Fall 2003

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,

THE POWER

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