What is Amyotrophic Lateral Sclerosis (ALS)?

From the Guidelines

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, leading to muscle weakness and eventual paralysis, with the most recent and highest quality study suggesting that a multidisciplinary approach to care is crucial in improving quality of life and survival rates 1.

Definition and Overview

ALS is a condition that causes motor neurons to gradually deteriorate and die, preventing them from sending messages to muscles, which then weaken, twitch, and waste away.

• ALS primarily affects voluntary muscles used for activities like walking, talking, chewing, and breathing.
• Most patients with ALS die from respiratory failure within 3-5 years of symptom onset, though some may live 10 years or longer.
• The disease typically begins with muscle weakness, stiffness, or twitching in a limb, slurred speech, or difficulty swallowing.

Current Treatment and Management

• Currently, there is no cure for ALS, but medications like riluzole and edaravone can modestly slow disease progression.
• Riluzole extends survival by about 2-3 months, while edaravone may slow functional decline in some patients.
• Treatment focuses on managing symptoms, preventing complications, and maintaining quality of life through a multidisciplinary approach involving neurologists, respiratory therapists, physical therapists, speech therapists, and nutritionists.
• The exact cause of ALS remains unknown for most cases, though about 5-10% are inherited genetically.

Recent Recommendations for Improving Quality of Life

• The 2024 National Academies of Sciences, Engineering, and Medicine report emphasizes the importance of equitable access to affordable multidisciplinary care, services, and caregiver support in improving quality of life for individuals with ALS 1.
• The report also recommends expanding coverage for respiratory assist devices and services, as well as improving access to clinical trials and research studies.
• A recent study published in the Annals of Neurology highlights the need for a reimagined, inclusive, and integrated multidisciplinary care and research system to address the complex needs of individuals with ALS 1.

From the FDA Drug Label

Edaravone injection is prescription medicine used to treat people with amyotrophic lateral sclerosis (ALS). The FDA drug label does not answer the question.

From the Research

Definition and Overview of ALS

• Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative motor neuron disease, resulting in the destruction and ultimate death of neurons that control muscles 2.
• ALS affects motor neurons in the brain, brainstem, and spinal cord (upper motor neurons, bulbar region of the brain, and lower motor neurons) 2.
• It is characterized by chronic degeneration of upper and lower motor neurons and finally death within 3-5 years usually because of respiratory failure 3.

Symptoms and Diagnosis

• ALS patients have an average life expectancy of 3-5 years, therefore, proper diagnosis, care, and treatment is essential in order to provide the best quality of life for these patients 2.
• Electrodiagnostic examination, specifically electromyography (EMG) and nerve conduction studies (NCS), is one of the key diagnostics of ALS 2.
• Diagnosis is determined by excluding other conditions and utilizing clinical examinations, laboratory tests, and nerve conduction/electromyography studies 4.

Treatment and Management

• Riluzole and edaravone are presently available treatments for ALS 3, 5.
• Edaravone is a novel neuroprotective agent, indicated to slow down progression of ALS 6.
• The best practices for the management of ALS include an interdisciplinary approach aimed at addressing the physical and psychological needs and desires of patients and their families and caregivers 4.
• Combined with riluzole, edaravone slows disease progression and is safe, but the effect is short-term 3.

Types and Prognosis

• There are 2 types of ALS differentiated by genetics: familial and sporadic (idiopathic) 4.
• ALS affects approximately 16,000 individuals, with a prognosis for survival of 2 to 5 years 4.
• Edaravone has been shown to slow down the loss of physical function in ALS patients by 33% as compared to placebo 6.