Download Aug - 2012 - ALS Support Group Website

ALS Support Group of NW WI
A Gathering of Individuals Touched by ALS
Share Joy, Sorrow, Laughter, Tears, and Hope.
Receiving a diagnosis of ALS is challenging and can be very overwhelming.
The ALS support group provides a safe place where patients, families, friends, and caregivers
Gather to share information, support, and resources with others who understand.
Second Thursday of each month, 3:00pm – 5:00pm At Chippewa Valley Bible Church in Chippewa Falls
Support Group Meeting Notes:
8-9-12 meeting attended by ten people. We spent time looking over some of
this year’s walk & wheel-a-thon photos. We discussed this year’s turnout,
money raised & potential sponsors for next year’s fundraiser. Those who
desired shared their stories about ALS, which led to several different aspects
& topics of AlS discussed. We also discussed the research being done in MN on
Familiar AlS & research being done by the Military who are twice as likely to
get ALS as those not in the military.
Misc. articles provided by ALS Association Connections Newsletter.
Research Update
Anti-SOD1 Immunization Delays Onset, Increases Lifespan
in ALS Mice
Anti-SOD1 Immunization Delays Onset, Increases Lifespan in ALS Mice
Immunizing ALS mice against the mutant SOD1 protein delays disease onset and increases lifespan,
according to research funded by The ALS Association and published this week in Journal of
Neuroscience. “This study supports previous data demonstrating the potential of immunization as a
treatment strategy,” said ALS Association Chief Scientist Lucie Bruijn, Ph.D., “and furthermore
describes the exact binding domain of the antibody on the mutant protein.” The study, led by Janice
Robertson, Ph.D. at the University of Toronto, Canada, provides crucial insight into the mechanism
through which mutant SOD1 causes disease.
Previous research has shown that immunization was possible and could provide protection in ALS
mice, but the precise target on the surface of the SOD1 protein was unknown. In this study, the
authors used an antibody designed to bind to the surface linking two SOD1 molecules (monomers) into
a single protein (dimer). Normally, that surface is hidden between the two monomers. A feature of
most, if not all, SOD1 mutations is that the linking surface is exposed. It is thought that this may
increase the likelihood that SOD1 will aggregate into protein clumps, and those aggregates are
thought to be toxic to motor neurons or other components of the nervous system. When antibody binds
to this surface, it is a signal for the body’s immune system to clear the protein, reducing the likelihood
of aggregation.
In this study, mice were injected with a short protein chain (peptide) similar to the linking surface,
causing the immune system to make antibodies to it. (This injection strategy is similar to the
immunization process used for polio and other infectious diseases.) Injections occurred weekly from 6
weeks to 5 months of age. Immunization delayed disease onset by about 26 days and lifespan by
about 40 days in a less severe mouse model; in a more severe model, benefits were less. Treatment
reduced accumulation of misfolded and aggregated protein in the spinal cord and induced a less
inflammatory and more protective immune response.
“We are optimistic that now we have established monomer/misfolded SOD1 as a clear therapeutic
target for ALS, the immunotherapy approach we describe here is only the first step to accelerating the
pace toward providing an effective treatment for patients,” commented Dr. Robertson. “In addition to
this approach, we are searching for small molecules that will ameliorate the toxicity of
monomer/misfolded SOD1 in vivo". Because misfolded SOD1 may also occur in sporadic ALS, it is
possible that immunization may also benefit forms of the disease not caused by mutation of SOD1.
Ask the Doc: by Edward Kasarskis, M.D., Ph.D
Edward Kasarskis, M.D., Ph.D. is Director of the multidisciplinary ALS Center at the University of
Kentucky Neuroscience Center in Lexington, Kentucky, professor in the Department of Neurology at
the University of Kentucky, and Chief of Neurology at the VA Medical Center in
Lexington KY.
Q: What would you say are the biggest payoffs in ALS treatment? What are we reasonably able to
expect might be the best and most likely outcome of continued research?
A: That’s a very interesting question. Most people simply ask, “When will we have a cure for ALS?”
What people probably mean when they pose that question is “When will I be restored back to my
previous level of health?” But many ALS patients tell me that they would be very pleased if their
disease progression would be halted where it is.
At least in the near future, the "perfect" ALS drug would stop the ongoing degeneration and death of
motor neurons in the spinal cord and brain, and therefore prevent any further progression of
weakness.
Rilutek, of course, attempts to do that, but doesn’t go far enough. The gold medal for ALS drug
development to borrow a term from the Olympics starting later this month in London would go to a drug
that would stop degeneration in its tracks and prevent the disease from progressing any further. This
would be a high value outcome, for sure.
To understand how this could come about, we need to dig a little deeper into the disease process.
What’s interesting is that, early on, apparently healthy motor neurons try to fill in for the cells lost in the
ALS process. We use this fact when trying to diagnose ALS in a patient. By this I mean that the
neurologist doing the EMG test looks for larger "motor units" (sorry for the technical term but this refers
to an individual spinal motor neuron and the number of muscle fibers that the single motor neuron
connects to). This indicates that surviving, healthy motor neurons are essentially trying to “cover up”
for the lost ones that are dead to ALS.
This is the process of "denervation" of muscle (loss of nerve connection to muscle) and "reinnervation" (the reconnection of muscle fibers to one of the remaining motor neurons). It’s a sign that
all ALS motor neurons don't die off at once. This important fact offers hope that a drug could be
developed to arrest the disease process in the spinal cord and halt progression of weakness. This also
suggests that after such a drug stops the degeneration and death of motor neurons, it would also allow
the reconnection of nerve to muscle and stabilization of muscle power.
The patient would recognize that the disease progression stopped. Imagine such a drug could be
developed and available. The ALS patient would still have to live with some amount of permanent
muscle weakness but the weakness would not progress further. Patients tell me that they would be
able to handle this situation.
How could such an amazing drug be developed? The answer will probably involve finding a biomarker
or biological signal that can identify the presence of ALS and measure its progression. Then you could
take the drug and determine if the drug actually slows down, or stops the illness. Of course, the
ultimate Holy Grail for ALS would be to be able to either restore/replace lost motor neurons using stem
cells, or to prevent the disease altogether, perhaps through a vaccine. That accomplishment, at this
point in time, is still quite far off into the future. The answer will only come with more research.
Please let us know if you no longer desire to receive a copy of the ALS Monthly Report
as we will gladly remove your name from the list. Thank you.
Take good care.
Julie Chamberlain, LPN
ALSNWWI Patient Services Outreach
715.271.7257
alsnwwi@gmailcom