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The Stop ALD Foundation
October 2011 Update
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Dear Stop ALD Foundation Supporter,
 To
date, the Foundation has played a key role in finding a therapy
for ALD patients whose disease has just started to progress.
Looking forward to the fourth quarter of 2011, we will turn our
attention to two different and, for us, very significant goals:
(1) Driving forward a therapeutic approach for patients whose
disease has progressed beyond the ability of current therapies
to be effective, and (2) determining whether anti-oxidant
treatments are of benefit to help treat AMN patients.
The
gene therapy work which has been done to date works with the
patient's own stem cells and, even when successful, takes at
least 12 to 18 months to halt the disease. Because so many ALD
families experience their first diagnosis after the disease has
started to progress, their children do not have the luxury of 18
months of time before a successful therapy may be given a chance
- they need a therapy which begins to work much more quickly in
order to save these children. To address this critical need, we
will work with physicians and scientists to investigate using a
different gene therapy approach which targets brain cells and
which can go into the brain immediately and stop the disease
in a much shorter time frame. In order to progress this
approach, we have to test this theory in laboratory mice, and
our aim is to have this initiative launched in mice by year end.
If this therapy proves to be successful in mice, we will
immediately initiate the additional pre-clinical steps necessary
before the therapy would be ready to be tested in humans.
Looking to our second goal, in order to properly explore
whether various anti-oxidant compounds may be effective in
treating symptoms of AMN, the compounds will similarly need
to be tested first in mice. Toward that end, and because AMN
mice need to age before they exhibit AMN symptoms, we will work
with a lab to begin breeding and aging the mice as the
therapeutic agents are investigated and the approach is formally
defined. Our goals is to launch these activities by year end as
well.
As we pursue these two near-term primary goals, we of course
will continue to support and work with others to find additional
promising therapeutic approaches to ALD and AMN, to implement
newborn screening in as many states as will permit it, to find
ways to accelerate ongoing initiatives managed by others working
in this field, and to continue to help families faced with
managing ALD and AMN patients.
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