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Running Head: QUALITY OF LIFE FOR ALS PATIENTS

Treatments to Help Improve the Quality of Life for ALS

Patients
Nicole Katyryniuk
Liberty Senior Inquiry

QUALITY OF LIFE FOR ALS PATIENTS

So I close in saying that I may have had a tough break, but I have an
awful lot to live for (Gehrig, 1939). Lou Gehrig, one of the most well-known
figures in baseball history, was diagnosed with ALS in the year of 1939. After
Gehrigs speech, ALS was often referred to as Lou Gehrigs disease.
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive and universally
fatal neurodegenerative disorder of motor neurons in the spinal cord,
brainstem, and the motor cortex (Vucic, 2013, p 1361). There is not a cure
for ALS, but there are treatments that slow down this horrible disease.
Riluzole helping to lower the glutamate levels in patients, guanabenz has
made promises involving the extension in life span, and NIPPV (noninvasive
positive pressure ventilation) has improved patients respiratory systems. All
three of these treatments have had successful clinical trials. As of right now,
riluzole is the only drug approved by the Food and Drug Administration for
patients to take, therefore being the main treatment for ALS, guanabenz and
NIPPV are on the rise to help improve the quality of life in ALS patients.
Based on U.S. population studies, a little over 5,600 people in the
United States are diagnosed with amyotrophic lateral sclerosis (ALS) each
year, attacking people who are between the ages of 40 to 70, but there are
cases of people are younger (ALS Association, 2011). Even though these
numbers are not willingly as high as breast cancer, this disease is
nonetheless important. Only 2 out of 100,000 people get ALS and with a 50%
chance they will live to three years after being diagnosed (MDA, 2009).
Diagnosing ALS cannot be done with only a single test, patients are tested

QUALITY OF LIFE FOR ALS PATIENTS

with, electro diagnostic and nerve conduction velocity, blood and urine
studies, x-rays, myelogram of cervical spine, muscle/nerve biopsy, and a
neurological examination (ALSA, 2011). After receiving the results patients
are given a second opinion by another doctor. Throughout the disease the
symptoms from the beginning will worsen all the way until the end. ALS
symptoms include, muscle weakness, twitching and cramping of the
muscles, limb impairments, difficulty in speaking, and difficulty in breathing
and swallowing (ALSA, 2011). ALS symptoms are constantly overlooked by
patients. There are three different types of ALS such as; sporadic, familial,
and Guamanian (ALSA, 2011). Sporadic is the most common form of ALS
effecting 90 to 95% of all patients. Familial is the inherited form of ALS only
effecting approximately 5 to 10% of people. The last and final form is
Guamanian, was found in the Pacific around 1950 (ALSA, 2011). Each form of
ALS is very dangerous and in the end will be treated all the same.
Treatments for ALS are very limited in this day and age, Guanabenz
has not yet been approved by the FDA to be given to patients but has shown
successful results in clinical trials. Guanabenz is an alpha agonist of the
alpha-2 adrenergic receptor that is used as an antihypertensive drug (Wang,
Popko, Tixier, & Roos, 2014). This treatment is used to help maintain a
healthy blood pressure being that hypertension is one of the main killers for
ALS patients. Wang states, that Guanabenz enhances the unfold protein
reaction, improves mutant SOD-1 induced in ALS (Wang, 2014). Wang
believes, that in the drug Guanabenz, the treatment can help maintain the

QUALITY OF LIFE FOR ALS PATIENTS

mutant SOD-1 in patients instead of having ALS destroy it. This treatment
helps expand the lifespan of patients, delays the symptoms, and improves
motor performance and motor neurons (Wang, 2014). According to Wang,
approximately 20% of familial cases are caused by mutant superoxide
dismutase type 1 (Wang, 2014). Basically, Wang is stating that the inherited
form of ALS that only 10% get is caused by this mutant superoxide
dismutase type 1, in which Guanabenz does help towards. In the
Neurobiology of Disease Journal, Wang (2014) found that, Guanabenz
ameliorates disease in transgenic mice, and therefore is a candidate for the
treatment of ALS patients (Wang, 2014). In other words, Wang believes that
this drug could help and save some ALS patients from going through this
disease without any help or a chance. In recent studies in the Neuroscience
journal (2014) Guanabenz has been found to inhibit dephosphorylation and
protect wild type ER-stressed cells from dying (Jiang, 2014). Jiangs point is
that Guanabenz can effect countless more areas in which riluzole, the only
approved drug, cannot.
It is known, however, that military veterans, particularly those
deployed during the Gulf War, are approximately twice as likely to develop
ALS (ALSA, 2011). The ALS Association states, the veterans from that era
are currently the only people who are twice as likely to get ALS. In Cleveland,
at a veterans hospital, they believe that patients with ALS are likely to die
from respiratory failure than anything else (ALSA, 2014). NIPPV (noninvasive
positive pressure ventilation) has had effective benefits from this treatment

