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Proposal Requirement 4 Example: ALS

Writer: Natasha Matta

Instagram: @natashakmatta

Email: matta.natashak@gmail.com


Current Solutions and Their Insufficiencies

Amyotrophic lateral sclerosis (ALS) is difficult to diagnose, and as such, there is no single test or marker to definitively diagnose the condition. Only through a series of tests, many of which are aimed at ruling out diseases that present themselves similarly to ALS, can a diagnosis be made. These include electromyography (EMG), nerve conduction velocity (NCV), analysis of thyroid and parathyroid hormone levels and amount of heavy metals in blood and urine samples, spinal tap, x-rays and MRIs of the brain and spinal cord, myelogram of cervical spine, muscle and nerve biopsies, and genetic testing for the familial (inherited) form of ALS.

Currently, there is no cure for ALS, but various treatments can slow the loss of muscle strength and control and make patients more comfortable and able to function independently. The United States Food and Drug Administration (FDA) approved drugs Rilutek (riluzole) and Radicava (edaravone) for treatment of the disease. Rilutek is being used in Canada, Australia, and some countries in Europe, and Radicava is available in countries such as Canada, Japan, and South Korea. Rilutek interferes with glutamate toxicity, a phenomenon which has been linked to ALS severity. Radicava combats oxidative stress, an imbalance between reactive oxygen species (free radicals) and antioxidants, which has been found to be heightened in ALS cases. In edaravone Study 19 (MCI186-19), 137 patients were randomly given either Radicava or a placebo through intravenous infusions for a period of 24 weeks, and the ALSFRS-R scale was used to measure the progression of the disease. For the ALSFRS-R scale, patients rate their ability to complete a task on a five-point scale with 0 meaning unable to complete the task and 4 meaning normal ability, and the scores for each task are totaled with 0 being the worst and 48 being the best. After the six months, patients who received the placebo experienced an average loss of 7.5 points on the ALSFRS-R scale, and those who were given Radicava experienced an average loss of only 5 points, making a strong case that Radicava slows deterioration in ALS patients compared to the placebo.

Although they are not treatments for ALS, various medications like muscle relaxants (diazepam and baclofen) and pain medication (gabapentin) can be used to manage symptoms such as muscle cramps, spasms, and pain, difficulty swallowing, insomnia, fatigue, and lack of energy. Physical therapy, use of a brace, cane, walker or wheelchair, speech therapy, a feeding tube, and a ventilator to aid with breathing in later stages of ALS can all be helpful in managing the disease.

After diagnosis, about half of ALS patients live three years or longer, 20% live five years or longer, and 10% live ten years or longer. Current treatment options can in the best case slow down progression of muscle loss, weakness, and paralysis, giving patients 10 years to live. However, patients will still have difficulty with basic bodily functions such as eating, drinking, moving, and breathing up until they pass away. ALS takes a heavy physical and mental toll on the patient and emotional toll on their family, friends, and healthcare workers that watch them suffer as their muscles waste away as well as incurs costly medical bills. New treatments need to address the core of the issue of ALS: death of motor neurons. Only then, could an eventual cure be found, which would give ALS patients longer, happier, and more productive lives.


Analysis

The author of this piece methodically discusses each of the current treatment options for amyotrophic lateral sclerosis (ALS) ranging from medications to physical therapy, addressing the methods, reasoning behind the treatment, and drawbacks in detail. She successfully convinced the reader that although some treatments were effective in lessening symptoms like muscle cramps, spasms and pain, fatigue, and insomnia and treatments, treatments need to be created to better address difficulty with basic functions like walking, eating, drinking, and breathing, ultimately patients, so that patients will not have to undergo a long and painful process of feeling their muscles waste away, constantly having to rely on others, and heavy mental, emotional and physical toll on them and their families. The author used reputable sources from a combination of hospital and university websites and scholarly sources like science journals and wrote professionally, using third person, correct spelling, grammar, and formatting.


Bibliography

What is ALS? The ALS Association. https://www.als.org/understanding-als/what-is-als

Stages of ALS. ALS Association Texas Chapter. https://www.alstexas.org/understanding-als/stages/#:~:text=As%20the%20disease%20progresses%20to,%2C%20eat%2C%20or%20drink%20normally

Stages of ALS. Muscular Dystrophy Association. https://www.mda.org/disease/amyotrophic-lateral-sclerosis/signs-and-symptoms/stages-of-als

Amyotrophic Lateral Sclerosis (ALS) Fact Sheet. National Institute of Neurological Disorders and Track. https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-Lateral-Sclerosis-ALS-Fact-Sheet

ALS Treatment. ALS News Today. https://alsnewstoday.com/als-treatment/

Glutamate. ALS Association. http://web.alsa.org/site/PageServer?pagename=ALSA_Glutamate

Kegel, Magdalena. ALS Severity, Duration May Be Linked to Glutamate Toxicity. ALS News Today. https://alsnewstoday.com/2016/01/15/n-acetyl-aspartate-to-glutamate-ratio-correlates-with-als-disease-duration/

Betteridge, D. J. What is oxidative stress?. Metabolism. https://pubmed.ncbi.nlm.nih.gov/10693912/

Martins, Ines. Radicava Effective in Slowing Progression Across Year of Use, Trial Data Show. ALS News Today. https://alsnewstoday.com/2020/03/30/radicava-effective-slowing-als-progression-across-1-year-use-patients-trial-data/

Pattee, Gary, et al. Post Hoc Analysis of Edaravone Study 19: Efficacy in Bulbar Onset ALS Patients With and Without Reduced Pulmonary Function. Muscular Dystrophy Association. https://www.mdaconference.org/node/942


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