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

HDSA Coalition for the Cure –
New Pathways to Discovery
By James Gusella, Ph.D. Chair, HDSA Medical and Scientific Advisory Committee
An important part of HDSA’s
mission is to develop an
effective therapy or a cure for
Huntington’s Disease. Most advances
in our understanding of HD and its
processes have come through basic
research which aims to gather the
fundamental knowledge and
understanding that is needed to develop
successful treatments for disease. It is
best conducted in an investigator-driven
manner, giving the individual scientist
the freedom to choose what he or she
thinks are important questions, as well as
the means to answer them, either
individually, or cooperatively with
others who have similar interests and
technologies. Each answer leads
inevitably to the next question that
needs to be addressed, and gradually
the knowledge critical to developing an
effective treatment or cure is uncovered.
The strength of the basic research
approach is that outcomes cannot be
predicted and therefore investigations
can lead in unexpected and very fruitful
directions. Basic research is the source
of most of the significant advances in
science and technology, and the
knowledge that it provides sets the
stage for applied research to engineer
a treatment. Although we have made
great progress in uncovering the exact
mechanism through which HD takes
effect, there is still much to understand.
It is vital, therefore, that we continue to
support basic research.
The flagship research program of HDSA
is the Coalition for the Cure, which
began in 1997 as a major new initiative
to bring together the leading HD
researchers who had proven to be
pioneers in the field, and provide them
with stable funding that would
1. foster cooperative efforts,
2. accelerate their ongoing research or
3. allow them to try high risk new
directions.
Early HDSA funded research was decided
upon by investigators who were deeply
familiar with the HD problem, and were
advised by a Steering Committee of
scientists typically not involved directly
in HD research but generally familiar
with the field. Over the last six years, the
original collaborative group of Coalition
scientists has been augmented by the
Basic research is the
source of most of the
significant advances in
science and technology.
addition of labs new to HD, but selected
by the Steering Committee as having
technologies and/or research interests
that have been deemed important to
finding answers to the HD puzzle. The
HDSA Coalition for the Cure currently
unites 17 laboratories from around the
world that have committed themselves to
understanding how the defective HD
gene causes the ravages of the disease and
to thereby add to the knowledge
necessary to cure it. Since its inception,
the Coalition has been incredibly
successful, as HDSA Coalition for the
Cure investigators have been at the
forefront of the recent fundamental
advances in HD.
As we have learned more about HD, the
questions that still need to be addressed
have become clearer and individual
laboratories have each begun working in
their own way to answer these critical
questions. Consequently, to take greatest
advantage of the emerging synergies and
interactions, HDSA is altering the
structure of the Coalition for the Cure.
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