An email from Louise Vetter, CEO at HDSA
Truth or Fiction
HDSA and the Induced Pluripotent Stem Cell Consortium
As a friend of HDSA and our research efforts, I want to share with you exciting scientific
developments that may help further our understanding and accelerate the development of
treatments of Huntington’s disease.
Thanks to your fervent and generous support we have been able to foster and actively
participate in a collaboration with the National Institutes of Health (NIH) on a relatively new type
of stem cell – the induced pluripotent stem cell (iPSC). Unlike embryonic stem cells, iPSCs are
skin cells that have been reprogrammed through science to behave like stem cells. They can
then be cultivated to become neurons or other types of cells that are valuable to HD research.
Three years ago, the Society was inundated with calls and emails asking how stem cells could
be used to treat HD. At the time, the National Institute of Neurological Disorders and Stroke
(NINDS), a division of the NIH, was working to establish an Induced Pluripotent Stem Cell
(iPSC) Consortium that would not only investigate the potential of stem cell therapy for people
with Huntington’s disease, but would also bring HD researchers into the stem cell realm.
Notably, the Consortium would foster the type of cooperative, team-based work that HDSA
pioneered with the HDSA Coalition for the Cure.
HDSA was involved from the onset in the launch of this Consortium and remains a direct
supporter of the successful efforts which have created 46 stem cell lines with a range of CAG
repeats from 18 to 150 to date. These cells are available for any researcher to use in the
hopes of expanding our knowledge of HD and working towards treatments and a cure.
It’s already been proved that these stem cell lines can be used to create neurons that work
like natural human brain cells. This means that they can be used to expedite clinical trials and
test whether a potential therapy may be beneficial in human cells – not animal model – and, in
the future, they will also help us determine if a potential therapy is working on someone who is
not yet symptomatic.
Your donations to HDSA research have literally put a new tool in the hands of top researchers
in academia, as well as scientists from big pharmaceutical companies and biotechs which
have become active in HD research because of the opportunity provided by this Consortium.
All in less than 3 years!
So why are we sharing this exciting news with you? We wanted to let you know that HDSA
continues to drive groundbreaking HD research, such as this iPSC Consortium – a direct
result of the ongoing support we receive from friends like you.
Clearly research takes money, so I ask you to be as generous as you can. Please help us
continue to provide leadership and direction that will enable the type of innovative research
that will yield important discoveries and ensure that they make their way quickly from the lab
to those who need it the most – our loved ones.
Thanks again for all you do to bring Help for Today, Hope for Tomorrow to everyone with HD.
We will not stop until our vision of a world free of HD comes true!
Sincerely and With Thanks,
Chief Executive Officer
P.S. To make an online contribution please visit www.hdsa.org/lresearchnews
To learn more about the progress of theIPSC Consortium:
The Marker — 2nd Meeting of the IPSC Stem Cell Consortium
Patient-Derived Induced Pluripotent Stem Cells Advance Research, Could Lead to Restorative
Genetic Defect Corrected in Induced Pluripotent Stem Cells
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