THE BURNHAM INSTITUTE
IN THIS ISSUE:
> Leukemia treatment on the fast track
FROM RESEARCH,
> At the vanguard of stem cell research
> A new drug for Alzheimer’s
THE POWER TO CURE.
The Burnham Report FALL 2003
Vol. 1, No. 2
Leukemia meets its match. A new treatment should soon be available for a common form of leukemia. Based on the pioneering work of Professor and CEO John Reed, the new therapy, called Genasense, received “Fast Track” designation from the FDA in June, 2003.
In this edition of The Burnham Report, you’ll find exciting news of medications making their way to the clinic, thanks in part to the contributions of Burnham scientists. A new drug for Alzheimer’s, the first that actually prevents the death of brain cells, is currently in use in Europe and recently received FDA approval. A treatment for a common form of leukemia has been fast-tracked for FDA approval; this treatment is showing efficacy in clinical trials against many other forms of cancer as well. The Institute recently launched a program in human embryonic stem cell research, which we expect will lead to cell replacement therapies for many currently incurable diseases. I hope you are as heartened as I am by these advances. On behalf of all at The Burnham Institute, I thank you for your interest and support.
Genasense works by blocking the
Reed first derived its concept but
showed promise, Reed was successful
production of a protein called Bcl-2,
encountered difficulty obtaining
in obtaining a grant from the National
which is made in high levels by many
support. Genasense is likely to be the
Cancer Institute.
cancer cells. Bcl-2 prevents cell death
first approved treatment of a class
and, at high levels, protects cancer cells
based on what is known as anti-sense
likely be used to treat thousands of
from chemotherapeutic drugs. Clinical
DNA technology. Conventional drugs
patients—approximately 50,000 people
trials have shown that Genasense and
bind to proteins and inactivate them,
in the U.S. are living with CLL. The
traditional chemotherapy, administered
but anti-sense DNAs prevent the
disease affects the white blood cells that
in conjunction, can effectively reduce
production of proteins.
normally produce antibodies. Their
the number of cancer cells in patients
If approved for CLL, Genasense will
“It was an emerging idea,” recalls
abnormal growth alters the develop-
with CLL (chronic lymphocytic
Reed, “and people were skeptical.” But
ment and function of normal blood
leukemia), the most common form
he persisted and eventually obtained
cells, compromising patients’ immune
of adult leukemia.
the $2,000 needed to purchase
systems, and thereby their ability to
reagents that would enable him to
fight off infections. Treatment with
test the idea. After initial experiments
Genasense kills the leukemic cells.
Genasense is displaying efficacy in clinical trials for other leukemias as well, and for cancers including myeloma,
“I’ve been working on the concept
melanoma, lymphoma, and prostate,
of anti-sense to Bcl-2 for almost 14
breast, lung and colon cancers. This
years,” said Reed. “It’s highly gratifying
promising treatment had modest
to see this treatment so close to avail-
beginnings. As a postdoctoral fellow,
ability for patients and their families.”
JOHN C. REED, M.D., PH.D.
President and CEO
Researchers examine the effects of antisense DNA agents on cancer cells.
The Burnham Report FROM RESEARCH,
THE POWER TO CURE.
THE DEL E. WEBB CENTER FOR NEUROSCIENCE AND AGING
At the hub
of international research.
Human embryonic stem cells are
stem cell lines have been procured.
derived from fertilized eggs produced
In their initial projects, Burnham
during IVF (in vitro fertilization)
investigators are exploring methods of
that are not implanted in a woman’s
directing the cells to develop along
womb. Couples who have undergone
specific paths, including neural, cardiac
IVF and decided they have completed
and pancreatic. The hope is to develop
their families routinely have embryos
cells that could be used to treat neu-
remaining in frozen storage. A recent
rodegenerative diseases, heart failure,
study estimated that more than
and diabetes, respectively.
400,000 embryos are currently stored Working with stem cells, Burnham Professor Evan Snyder and his team
his stem cell team are making regenerative
Embryos at this early stage—called
to treat patients, however, they must be shown to be free of contaminants.
have restored, to partial but consider-
blastocysts—are roughly spherical
All of the currently federally approved
able degree, the ability of paralyzed rats
clusters of between 16 and 64 cells.
lines have been grown using mouse
to walk. The team is among a group
Some of these cells can be cultured in
cells as “feeder” or support layers, and
of scientists, at The Burnham Institute
laboratory dishes, and have the ability
these cells could release dangerous
and elsewhere, who have achieved
to give rise to many of the different
viruses and other pathogens. Therefore
impressive successes in animal models
cell types that exist in the body. Thus,
the Burnham stem cell team is devel-
of debilitating conditions. Before the
new heart, brain, muscle and skin
oping protocols to grow embryonic
promise of stem cells can be realized in
tissue can be derived from stem cells,
stem cells using human feeder layers.
people, however, research must be done
and used, it is hoped, to replace tissue
Other important steps before clinical
using human cells. Snyder and his col-
lost to disease or injury.
translation is possible include showing
leagues recently initiated a program of Evan Snyder (right) and
in the United States.
