Fall 2003

Page 1



> Leukemia treatment on the fast track


> At the vanguard of stem cell research

> A new drug for Alzheimer’s


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.”


President and CEO

Researchers examine the effects of antisense DNA agents on cancer cells.