Amyotrophic Lateral Sclerosis, commonly referred to as ALS, is a disease that affects neurons involved in voluntary muscle movement. Voluntary muscle movement features any movement not triggered by a reflex or internal mechanisms such as heart rate and blood pressure. The word “Amyotrophic Lateral Sclerosis” can be broken up into two meanings: muscle atrophy and hardness of the lateral columns of the spinal cord (Rowland & Shneider, 2001). Symptoms typically begin with hand or leg weakness, followed by slurred speech and dysphagia (difficulty swallowing). Eventually, the disease worsens into significant weakness in muscle activity and for some, eventual paralysis (Rowland & Shneider, 2001).
ALS is a heartbreaking illness to live with, particularly since it has the ability to affect people as young as 30. Early diagnosis is vital to prevention of the progression of the disease (Priest, 2015)). In recent years, medications such as the drug Rilutek, have been found to delay this progression. Rilutek is a glutamate blocker. Glutamate is an excitatory neurotransmitter in the central nervous system and is vital in the firing of action potentials in a functioning neuron, which signals for the muscles to constrict or relax, resulting in normal bodily movement when working properly. However, excessive stimulation of glutamate receptors has been found to cause neuronal death, resulting in muscles no longer being able to move, leading to the paralysis patients with ALS suffer from (Foran & Trotti, 2009). By Rilutek functioning as a glutamate blocker, it aims to prevent abnormal glutamate activity that negatively impacts muscle movement. As of early 2020, Rilutek was the only medication that has been found to have a long-term effect on ALS progression.
Fortunately, as medicine continues to advance, so do new forms of treatment for existing diseases. Recent studies have begun studying AMX0035 (Relyvrio). Relyvrio is a combination of sodium phenylbutyrate and taurusodiol aimed to prevent cellular stress and stop the progression of the disease before its progression. This medication was found to result in a slower functional decline by extending life an average of 10 months for patients treated with it (Paganoni et al, 2020). It was accepted by the FDA in September of 2022 and is awaiting more clinical trials to assess long-term symptoms (als.org).
Being such a devastating illness, it is crucial that research involved in ALS continues to be funded for future studies to test new, hopefully, more effective treatments, and those with ALS have options to assist in their care and well-being. It is treated as a necessity to the care and well-being of those who live with ALS.
AMX0035 (RELYVRIO). The ALS Association. (n.d.). Retrieved October 6, 2022, from https://www.als.org/navigating-als/living-with-als/fda-approv ed-drugs/amx0035#what-is
Foran, E., & Trotti, D. (2009). Glutamate transporters and the excitotoxic path to motor neuron degeneration in amyotrophic lateral sclerosis. Antioxidants & Redox Signaling, 11(7), 1587–1602. https://doi.org/10.1089/ars.2009.2444
Paganoni, S., Macklin, E. A., Hendrix, S., Berry, J. D., Elliott, M. A., Maiser, S., Karam, C., Caress, J. B., Owegi, M. A., Quick, A., Wymer, J., Goutman, S. A., Heitzman, D., Heiman-Patterson, T., Jackson, C. E., Quinn, C., Rothstein, J. D., Kasarskis, E. J., Katz, J., … Cudkowicz, M. E. (2020). Trial of sodium phenylbutyrate–Taurursodiol for amyotrophic lateral sclerosis. New England Journal of Medicine, 383(10), 919–930. https://doi.org/10.1056/nejmoa1916945
Priest, A. (2015). Living with ALS. Canadian Medical Association Journal, 187(3), 210–211. https://doi.org/10.1503/cmaj.141517
Rowland, L. P., & Shneider, N. A. (2001). Amyotrophic lateral sclerosis. New England Journal of Medicine, 344(22), 1688–1700. https://doi.org/10.1056/nejm200105313442207