As
many of you already know, the MDA - Wings Over Wall Street event was a
magnificent success! On behalf of the Eleanor and Lou Gehrig MDA/ALS Research
Center and Columbia University, I would like to express my sincere gratitude
to Michael Beier, the driving force behind this fund raiser for ALS research,
to Toni Diamond and her husband Warren Schiffer who began this event in
2001, and furthermore to the organizing committee and MDA, who executed
the evening -- an unprecedented success in ALS fundraising.
I am also proud and sincerely delighted to know that many of our patients
from Columbia took leading roles in this incredible event by raising individual
funds and rallying friends to support the event. Columbia basic scientists
have already begun new and exciting research ALS projects with the support
of the Wings Over Wall Street funds: Dr. Makiko Nagai, the recipient of
David A. Gardner MDA Neuromuscular Research Fellow has begun a new embryonic
stem cell research under the direction of Dr. Thomas Jessell, the world-renowned
neuroscientist in motor neuron and stem cell biology, and Dr. Serge Przedborski,
one of the top researchers in ALS and neurodegeneration. Dr. William Dauer,
Assistant Professor of Neurology, is investigating the effects of the
disease gene when it is turned on (causing the disease) and turned off
(halting the disease process ). This is a novel approach - testing the
effects of the SOD1 gene in ALS mice. Dr. Dauer is now in full swing since
the Wings support has become available. This is so important because if
we are able to control the gene expression (turning it on and turning
off), we should be able to develop a novel therapeutic approach to ALS.
Additionally, under the direction of Dr. Przedborski, Dr. Hitoshi Kikuchi
is investigating endoplasmic reticulum stress in ALS mice and rats. This
is a newly recognized pathway that leads to motor neuron death. Endoplasmic
reticulum is a protein factory where the particular shape of the protein
is formed. When misfolding takes place, abnormal proteins must be expeditiously
removed, otherwise, they can cause serious congestion, resulting in endoplasmic
stress leading to cell death. There are a series of proteins involved
in cleaning up those bad proteins. Dr. Kikuchi and Dr. Przedborski are
therefore analyzing how much stress occurs during this process and subsequently,
how cell death is induced. If we know more about these mechanisms, new
therapies can be introduced. Other investigators in Dr. Przedborski’s
laboratory continue to investigate mechanisms of inflammation surrounding
the degeneration of motor neurons. Their goal is to find out how to reduce
this inflammation process, which may defer the cell death process and
effect a change in ALS.
We have a new collaboration with Dr. M. Flint Beal, Professor and Chairman
of Department of Neurology at Weil Cornell Medical School. He is a world
expert in neuro-generation and oxidative stress. He is researching biochemical
markers for ALS. These can be small molecules in the blood or cerebrospinal
fluid. The identification of a reliable disease marker is essential for
the accurate diagnosis of the disease and for the measurement of the disease
progression, as well as changes that may occur with new treatments.
We are also preparing a new genetic-environmental epidemiology study
that is focused on finding other potential causes for this disease outside
of motor neurons. Most investigators believe that ALS is caused by an
unusual interaction between genetic makeup and environmental factors.
We need to unravel these factors in order to solve this disease. Dr. Robert
Brown, Professor of Neurology at Massachusetts General, and Dr. Teepu
Siddique at North Western will be collaborating with us on this project.
These are only a few examples of what we at Columbia have been able to
do as a direct result of last year’s Wing of Hope and this year’s
Wings Over Wall Street support. Not only has the Wings’ gifts enhanced
our research at Columbia University, but it has also served as a source
of motivation for more research and faster discovery of the cause and
treatment of this disease throughout the world.
I send you all my best during this hope filled Holiday Season!
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