitating, disease.
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Treating Baby Amy
The Lipton lab has demonstrated that attacks on the mito-chondrial protein Drp1 by the free radical nitric oxide, a process called
S-nitrosylation, controls the synaptic degradation associated with Alzheimer’s disease. Mitochondria are the energy store-houses of the cell, and their compromise causes synaptic injury and eventually nerve cell death.
Prior to this study, the molecular mechanism by which beta-amyloid proteins damaged neurons was unknown. These findings
suggest that preventing S-nitrosylation of Drp1 may reduce or even prevent neurodegeneration in Alzheimer’s patients. The paper was published in the April 3 issue of the journal Science.
The team, led by neuroscientist and clinical neurologist Stuart A. Lipton, M.D., Ph.D., director of the Del E. Webb Center for Neuroscience, Aging and Stem Cell Research, showed that S-nitrosylated Drp1 facilitates mitochondrial fragmentation, thus damaging synapses, which are critical for learning and memory. Synaptic impairment leads to the dementia seen in Alzheimer’s.
“We now have a better understanding of the mechanism by which beta-amyloid protein causes neurodegeneration in Alzheimer’s disease,” says Dr. Lipton. “By identifying Drp1 as the protein responsible for synaptic injury, we now have a new target for developing drugs that may slow or stop the progression of Alzheimer’s.”
Stuart A. Lipton, M.D., Ph.D.
José Luis Millán, Ph.D.
Alzheimer’s Disease Linked to
Mitochondrial Damage
Pam Itkin-Ansari, Ph.D., Seung-Hee Lee, Ph.D., and colleagues have demonstrated in mice that transplanted pancreatic beta cells are protected from the immune system when encapsulated in a polytetrafluorethylene (PTFE) device.
The study, which suggests a new approach to treating type 1 diabetes, was published online on April 8 in the journal Transplantation.
The team showed that, after transplantation, the precursor cells mature into functional beta cells that are glucose-responsive and control blood sugar levels. Also, the study demonstrated that using precursor cells, instead of more committed beta cells, enhanced the cell transplant’s chances of success.
“The results exceeded our expectations,” said Dr. Itkin-Ansari, assistant adjunct professor at Burnham and the UC San Diego School of Medicine. “We thought that T-cells, although unable to penetrate the device, would cluster around it. But we found no evidence of an active immune
response, suggesting that the cells in the device were invisible to the immune system.”
The investigators used two different mouse models in the study. The team trans-planted mouse islet cells into other mice to show that the cells were protected from the immune system when encap-sulated in PTFE. Human cells encased in PTFE were then transplanted into immunodefi-
cient mice to study the viability and function of both mature beta cells and precursor cells inside the device. Itkin-Ansari’s team found that by using precursor cells that had not completely differentiated, the transplanted cells could regenerate into fully functional beta cells. This has important implications for how stem cell-derived tissue should be transplanted in the future.
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Pam Itkin-Ansari, Ph.D.
Pancreatic Beta Cells
and the Immune System
Investigators in the Wolf lab have deciphered a large percentage of the total protein complement
(proteome) in Schizosaccharomyces pombe (S. pombe) fission yeast.
Laurence Brill, Khatereh Motamedchaboki, Ph.D., and lead investigator Dieter Wolf, Ph.D., developed a novel method to iden-tify 4,600 proteins in the organism using an array of sophisticated techniques. The research was published online on March 9 in the journal Methods.
“Analysis of the proteome of an organism tells us so much more than simple DNA sequence analysis,” says Dr. Wolf. “Proteome analysis gives us a snapshot of what proteins are being expressed in the cell at any given point in time. This can tell us how protein expression changes in response to certain stimuli and in disease states, which may help identify new biomarkers for diseases. We are now applying the methodology to protein profiling of human stem cells in collaboration with Burnham’s stem cell program director, Dr. Evan Snyder.”
DNA analysis of the yeast’s genome predicts 5,027 proteins. The team identified 4,600 proteins, which is not quite the entire proteome. The remaining 400 are only expressed during S.pombe’smating state.
Wolf Lab Deciphers
Yeast Proteome
Dieter Wolf, Ph.D.
Anatomy of a MoveIn late April, Burnham began the three-week process of packing and transporting files, beakers and high-tech equip-ment to its new campus at Lake Nona in Orlando, Florida. More than a dozen moving companies and equipment specialists participated in the multi-phase move.
