2004 - 2005
Children’s Memorial Hospital
Dr. Michael Burke (Right), Washington Square Health Foundation Fellow in Hematology, Oncology
Research (2004-2006) at Children’s Memorial Hospital, along with Dr. William Tse, are analyzing the
developmental properties of blood and muscle cells.
Washington Square Health Foundation is helping to make great strides in promising stem
cell transplantation and muscular dystrophy research at Children’s Memorial Hospital
through grant support over the past three years of Dr. Michael Burke, the recipient of the
Washington Square Health Foundation funded Hematology, Oncology Research
Fellowship (2004-2006).
Dr. Burke is part of a hematology/oncology team treating over 1,300 patients each year.
The division’s stem cell program, moreover, is among the largest in the nation,
performing more than 70 transplants each year and 600 transplants since the programs
inception in 1992. The level of expertise available at the institution combined with its
large clinical population makes it an excellent place to test and develop novel stem cell
transplantation therapies, which, when completed, will directly translate to better
therapies and cures for diseases.
Dr. Burke, under the mentorship of highly regarded stem cell researcher William Tse,
MD, PhD, is focusing his research on the potential of stem cell therapy to regenerate
tissue that has been damaged by disease, particularly muscular dystrophy. Muscular
dystrophy (MD), is a term used to describe a group of nine inherited disorders that cause
progressive muscle weakness, and affects as many as 110,000 people in the US.
Currently, there is no cure or way to prevent MD, which is why Dr. Burke’s
groundbreaking research is so important.
In the past several decades, blood and bone marrow stem cell transplantation has been
demonstrated to be a very successful treatment for many blood diseases in childhood. It
has recently been shown that these stem cells might possess an astonishing degree of
developmental plasticity; that is, they appear to have the capacity to develop into other
tissues unrelated to blood, such as nerve and muscle tissues. This raises the intriguing
possibility that bone marrow transplantation can be used to treat genetic diseases
involving non-hematopoietic tissues.
Using mouse models, Drs. Burke and Tse are finding that bone marrow stem cells, called
mesoangioblasts, have the capacity to develop into mature muscle fibers. This
phenomenon illustrates the extraordinary degree of developmental plasticity
demonstrated by stem cells, and are looking for ways to enhance the development of
mesoagnioblasts into muscle cells after transplantation.
Dr. Burke, along with Dr. Tse, is analyzing the properties of blood and muscle stem cells
and establishing new functional assays to test their developmental potential. They are
manipulating mouse embryonic stem cell genes that are important in muscle formation
and following the maturation of these stem cells into muscle cells. Using these
techniques, Drs. Burke and Tse obtained evidence that inflammatory cells in regenerating
muscles provide important signals that recruit stem cells to muscles and promote their
growth into muscle fibers.
One of these growth factors mediating signals that promote stem cell growth into muscle
fiber, upon which Dr. Tse’s laboratory is focusing, is wnt. wnt is important not only in
muscle stem cell development but also in the formation of childhood muscle cancer
(rhabdomyosarcoma). Understanding how this growth factor functions should also lead to
the discovery of better treatments for childhood muscle cancer. Drs. Burke and Tse are
now working on novel ways to harness this and other factors to promote donor stem cell
engraftment after transplantation. The goal of the study, to understand the biology and
behavior of muscle stem cells, is the important first step in developing stem cell
transplantation into an effective cure for muscular dystrophy.
Dr. Burke’s ongoing stem cell transplantation research, made possible through the
support of Washington Square Health Foundation, is helping to uncover new knowledge
of how stem cell transplantation therapy might be used to treat other devastating illnesses,
particularly, muscular dystrophy, which affect hundreds of children and adults each year.
Dr. Burke’s work as a physician and researcher has benefited – and continues to benefit –
patients and families who come to Children’s Memorial Hospital for specialized care, and
is paving the way for a day when a cure for muscular dystrophy and other muscle
disorders is a reality.