ALS

Jeremy Goldwasser
ALS (Lou Gehrig's Disease)

Hello, my name is Jeremy Goldwasser. This year in science class, we are doing
a project called the Take Action Project (TAP). In this project, we choose a
problem in the world that relates to 7th grade science. We research it, and try
to do something about it. The problem that I chose was Amyotrophic Lateral
Sclerosis (ALS), or Lou Gehrig's Disease. The reason that I chose this was
because my dad's friend's brother Zevi Kahn has it. When I heard what the
horrifying symptoms of ALS were, I immediately decided to choose it. It
seems like a terrible disease, and it is awful that there is no known cure.

Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative neuromuscular
disease. It strikes about 1 or 2 out of 100,000 people, and 5,000 people are
diagnosed annually in the US. The disease was brought to the public's
attention when the Yankees baseball star Lou Gehrig unconditionally retired
in 1939. He had ALS, and since there was little medical knowledge at the time,
he died quickly. This tragedy marked the beginning of the extensive research
for ALS. ALS has also claimed the lives of Catfish Hunter, Dmitri Shostakovich,
Jon Stone, Jacob Javitz, Huddie Ledbetter, Morrie Schwartz, Charles Mingus,
and Mao Tse Tung.




Lou Gehrig


Dmitri Shostakovich

Yankees Baseball Player


Russian Composer

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Jeremy Goldwasser




Jon Stone



Mao Tse Tung
Writer/Producer/Director of Sesame Street
Leader of the Chinese Revolution

The symptoms of ALS are horrible. A random person will start to feel
weakness in some muscles. After much testing, they will be diagnosed with
ALS. From there, it is all downhill. There is no cure, and scientists are not
close to finding one. 80% of people die within 4 years, and 90% die within 10.
Common symptoms include difficulty eating, speaking, walking, swallowing,
moving and breathing; these get progressively worse until death. Usually a
person dies because their respiratory muscles are not strong enough, and the
person cannot breathe.

Scientists do not know what stimulates ALS, but they do know what happens
internally. In a patient (typically aged 4565), nerve cells called motor
neurons start dying at an increasing rate. A motor neuron is a cell that
transfers messages like voluntary movements through the nervous system to
muscles. When random motor neurons start deteriorating at an alarming rate,
the connection between the brain and the affected muscles gets increasingly
worse. As a result, the muscles get weaker and begin to atrophy.

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Jeremy Goldwasser


It may seem like there is little hope for people with ALS. There is no known
cause or cure, and our limited medical knowledge doesn't tell us much. This
chart shows us how long someone with ALS is likely to live after diagnosis.

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So what progress has been made? In 1995 the drug Riluzole was approved.
Riluzole prolongs the period of time the patient can breathe on their own
without the assistance of a ventilator by 35 months. The medicine decreases
glutamate stimulation on the damaged motor neuron cells; glutamate is an
essential neurotransmitter, but if there is too much it can damage the motor
neurons. Some nonmedical improvements include braces, walkers, and
wheelchairs. These make life much easier for the patientask Stephen
Hawking.

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Jeremy Goldwasser



Although the economic crisis has made funding for research hard, scientists
continue to study ALS. In 1993 a big discovery was made. Mutations in the
gene that produces the enzyme superoxide dismutase (SOD1) were present in
about 20% of familial ALS patients. Even though familial cases are only about
1 in 100 cases, finding any sort of commonality between patients may be a
key step to finding what causes ALS. There have been some other mutations
that have been discovered in patients; the next step is finding a link to how
they are associated with ALS--there is no clear connection. Scientists believe
that a number of unspecified factors may stimulate ALS, because after much
searching there is nothing all patients have in common.

On May 29 I contacted Dallas Forshew at the California Pacific Medical Center
to learn what researchers are doing there for ALS. Forshew is the Manager of
Clinical Research at the Forbes Norris MDA/ALS Research Center at CPMC. I
find it interesting that right here, right now, researchers are trying to make a
difference for ALS. Dallas has posted videos on YouTube describing what
scientists at CPMC are doing. Another thing you could do is browse the CPMC
website. These cover topics ranging from new medications to suspected
causes to respiratory machines. What I found was quite interesting. I have
trouble understanding these concepts, but I will do my best to explain.

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Jeremy Goldwasser

Even though they believe ALS may have more than one cause, researchers
are trying to find possible causes; some theories are oxidative stress,
excessive glutamine, and mitochondrial abnormalities. In oxidative stress,
extra free radicals damage cells, and no enzymes (like SOD1) will soak them
up. As stated before, glutamine is a necessary chemical messenger, but if
there is too much, it can damage and kill motor neurons. The mitochondria
idea is related to this. If a motor neuron's mitochondria is not functioning
properly, then the cell is energydeficient. It will then overload on glutamine,
which kills the cell.

At CPMC, scientists are also developing and testing drugs. Two that they have
made are called NP001 and Dexpramipexole. NP001 lessens
neuroinflammation, and it has been very effective in trials. Dexpraminpexole
slows the disease progress by strengthening the muscles' ability to move.
They are also having trials on other drugs, such as lithium, KNS760704,
Talampanel, Ceftriaxone, and SB509. These new drugs have produced
positive to ALS patients, and CPMC is doing more tests.

The team of researchers at the Forbes Norris Center is a wonderful,
determined group. Dr. Nancy M Lee, PhD, says, "Our goal is to help ALS
patients lead independent, productive lives. Even in the absence of an
immediate cure, preventing progression of the disease beyond the point
where patients can no longer take care of themselves would represent an
enormous medical breakthrough." Here are the links to a page on the CPMC
website about ALS, and the first of Dallas Forshew's videos:
http://www.cpmc.org/professionals/research/currents/2003currentsals.html

www.youtube.com/watch?v=0tS4oTKRYfA

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Jeremy Goldwasser
Thank you so much for your time. I hope that I have informed you about this
tragic disease. If you have some additional questions, feel free to contact me.
Please consider donating to any of these wonderful organizations from all
across the country:

ALS Association
http://www.alsa.org/

Forbes Norris ALS Research Center
http://www.cpmc.org/services/als/about/default.html

ALS Family Charitable Foundation
http://www.alsfamily.org/

Project ALS--Finding and Funding a Cure for ALS
http://www.projectals.org/

ALS Therapy Development Institute
http://www.disaboom.com/organizations/4345

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