This was not a simple feat. Moving sensitive microscopes, robots
and finely calibrated equipment requires specially-designed crates
to protect the cargo, liquid nitrogen to freeze delicate cells to
minus 346 degrees Fahrenheit (10 times colder than ice), lab-
trained movers and 3,000 feet of bubble wrap.
Björn Tyrberg, Ph.D., a diabetes researcher, moved a sophis-
ticated microscope while taking special precautions to protect
tiny insulin-producing cells during transport. Layton Smith,
Ph.D., who studies obesity-related heart disease, froze 10 million
cells in advance, so he could quickly restart his research.
“This is a complicated process,” said Cyril Doucet, vice
president of operations for Burnham Lake Nona. “But we started
planning for the move more than eight months ago. We had to
plan thoroughly to minimize the impact on our research and to
keep our science moving forward.”
In all, scientists moved more than 350 pieces of equipment,
from a vortexer (the size of a tea kettle), to a four by eight-foot
bio-safety cabinet. Each piece of equipment was disassembled,
reassembled and recalibrated. Because the equipment is so sensi-
tive, if a piece breaks, it can take months to repair or replace.
Burnham is the first facility to be completed at the new
medical city at Lake Nona, which will include the University
of Central Florida College of Medicine, Nemours Children’s
Hospital, VA Medical Center, M.D. Anderson Cancer Center
Orlando and a proposed University of Florida research facility.
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B U r n h a m F l o r I d a N E w S
LaytonSmith,Ph.D.,whostudiesobesity-relatedheartdisease,froze 10
million cellsinadvance,sohecouldquickly restart his research.
Layton Smith, Ph.D.
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Burnham’s new facility in Lake Nona, Florida was designed with open labs and extensive telecom-munications technology, so scientists can easily collaborate with colleagues in the next lab or around the world.
The new facility will house a variety of specialists, from biochemists to cell biologists, pharmacologists and medic-inal chemists, and is intended to encourage collaboration among these specialties to advance science.
“When you put different disciplines together in a building like this, exciting things happen,” says architect Gary C. Shaw.
In addition, the 175,000 square foot facility is poised to become the largest private research institute in Florida with a Gold Level Leadership in Energy and Environmental Design (LEED) certification, which will be awarded after two to three months.
“Burnham is on its way to a Gold Level certificate, which indicates that it is one of the greenest of the green, and has been built to the highest environmental standards,” says Shaw.
The building’s design and construction must meet a variety of criteria to gain LEED certification. For example, a project must use at least 10 percent of its materials from regional sources to reduce emissions from long distance transpor-tation. More than 20 percent of Burnham’s materials were made within 500 miles of the site.
Building an energy-efficient scientific building offered unique challenges because of the labs’ exten-sive exhaust and air filtration needs. “We incorporated a tremendous amount of reflective, glazed window glass into the construction to allow low levels of solar heat while welcoming ample natural light,” says Shaw.
MEaSURING ThE SaVINGS
While building a green facility is very demanding, the rewards are substantial, both environmentally and monetarily. For example, 95 percent of construction waste was diverted from landfills. Metal was recycled, concrete waste was crushed and used for road base and wood was chopped into mulch. Even leftover drywall scraps were ground into fertilizer for local sod farmers.
Energy costs at Burnham Lake Nona are expected to be 25 percent below those of a comparable building. The roof is made of a special membrane that reflects heat, and labs and offices have motion sensors to control lighting. The building is oriented east/west with over-
hangs and shading devices on exterior walls to minimize heat and sun reflection. More than 90 percent of the interior spaces have an outside view, decreasing the energy needed to light them. Burnham’s annual energy savings will be equivalent to the average yearly consump-tion of 6,600 homes.
“Burnham is proud to have participated in the design and construction of a state-of-the-art research facility that was built using environmen-tally-friendly methods and will require less energy and water than a similar scientific struc-ture,” says Daniel Kelly, M.D., Burnham’s Scientific Director at Lake Nona.
Intellectually Sound,
Energy Efficient
Burnham Lake Nona
p h I l a n T h r o p y U P D a T E
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Caroline Nierenberg and Kathryn Stephens will chair this
year’s gala. No strangers to high-profile charitable events, both
have independently chaired the Las Patronas Jewel Ball.
Why focus on the power to cure? Burnham discoveries benefit
people of all ages, and Burnham researchers have discovered valu-
able new treatments. The Institute has generated more than 250
patents, and applications are pending on 300 more.
