Robert Wood Johnson Medicine • Spring 2011 • Population Science

University, Dr. Ma uncovered
another gene, named junctophilin,
as a biomarker for
cardiovascular diseases.
Junctophilin regulates how the
different parts of the cell communicate
with one another to coordinate
the onset and termination
of calcium inside the cells.
Genetic mutations in junctophilin
can cause heart failure, muscle
wasting, or neurodegeneration.
Dr. Ma’s research team is
currently conducting laboratory
research to understand how
junctophilins function in normal
physiology and how to apply the
Cheryl F. Dreyfus, PhD
In the demyelinating disease
multiple sclerosis (MS), the
myelin sheaths that surround
nerves in the brain and spinal
cord are inflamed and damaged.
Cheryl F. Dreyfus, PhD, professor
and acting chair, Department of
Neuroscience and Cell Biology, is
JOHN EMERSON
Jianjie Ma, PhD
team’s bench work to clinical
medicine.
Recently, a protein named MG53,
a key initiator of cell membrane
repair in damaged tissue, was
discovered in Dr. Ma’s lab. This
finding has generated wide interest
in both academic research and
the pharmaceutical industry.
The protein is expected to have
applications in repairing tissue in
patients who suffer from severe
complications of disease and
aging, such as muscular dystrophy
and heart failure.
The translation of this research,
through collaboration with clinicians
and pharmaceutical companies,
will help determine if what
was discovered using animal
prototypes can be applied to
human treatment. To accelerate
the development of these discoveries
into clinical therapies, a
university spin-off company,
TRIM-edicine Inc., was created to
commercialize the intellectual
properties discovered in Dr. Ma’s
laboratory at UMDNJ-Robert Wood
Johnson Medical School.
The discoveries could have
far-reaching applications in
geriatric and regenerative
medicine, to treat chronic human
diseases as well as acute or
traumatic tissue injuries. Funding
for Dr. Ma’s research is provided
by grants from the National
Institutes of Health, with support
from the New Jersey Commission
on Science and Technology.
Research Examines Potential for Treatment of Multiple Sclerosis
leading a study to determine if
there is a connection between
the neurotrophin BDNF (brainderived
neurotrophic factor) in
the brain and reduction or even
repair of the myelin damage.
The study is currently supported
by a four-year, $1,365,000
grant from the National Institutes
of Health and additional funding
through a grant from the National
Multiple Sclerosis Society.
“In a culture dish, BDNF
causes cells that are destined to
become oligodendrocytes to
divide and mature and to start
to express the proteins associated
with oligodendrocytes — the
myelinating cells of the central
nervous system,” explains Dr.
ANDREW HANENBERG
Dreyfus. “When we observed mice
that have a reduced ability to
make BDNF, we discovered that
they exhibited a reduction in
the number of oligodendrocyte
progenitors and a reduced ability
to express mature traits.”
It was then that Dr. Dreyfus
began to ask, “Could this have
something to do with MS?”
Dr. Dreyfus and her team
evaluated effects of BDNF in
a demyelinating mouse model
of the disease. “In this model,
a lesion in the myelin appears,
which then repairs itself,” says
Dr. Dreyfus. The mice that
exhibited reduced BDNF did
much worse in this model.
Reduced BDNF affected a number
of oligodendrocyte progenitors
and their ability to repair the
damage. Conversely, when BDNF
was infused into the lesion site,
it was able to partially reverse
damage.
Current work in the laboratory
is investigating drugs that might
be able to increase levels of
BDNF in the brain. Dr. Dreyfus
emphasizes that there is a long
way to go before we understand
how to manufacture such a drug
that would increase BDNF and
lead to repair. The hope is
that one day we could have a
significant effect on a devastating
disease.
Robert Wood Johnson ■ MEDICINE 11