University of Nebraska Medical Center researchers have begun the first
clinical study in the United States to examine the safety and effectiveness
of a unique new drug that may have potential in the treatment of acute
myeloid leukemia (AML). AML, a cancer of the blood and bone marrow, is
the most common type of acute leukemia in adults, with an estimated 10,000
new cases a year.
UNMC is conducting the first and second phase of a clinical study in
adults, age 19 and over, whose treatment has failed or whom are not eligible
for conventional chemotherapy. Those who have not yet received any treatment
for their disease also may be considered for the study.
The goal of the study is to evaluate how safe the drug is and how effective
it is in inhibiting the growth of leukemia cells.
Participants will receive treatment at no extra cost on an outpatient
basis in the Lied Transplant Center on the UNMC/NHS campus. Study participants
will receive the drug on an outpatient basis. Participants can be part
of the study and continue to see their own physicians. They may refer themselves
or be referred to UNMC by their physicians.
James Foran, M.D., principal investigator of the UNMC study, said the
oral drug, whose name is currently confidential because of proprietary
issues, is part of a new class of drugs called receptor tyrosine kinases
(RTK) inhibitors, which appear to play an important role in the disease
process. He said the drug might be a potential adjunct, or additional treatment,
in what frequently is an incurable disease.
Leukemia cells in most patients express RTKs, or growth receptors, which,
when interacting with their respective growth factors, cause leukemia cells
to multiply.
There is no standard therapy for those whose initial treatment has
failed or are not eligible for conventional chemotherapy. Even with any
successive standard therapy, prognosis is poor, with an average survival
in the range of three months, said Dr. Foran, an oncologist and assistant
professor at UNMC.
This is a new treatment approach to leukemia. Initial studies in healthy
volunteers suggest that single doses of the drug are well-tolerated, and
about a dozen patients are currently being treated with this medication
for other cancer, in Europe and the United States, Dr. Foran said. This
is the first study in leukemia, and it is a very exciting opportunity at
UNMC to improve our understanding of how leukemia cells survive, how we
can bypass those survival mechanisms, and ultimately develop better treatments
for this disease.
The study is funded by Sugen, Inc., now part of Pharmacia Corporation,
a worldwide pharmaceutical company. Dr. Foran said UNMC was the first site
chosen to test the drug in AML because of its cancer research and clinical
programs.
Dr. Foran said studies like this enable researchers to learn more
about the biology of the disease in order to design new treatments with
better results and fewer side effects.
There are major changes, astonishing developments in our approach to
the treatment of cancer. This is due to research and the different way
we are thinking about and targeting cancers specifically. The changes Ive
seen in 10 years are incredible, he said.
Drugs like this are changing the way we think about leukemia, and I
believe will change the way we treat leukemia in the next few years, Dr.
Foran said. We probably wont get rid of chemotherapy because of its
effectiveness, but drugs like these will be important additions to therapy.
The Leukemia and Lymphoma Society estimates about 21,500 deaths in the
U.S. in 2001 resulted from leukemia. Of that, about one-third (7,200) died
from acute myelogenous leukemia.
AML, a rapidly progressing disease that originates in a cell in the
marrow, causes uncontrolled growth of developing marrow cells that affect
the body’s ability to fight infections. Dr. Foran said about one in four
patients under 60 years old with AML can be cured, but the disease in those
60 and over is much more difficult to cure.
AML is the most common form of acute leukemia and occurs mostly in people
in their 60s, 70s and 80s. The cause is unknown in most cases.
UNMC/NHS is recognized internationally for its expertise in the diagnosis
and treatment of leukemias and lymphomas and is one of the busiest bone
marrow and stem cell transplant centers in the world. James Armitage, M.D.,
now dean of the UNMC College of Medicine, launched the program in 1982.
Other physicians working on the study are UNMC oncologists Phil Bierman,
M.D., Gregory Bociek, M.D., Anne Kessinger, M.D., Steven Pavletic, M.D.,
Elizabeth Reed, M.D., Stefano Tarantolo, M.D. and Julie Vose, M.D.
For more information about the study, call Maribeth Hohenstein at (402)
559-9053.