“The benefit that Burnham research brings to the world is
beyond calculation,” says Nierenberg. “We want to celebrate that
contribution and highlight the beauty of the work they do.”
This year’s presenting sponsor is Life Technologies. Greg
Lucier, CEO of Life Technologies, is vice chair of Burnham’s Board
For 32 years, Melvin Clause worked for Convair as a test pilot, flight engineer and field service engineer. He was onboard a 42-hour, non-stop, non-refueling flight, tested the XC99 transport (one of the largest planes ever flown) and flew everything from 2-cylinder prop planes to 168-cylinder jets. But the biggest chal-lenge he ever faced was watching his wife Phyllis McArdle die of Alzheimer’s disease.
“Phyllis lived in fear that she
would inherit the disease, which
did indeed happen,” says Clause.
“She was diagnosed in 2001 and
passed away in 2008.”
Phyllis had a long career as a
school counselor and teacher in
Salt Lake City. She played violin
in the symphony and liked to
camp, fish and four-wheel drive
through the back country.
“She tolerated my accordion,
and even put up with a month
in a tepee,” says Clause.
Melvin Clause pays tribute
to Phyllis through the Melvin
and Phyllis McArdle Clause
Scholarship Fund at Burnham.
Created through a bequest, the
scholarships are both a gift and
a challenge to the next genera-
tion of scientists: find a cure for
Alzheimer’s disease.
“She was an angel, or as
close to one as you could find,”
says Clause. “The hardest part
is that the woman I loved grad-
ually faded away. I want to keep
her memory alive through this
scholarship fund and support
scientists in search of a cure.”
Ifyouwouldliketoestablish
anamedscholarshipfundto
supportgraduateandpost-
graduateeducationatBurnham
throughanestategift,please
contactPattyFullerat858-795-
Save the Date: The 2009 Burnham Gala
The Power to Cure willbeheldattheHyattRegency
LaJollaAventineonNovember14.
of Trustees. Additional lead sponsors include Jeanne and Gary
Herberger, Peggy and Peter Preuss and Roberta and Malin Burnham.
Formoreinformation,pleasecontactChelseaJonesat
Honoring an AngelBy PaTT y FULLER
Caption: Gala co-chairs Caroline Nierenberg and Kathryn Stephens stand next to Burnham’s
Shimadzu Xcise robot, which Las Patronas helped purchase.
Phyllis McArdle and Melvin Clause
p h I l a n T h r o p y U P D a T E
www.burnham.org | The BUrnham reporT 11
Forget the fine food and wine, the cheering crowd, the red carpet, the noise-makers. Forget all that.
At 7:30, the games began
and people got down to
some serious fun. On April
30, more than 300 guests
packed a transformed Mission
Tower Ballroom at the Del
Mar Fairgrounds to BringIt!
Presented by Burnham and
the HeadNorth Foundation,
the event was a crazy combi-
nation of trivia questions,
game show enthusiasm
and spirited competition to
support stem cell research
at Burnham to treat chronic
spinal cord injuries.
Imagine 30 teams of 10
facing off in a series of trivia
contests with wacky chal-
lenges thrown in, leading to
the final face-off for the cham-
pionship. Between games,
Eric Northbrook introduced
the fund-a-need; StandUp
forEric. And people did stand
up. BringIt! raised nearly
$170,000 to support the
HeadNorth Chronic Spinal
Cord Injury Project.
Congratulations to
2009 BringIt! champions,
Team HeadNorth. Also,
many thanks to emcee Bill
Menish, lead sponsors Life
Technologies and Biomed
Realty and co-chairs Stath and
Terry Karras.
Did you miss it? Sorry
to hear that, but it’s okay.
Burnham and HeadNorth
are doing it again next
year—bigger, better, faster,
funner—on April 24, 2010, at
the Del Mar Fairgrounds. Is
someone going to take the title
away from Team HeadNorth?
They dare you.
Formoreinformation,
contactChelseaJones
at858-795-5239or
Bring It!
5Above, Ryan Baker belts out
the song he’s listening to during
the iPod challenge.
4Right , Team HeadNorth:
Matt Carlson, Eric Northbrook,
Chris High—Bring It! 2009 Champions
6Below, the frost-a-cake
challenge nearly escalated into an
on-stage food fight. Andrew Morse,
James Pires, John Garrigan, David
Thomas, Ron Lack, Dawn Saunders,
Chris High and Mary Macie.
