THE BURNHAM INSTITUTE
IN THIS ISSUE:
> Leukemia treatment on the fast track
> 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 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.