QUALITY OF LIFE FOR ALS PATIENTS

that hasnt been obtained by riluzole (McCellan, 2013). McCellan states, that
patients are dying from respiratory failure the most with this disease and
NIPPV can help give patients a better quality of life in there last stages of
their life. Studies suggest that NIPPV improves the quality of life in patients
by enhancing their energy, concentration, and physical fatigue (McCellan,
2013). McCellan point is to inform people that these veterans helped shape
our countrys history, in which we need to help make their life easier when
theyre needing our help the most. NIPPV is difficult to use for many ALS
patients; studies show documented compliance in less than 65 percent of
patients, with compliance decreasing to 25 percent in patients with
concomitant frontal temporal dementia or bulbar onset disease (McCellan,
2013). According to McCellan, he states that NIPPV is very uncomfortable for
patients to use and therefore is starting to downward fall in the percentage
of patients taking the NIPPV treatment. As NIPPV is not yet FDA approved,
technology is enhancing in the next couple years, McCellan and his team
believe NIPPV will be just as successful as riluzole in ALS patients, especially
our veterans.
Riluzole (rilutek) is currently the only drug that is registered to treat
patients with amyotrophic lateral sclerosis (Defiorio, 2012). Defiorio presents,
that riluzole is the only drug that will make an impact on a patients quality
of life who are effected with ALS. According to Defiorio, riluzole is an oral
drug in the benzothiazole class that was approved by the FDA in 1995
(Defiorio, 2012). Riluzole is currently helping to reduce damage to the motor

QUALITY OF LIFE FOR ALS PATIENTS

neurons in your brain by decreasing the release of glutamate (National

Institute of Neurological Disorders and Strokes, 2014). The NIH and Defiorio
states that with riluzole patients are releasing glutamate which will indeed
reduce the motor neurons in the brain of ALS patients and help them block
out the disease. The cost of the riluzole with patients taking 100 mg per day
in the United States is roughly $728 dollars per month with an average whole
sale price of $727.75 for 60 tablets each containing 50 mg of riluzole (Red
Book, 1996). Data suggest that the incremental cost of riluzole therapy is
around $US 12,000 per patient (Messori, 1999). With the treatment of
riluzole at an amount where patients can agree upon, making this treatment
an even better match for ALS patients. Riluzole has been shown to delay the
time of death in a mouse model for ALS, These mice express human
superoxide dismutase bearing one of the mutations found in one of the
familial forms of human ALS (Sanofi-Aventis). In other words, mice are being
injected with the disease and riluzole is indeed helping these mice models
overcome this disease. However, riluzole being the first therapy for ALS,
offers hope that the progression of ALS may one day come down to curing
this disease that has taken many lives.
With these three treatments, ALS has been targeted in many different
ways such as, taking oral prescription drugs by slowing down the progression
of the disease and giving the respiratory system when thats the one area
that kills the most. Riluzole is perhaps the strongest treatment because of
the fact that it is FDA approved and it improves the quality of life in patients

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with ALS, unlike treatments like NIPPV and Guanabenz that are not approved
by the FDA, which means that they are not given benefits or a price for
patient use yet. The ALS Association has received $88.5 million dollars in
donations to help find the cure for amyotrophic sclerosis (ALSA, 2011) from
this summers Ice Bucket Challenge. This chilly challenge gave people the
choice between dumping ice cold water on themselves and donate $10
dollars or donate $100 dollars to ALS. With this money, finding the cure to
ALS is closer and it gives the patients with the disease hope that one day this
disease will be eliminated.

References
Andreadou, E., Kapaki, E., Kokotis, P., Paraskevas, G., Katsaros, N., Libitaki, G., ...
Vassilopoulos, D.

(2014). Plasma glutamate and glycine levels in patients with

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amyotrophic lateral sclerosis: The

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Neurology and Neurosurgery, 110(3), 222-226.

ALS Untangled NO. 20: The Deanna Protocol. (2013). Amyotrophic Lateral Sclerosis
& Frontotempal

Degeneration, 14(4), 319-323.

Drug Topics Red Book. Montvale (NJ):Medical Economics, 1996

Jiang, H., Ren, M., Jiang, H., Wang, J., Zhang, J., Yin, X., Feng, H. (2014). Guanabenz
delays the onset of

disease symptoms, extends lifespan, improves motor

performance and attenuates motor

neuron loss in the SOD1 G93A mouse model

of amyotrophic lateral sclerosis. Neuroscience, 277,

132-138.

McCellan, F., Washington, M., Ruff, R., & Selkirk, S. M. (2013). Early and innovative
symptomatic care to

improve quality of life of ALS patients at Cleveland VA ALS

center. Journal of Rehabilitation Research & Development, 50(4), vii-xvi.

Steen, I. D., Berg, J. D., Buskens, E., Lindeman, E., & Van Den Berg, L. H. (2009). The
costs of amyotrophic

lateral sclerosis, according to type of care. Amyotrophic

Lateral Sclerosis, 10(1), 27-34. doi:10.1080/17482960802103131

Vucic, S., Lin, C., Chean, B. C., Murray, J., Menson, P., Krishman, A. V., & Kiernan,
M.C. (2013). Riluzole

exerts central and peripheral modulating effects in

amyotrophic lateral sclerosis. Brain: A journal

of Neurology, 136(5), 1361-

1370.
Wang, L., Popko, B., Tixier, E., & Roos, R. (2014). Guanabenz, which enhances the
unfolded protein response, ameliorates mutant SOD1-induced amyotrophic lateral
sclerosis. Neurobiology of Disease, 71, 317-324.

QUALITY OF LIFE FOR ALS PATIENTS