Help for Today, Hope for Tomorrow. Annual Report FY 07-08

HDSA In Action
Research
This past year was an exciting period in the
history of HD research, with many drugs,
supplements, and genetic approaches moving
through the drug discovery pipeline, bringing
treatments closer to HD families. The progress
we have seen, as well as the confidence drawn
from the progress of the comprehensive strategic
plan for research point to many potentially positive
findings on the horizon.
HDSA wants to ensure that our families
always have access to the most current research
news and developments. Therefore, we have
updated the national website,
www. HDSA.org, with an extensive
research section that presents and
explains the latest findings from the
HDSA Coalition for the Cure and other
leading HD researchers. A research
pipeline graph available on the site
shows how far promising compounds
and genetic approaches have advanced
from preclinical work with animal models
to clinical trials with HD patients.
The chart is updated each time a
potential new treatment is identified or
developed and each time a new clinical
trial begins.
During this past year, the first
drug to successfully emerge from the
pipeline, Tetrabenazine, was finally approved for
the treatment of chorea. This is the first drug
specifically approved for people with Huntington’s
Disease by the FDA.
As more potential therapies worked their
way towards Clinical Trials, HDSA led a community
wide initiative designed to increase awareness of
the importance of clinical trials and to help recruit
potential participants. HDTrials.org is a special
website, where people can anonymously register to
learn about clinical studies and trials that take
place in their locale. When a new study or trial is
announced, members of HDTrials.org will receive
an email notification, describing the objective of
the trial, the basic qualifications to participate and
contact information for those interested in learning
more and volunteering to participate.
Basic research conducted by HDSA
Coalition for the Cure scientists continues to
identify new targets for the development of
treatments as well as biomarkers which can be
used to more precisely measure disease onset and
progression, thereby shortening clinical trials and
providing guidance as to when treatments
should be given to people with the mutated gene.
In addition, as key questions are being
answered about what goes wrong in Huntington’s
Disease, we are seeing new interest from
pharmaceutical companies in developing
treatments. This has resulted in the development
of high throughput assays to screen for
compounds that rescue the dysregulation of gene
transcription and for compounds that enhance
protein degradation and clearance.
With several compounds now in final,
Phase III trials, there is a strong possibility that
Huntington’s Disease will have its first disease
modifying treatment within the next few years. In
addition, we expect new and more efficient drugs
to be entering the research pipeline as a result of
the identification of new targets based on critical
basic research by HDSA Coalition for the Cure
scientists and other leading researchers, and
through our partnership with CHDI.
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