Children's Hospital Researcher Leads Multi-pronged Attack on Infant Leukemia; New Grant Supports Targeted Treatments for Stubborn Disease


PHILADELPHIA, Dec. 6 /PRNewswire/ -- While survival rates for childhood
leukemia have dramatically improved over the past 30 years, infants with
this blood cancer continue to face difficult odds. Infant leukemia resists
treatments such as chemotherapy and stem cell transplants that may be
effective in older children, and infants are especially vulnerable to
treatment side effects.

A new research effort led by a leukemia expert at The Children's
Hospital of Philadelphia seeks to harness in-depth understanding of genes
and molecular pathways to develop highly specific drugs designed to kill
leukemia cells while causing few or no toxic effects on normal cells.

A five-year, $6.25-million Specialized Center of Research (SCOR) grant
from the Leukemia & Lymphoma Society brings together a diverse study team
of researchers from leading medical centers to develop innovative
treatments for infant leukemia. "Our goal is to streamline advances in
molecular medicine to find new treatment options," said Carolyn A. Felix,
M.D., principal investigator of the SCOR grant. A pediatric oncologist at
The Children's Hospital of Philadelphia, Dr. Felix also is a professor of
Pediatrics at the University of Pennsylvania School of Medicine.

The society recently announced its grant award in targeted therapies
for infant leukemia from its national office in White Plains, N.Y. The SCOR
grant draws on research collaborators from a variety of disciplines to
discover new approaches to treatment.

In addition to researchers from The Children's Hospital of Philadelphia
and the University of Pennsylvania, the grant includes collaborators from
the University of New Mexico, Johns Hopkins University, Stanford University
and Tulane University.

Especially integral to this effort to advance new treatments from bench
to bedside, the investigators also will work closely with the Children's
Oncology Group, the cooperative clinical research organization of pediatric
cancer centers throughout North America. "Even the largest medical center
has a relatively small number of patients with infant leukemia," said Dr.
Felix. "Collaborating with the Children's Oncology Group allows us to
capture data about and treat the vast majority of infants throughout North
America who have acute leukemia."

The full grant encompasses four projects, each of which hones in on a
piece of the infant leukemia puzzle. In infant leukemia, a gene called
mixed lineage leukemia (MLL) breaks and recombines with one of many partner
genes to form a translocation, an abnormal rearrangement within a
chromosome. Because MLL plays a critical role in blood cell development,
the translocation causes the overproduction of defective white blood cells that are the hallmark of leukemia.

Dr. Felix has pioneered methods to identify and describe the features
of MLL translocations. Her project within the SCOR grant will investigate
how potential drugs may trigger programmed cell death, called apoptosis, in leukemia cells in infants. Another project, led by Cheryl L. Willman, M.D.,
of the University of New Mexico, will use microarrays (DNA chips) to
identify drugs that may selectively attack cells with MLL translocations.

The project also seeks to unravel the molecular circuitry in leukemia cells
in order to predict those infants most likely to benefit from specific
targeted drugs.

Donald Small, M.D., Ph.D., of Johns Hopkins University, leads a SCOR
project to advance the development of new drugs to target abnormalities in
a gene called FLT3 that are associated with uncontrolled growth of the
leukemia cell population.

A final team under the SCOR grant, led by Michael Cleary, M.D., of
Stanford University, will characterize the MLL leukemia stem cell that
gives rise to all other leukemia cells, because the stem cells are
essential targets for new treatment to eradicate the disease. Core research
facilities at several other academic medical centers will provide
specialized support in analyzing data.

The SCOR officially began on October 1. "This grant from the Leukemia &
Lymphoma Society creates a unique opportunity to produce advances against a very challenging form of leukemia," said Dr. Felix. "The SCOR grant brings
together researchers from around the United States and harnesses our
efforts to build bridges between basic science and bedside treatments and
have the utmost impact for infants with this dread disease."

About The Children's Hospital of Philadelphia: The Children's Hospital
of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children
worldwide. Its pediatric research program is among the largest in the
country, ranking third in National Institutes of Health funding. In
addition, its unique family-centered care and public service programs have
brought the 430-bed hospital recognition as a leading advocate for children
and adolescents. For more information, visit
Contact: John Ascenzi
Phone: (267) 426-6055