As Malin Burnham steps down as Board of Trustees chair,
many people are noting what a profound impact he and his
family have had on the Institute. For nearly three decades, the
Burnham family has donated their time, talent and treasure to
grow the organization and advance basic biomedical science.
Here are some reflections on Malin Burnham’s 14 years as chair
and his ongoing passion for Burnham Institute for Medical
Research.
“AtthefirstNationalCancerInstitute(NCI)sitevisit,inresponsetoourapplicationforacancercentergrant,weneededtodemonstratewidecommunitysupport.Malinappearedinpersontotestifyonourbehalf.Tothisday,wearestillsupportedbyanNCIcancercentergrant.
“ButMalin’seffortsdidnotendthere.Hecontributedsignificantlybyjoiningtheboardoftrustees.Hisleadershipaschairmansetthestageforourfuturegrowth,andhealsohelpedbuildtheboardbyrecruitingstrongleaderswhowereenthusiasticaboutourmission.”
—LillianFishman,co-founder
“Malinisoneofthoselargerthanlifepeoplewhohasanimpactoneverythinghegetsinvolvedwith.HeisbroadlyfocusedonSanDiegoandistheguardianofourregion’swellbeing.AddinghisnametotheInstituteandservingaschairmanoftheboardputtheInstituteonthelocalmap.ThescientificteamfollowedthroughbyputtingBurnhamontheworldmapofbestresearchinstitutes.”
—DuaneRoth,CEO,CONNECT
“TherearenoshortageofpositiveadjectivestodescribeMalinBurnham.Heisdedicatedtothemission,decisive,experienced,persuasive,engagedandproactive.HeiscompletelyvestedinthesuccessofBurnhamInstituteforMedicalResearch.”
—GayleWilson,BurnhamTrustee
Editor’snote:TheInstituterecentlycommissionedaformalportrait
ofRobertaandMalinBurnhamthatwillhangintheexecutivesuite.
ItwillserveasaremindertoBurnhamleadership,staffandallwho
visitourcampusoftheimpacttheBurnhamfamilyhashadonthe
Institute.
When I first visited the La Jolla Cancer Research Foundation (LJCRF) in 1982, I was extremely impressed.
I especially liked what
I heard and saw about co-
founders Dr. William and
Lillian Fishman. I recognized
their scientific talent and the
quality of the culture they had
established, which was very
open and
transparent.
Everyone
was in it
together, and
there were no
silos, artificial
walls or rules
to inhibit
collaboration. I came to the
conclusion that LJCRF was a
hidden gem, and that I should
do what I could to help.
One of the more personal
aspects of my relationship
with the Institute was my
own experience, about seven
years ago, with cancer. I had a
PSA test come back high, and
the biopsy showed that I did
indeed have prostate cancer.
I was lucky we found it early.
But it’s important to note that
the PSA was perfected as a
result of work done by Dr. Eva
Engval, a Burnham researcher.
Roberta and I, along with
the Burnham family, have had
the wonderful opportunity of
supporting
the
Burnham
Institute
for Medical
Research.
I particu-
larly enjoy
providing a
combination of our financial
resources with shoe leather
and brain power. Of all the
things I have done, business
and community-wise, helping
the Burnham Institute has
given me the most satisfaction.
B U r n h a m L E a D E R S h I P
12 The BUrnham reporT | www.burnham.org
In His Own Words
A Tribute to Malin Burnham
Malin and Roberta Burnham
p r e s I d e n T ’ s M E S S a G E
www.burnham.org | The BUrnham reporT 13
15. JohnReedessay
Fulfilling the Promise of Stem CellsIn 1974, Burnham co-founder Dr. William Fishman discovered placental alkaline phosphatase,
a pregnancy protein, in an adult human lung tumor. Soon thereafter, Dr. Fishman found several
examples of tumors producing fetal proteins, prompting him to hypothesize that cancer might be
caused by adult cells reverting back to the primitive fetal state during which cells divide rapidly and
migrate through the developing body. This breakthrough helped create the field of oncodevelop-
mental biology and informed much of the early research at Burnham. We now recognize that the
primitive cells Dr. Fishman observed were actually adult stem cells that had become cancerous.
Our bodies have small numbers of stem cells that constantly renew tissues and organs with fresh
cells. Today, scientists believe that these adult stem cells are the source of many cancers. By
delving into the relationships between fetal development and cancer, Dr. Fishman’s work set the
stage for Burnham’s current work in stem cell biology.