Before any line of stem cells is used
Adult stem cells are found in vari-
that the stem cells do not become
human embryonic stem cell research
ous locations in the adult body, and
the wrong cell type in a given organ
that positions The Burnham Institute as
some studies have suggested that stem
(for example, heart cells in the brain),
one of the hubs of a worldwide effort.
cells from one organ can “transform”
and that they do not make tumors.
medicine a reality.
under certain conditions to cells of
The Burnham Institute has provided
a different type. Other studies have
an infrastructure for some of the region’s
called these results into question.
most talented researchers in stem cell,
“At this point in time, we believe
developmental, and regenerative biology
stem cells obtained from an embryo
in order to collaborate as the Southern
or a fetus are the most plastic, with the
California Stem Cell Consortium. The
potential to be used for the widest range
consortium meets monthly to exchange
of diseases,”said Professor Evan Snyder,
ideas and foster joint projects. For
director of the Institute’s Stem Cells
example, The Burnham Institute will
and Regeneration research program.
host one such collaborative effort—in
Since the National Institutes of
this case, between The Burnham
Health can only fund work with
Institute and the Children’s Hospital
human lines derived before August 9,
of Orange County—which is one of
2001, most of the human embryonic
only five NIH-funded courses on
stem cell work at Burnham will be
human stem cells (intended for the
privately funded. To date, five human
international community).
leadership, including the 1999 Model Citizen Award from the Alexis de Tocqueville Society and the 2000 Philanthropist of the Year award from the National Society of Fund Raising Executives. In 1995, Malin and his family pledged a lead gift in the Institute’s re-naming campaign. “It has been especially satisfying for me to
see the enjoyment my children and grandchildren derive from participating in the significant venture of helping to cure disease,” he said. “We are involved in many activities in the San Diego area, but I like to think that by supporting scientific research, we are helping to benefit people throughout the world.”
GIVING WITH A GLOBAL VISION M A L I N B UR N H A M
Malin Burnham was first elected a Trustee of The Burnham Institute in 1982 and served as Board Chairman from 1983-1986 and 19871994. One of his many important contributions has been leading the effort to expand the Institute’s base of support in the greater San Diego area and beyond. In addition, Malin has been a strong advocate of amplifying the
Institute’s technology transfer effort, to ensure that the results of basic research find application in the form of new drugs and diagnostics. Malin is well-known to the San Diego community as the president and CEO of John Burnham & Company Insurance and Burnham Real Estate Services. He has received numerous honors for his civic and philanthropic
THE BURNHAM REPORT JOHN C. REED, M.D., PH.D.
President and CEO CHERYL A. MOORE
Senior Vice President and COO TERRY GACH
Vice President Resource Programs SUZANNE CLANCY, PH.D
Editor The Burnham Report LIPMAN HEARNE, INC.
Graphic Design BOB ROSS
Photography www.burnham.org RESEARCH HIGHLIGHTS
> Professor Manuel Perucho was named
Nonprofit Organization U.S. Postage PAID The Burnham Institute
recipient of the Cancer Research Professorship in Basic Cancer Research for 2003. The $50,000 award, conferred jointly by the American Association for Cancer Research and the National Foundation for Cancer Research, is given annually through peer nomination. Dr. Perucho was recognized for his pioneering contributions to the field of cancer genetics, and the award will further his research on mechanisms underlying gastrointestinal cancer.
> The Burnham Institute has received a five-
> A team of Burnham scientists has secured
year $500,000 grant for cancer research from the Bristol-Myers Squibb Foundation. The award will provide seed funding for the cancer drug discovery initiative, which seeks to accelerate the translation of basic cancer biology research into new clinical therapies. “Our cancer drug discovery effort brings together the talents of biologists, chemists, biophysicists, and computational biologists, to discover new ways of treating cancer,” said President and CEO John C. Reed. “With this grant, Burnham scientists will have the resources to move more of our discoveries into pre-clinical testing.”
a multi-million dollar grant to develop new potential treatments for anthrax. The $3.3 million, from the National Institute of Allergy and Infectious Disease, will enable the investigators to screen more than a million drug candidates during three and a half years. The effort is led by Professor Alex Strongin (shown below); he is joined by Professor Robert Liddington, Associate Professor Maurizio Pellechia and collaborators at The Scripps Research Institute.
> Professor Barbara Ranscht has been awarded a grant from the Christopher Reeve Paralysis Foundation. The grant of $75,000 will support work in Ranscht’s laboratory aimed at deciphering the molecular codes that govern how nerves can grow and form functional connections after spinal cord injury.
The Burnham Report FROM RESEARCH,
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