The Marker - 2004 - Huntington's Disease Society of America

esearch
grants & fellowships
HDSA GRANT AND FELLOWSHIP RECIPIENTS 2003–2004
Though the HDSA Coalition for the
Cure serves as the flagship of HDSA’s
research program, HDSA’s prestigious
Grant and Fellowship program has
contributed significantly to advancing
our knowledge about HD and the
disease process.
The Grant and Fellowship program
provides essential funding to research
projects in their early stages of
development. This “seed money” allows
HDSA Grant recipients to generate
sufficient data and advance their project
from a promising hypothesis to a mature
study that will then be eligible for greater
funding from larger national agencies
like the National Institutes of Health
(NIH) or the National Institute of
Neurological Disorders and Stroke
(NINDS). The HDSA Grant program
provides grants of up to $100,000
(payable over two years if renewed).
HDSA Fellowships were created to
expand the pool of young researchers
who are interested in studying HD.
HDSA Fellowship awards can total
up to $80,000 (payable over two
years if renewed).
Therapeutic Initiative awards are special
grants that fund research projects that
will develop assays (tests) to look at
various promising compounds and drugs
that might yield an effective treatment
for HD. The HDSA Therapeutic
Initiative grant program will soon be
expanded into the HDSA Drug
Discovery Team – a collaborative that
will pursue partnerships with industry to
develop therapeutic remedies for HD.
New Grants 2003–2004
Ilya Bezprozvanny, Ph.D.
University of Texas Southwestern
Medical Center
Dallas, TX
Association of Inositol-(1.4.5)
Triphosphate Receptor with HAP1
and Huntingtin Proteins in the Brain:
Implications for Huntington’s
Disease
It appears that huntingtin-associated
protein-1 (HAP1), which binds with
huntingtin, interacts with a calciumrelease
channel, in yeast. Calcium
gradients affect a cell’s ability to
produce usable energy and Huntington’s
Disease seems to be related to this
misfunction. Dr. Bezprozvanny is thus
investigating the relationship between
the calcium channel, HAP1, and
huntingtin (protein) to determine how
alterations may contribute to and impair
Huntington's Disease.
Pamela J. Bjorkman, Ph.D.
California Institute of Technology
Pasadena, CA
Structural and Binding Studies of
Mono-, Bi-, and Trivalent Anti-Poly-
Gln Antibodies Bound to Huntingtin
Exon1
Mutant huntingtin protein aggregates
and forms large clusters within the
nucleus of HD cells. Dr. Bjorkman is
investigating the potential to physically
block this interaction using a variety of
reagents that are targeted to multiple
binding sites on mutated huntingtin.
Janet Dubinsky, Ph.D.
University of Minnesota
Minneapolis, MN
Proteomics of CNS Mitochondria
Inhibitors of mitochondria, the energy
factory within a cell, mimic the
pathology of HD, suggesting
mitochondrial disorder may contribute
to Huntington’s Disease. Recently, Dr.
Dubinsky discovered that mitochondria
in the striatum, the region of the brain
most affected in HD, are more at risk of
disruption. She now examines the
protein profile for both cortical and
striatal mitochondria in the hopes of
identifying a factor unique to striatal
cells that may provide a target for
therapeutic treatments.
Pietro Mazzoni, M.D., Ph.D.
Columbia University
New York, NY
Impairment of Motor Learning as a
Biologic Marker of Pre-symptomatic
Huntington’s Disease
Currently, when patients are diagnosed
with neurodegeneration, a great deal of
brain damage has already occurred.
Better exams, such as those being
designed by Dr. Mazzoni, are therefore
necessary so that once neuroprotective
treatments are discovered, they may be
administered as early as possible to
avoid extensive neurodegeneration.
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