Stem cell research provides a powerful approach to unlocking the mysteries of human disease.
Burnham scientists, for example, make disease models by synthetically creating the equivalent of
embryonic stem cells from patients with genetic predispositions to diseases, then converting those
synthetically-derived stem cells into specific types of cells in which the disease manifests (brain
cells, heart cells, insulin-producing cells). With these disease-specific cell lines, we can expand
our understanding of the complex biological mechanisms that lead to cancer, Parkinson’s disease,
diabetes and other conditions. We can test new medicines, rapidly gaining insights into the best
candidates to take, eventually, to clinical trials.
In addition, regenerative therapies have the potential to treat many conditions by replacing cells
lost to disease, trauma or aging. Once clinical protocols are perfected, stem cells will be grown and
differentiated into heart cells, nerve cells, insulin-producing cells and other therapeutically useful
cell types and transplanted into patients. But transplantation is only one way stem cells can benefit
human health. As forecasted by Dr. Fishman’s seminal work, our bodies also contain endogenous
adult stem cells that heal tissue damage, though inefficiently. Burnham researchers are working to
harness these cells and boost their efficiency. This research may lead to medicines that mobilize
endogenous adult stem cells to regenerate the heart, brain, spinal cord, liver or other tissues.
While we are excited about the enormous potential of stem cell research, we must recognize
that this line of investigation is at an early stage. Because stem cell-based therapies are largely
unproven, pharmaceutical companies are reluctant to invest resources to study how they work, and
thus that responsibility falls to non-profit organizations like Burnham. We encourage you to join
in supporting our mission to make stem cell-based therapeutics not just the passion of dedicated
scientists and the dream of those suffering from debilitating disease, but a reality that saves lives.
John C. Reed, M.D., Ph.D.
PresidentandCEO
ProfessorandDonaldBren
PresidentialChair
Withthesedisease-specificcelllines,wecanexpandourunderstandingofthecomplexbiologicalmechanismsthatleadtocancer,Parkinson’sandotherdiseasesandtestnew generationsofmedicinestotreatthem.
p h I l a n T h r o p y
Printedonrecycledpaper
Afteramotorcycleaccident
severedhisspinalcord,Eric
NorthbrookfoundedtheHeadNorth
Foundationtosupportthosewith
similarinjuriesandchampionspinal
cordresearch.Hiseffortsledhimto
EvanSnyder,M.D.,Ph.D.,director
ofBurnham’sPrograminStemCell
andRegenerativeBiology,whose
groundbreakingstemcellresearch
couldleadtonewtreatmentsfor
paralysis.Dr.SnyderandDr.Mark
TuszynskiatUCSanDiegohave
receivedagrantfromHeadNorth
todevelopstemcelltherapiesfor
chronicspinalcorddamage.
Partners in Science: EricNorthbrook
andDr.EvanSnyder
Nonprofit OrganizationU.S. Postage
PaIDThe Burnham Institute
“Livingwithachronic spinal cord injuryisaserious challenge,”saysNorthbrook.“TheHeadNorthChronic SpinalCordResearchProjectcouldbethesolution for thousandswholivewiththiscondition.”
Nonprofit OrganizationU.S. Postage
PaIDThe Burnham Institute
10901 North Torrey Pines Road
La Jolla, CA 92037
P h i l a n t h r o P y
Printed on recycled paper
After a motorcycle accident
severed his spinal cord, Eric
Northbrook founded the HeadNorth
Foundation to support those with
similar injuries and champion spinal
cord research. His efforts led him to
Evan Snyder, M.D., Ph.D., director
of Burnham’s Program in Stem Cell
and Regenerative Biology, whose
groundbreaking stem cell research
could lead to new treatments for
paralysis. Dr. Snyder and Dr. Mark
Tuszynski at UC San Diego have
received a grant from HeadNorth
to develop stem cell therapies for
chronic spinal cord damage.
Partners in Science: Eric Northbrook
and Dr. Evan Snyder
Nonprofit OrganizationU.S. Postage
PAIDThe Burnham Institute
“Living with a chronic spinal cord injury is a serious challenge,” says Northbrook. “The HeadNorth Chronic Spinal Cord Research Project could be the solution for thousands who live with this condition.”
6400 Sanger Road
Orlando, FL 32827