What is Amyotrophic Lateral Sclerosis (ALS)?

From the Guidelines

Amyotrophic lateral sclerosis (ALS) patients should receive multidisciplinary care, including physical therapy, occupational therapy, speech therapy, respiratory support, and nutritional management, to improve quality of life and survival, as recommended by the 2024 National Academies of Sciences, Engineering, and Medicine report 1.

Key Recommendations

• Early intervention with a neurologist specializing in ALS and connection to support services like the ALS Association can significantly improve quality of life throughout the disease course.
• The FDA-approved medications for ALS include riluzole (50 mg twice daily) and edaravone (60 mg daily for 14 days, followed by 14-day drug-free periods), which may extend survival and slow disease progression.
• Supportive care is essential and includes symptom management medications such as baclofen or tizanidine for spasticity, quinine or carbamazepine for cramps, and anticholinergics for excessive saliva.
• Nutritional management is critical, and patients should be advised to fractionate and enrich their meals with energy or deficient nutrients, and oral nutritional supplementation should be recommended if weight loss progresses 1.
• Gastrostomy feeding is recommended to provide long-term enteral nutrition therapy for patients with ALS with severe dysphagia, and the timing of gastrostomy insertion should be based on individual patient needs and prognosis 1.

Quality of Life and Survival

• The quality of life for ALS patients can be improved with multidisciplinary care, including physical therapy, occupational therapy, speech therapy, respiratory support, and nutritional management.
• Survival can be extended with the use of FDA-approved medications and supportive care, and early intervention with a neurologist specializing in ALS can significantly improve quality of life throughout the disease course.
• Nutritional status is a prognostic factor for survival in ALS patients, and malnutrition, weight loss, and BMI loss are associated with increased risk of death 1.

From the Research

Overview of Amyotrophic Lateral Sclerosis (ALS)

• ALS is a progressive disease with no effective treatment, characterized by progressive motor symptoms such as muscle weakness, muscle atrophy, and spasticity 2
• The disease course and progression are highly variable, with symptom severity and rate of progression determined by the ALS Functional Scale (ALSFRS-R) 2

Treatment Options for ALS

• Riluzole is the only disease-modifying drug approved for the treatment of ALS, which has been demonstrated to extend survival 3, 4, 5
• Riluzole has been shown to slow the course of the disease and extend survival in ALS patients, with a significant inverse dose response in risk of death 3
• The most common adverse reactions to riluzole are asthenia, dizziness, gastrointestinal disorders, and rises in liver enzyme activities 3, 4

Safety and Tolerability of Riluzole

• The overall tolerability of riluzole is good, but the drug can cause liver injury, with elevations of transaminases observed in 10-15% of patients 4, 6
• Concurrent use of CYP1A2 inhibitors and history of smoking are independent risk factors for liver injury in patients receiving riluzole 6
• Strict monitoring of liver enzymes is recommended in patients with ALS taking riluzole, and treatment is contraindicated in subjects with elevated transaminases before the start of treatment 4

Real-World Evidence and Survival Times

• Real-world evidence suggests that riluzole therapy may afford much greater extension of survival, with improvements in median survival times of more than 19 months reported in the overall ALS patient population 5
• Riluzole typically extends survival by 2-3 months and increases the chance of an additional year of survival by ~9% in randomized controlled trials 5