What is the primary treatment for Amyotrophic Lateral Sclerosis (ALS)?

Primary Treatment for Amyotrophic Lateral Sclerosis (ALS)

Riluzole 50 mg twice daily (taken at least 1 hour before or 2 hours after meals) is the primary disease-modifying treatment for ALS, as it is FDA-approved and has been shown to extend survival by decreasing the risk of death or tracheostomy by 35% compared to placebo. 1, 2

Disease-Modifying Pharmacotherapy

First-Line Treatment: Riluzole

• Riluzole is the established first-line disease-modifying therapy for ALS, with FDA approval since 1995 and the most extensive evidence base 1
• The standard dosing is 50 mg orally twice daily, administered at least 1 hour before or 2 hours after meals to optimize absorption 1
• In the confirmatory dose-ranging study of 959 ALS patients, riluzole 100 mg/day demonstrated a 35% decreased risk of death after adjustment for baseline prognostic factors (p = 0.002) over 18 months 2
• Tracheostomy-free survival rates at 18 months were 56.8% with riluzole 100 mg/day versus 50.4% with placebo 2

Monitoring Requirements for Riluzole

• Measure serum aminotransferases (liver enzymes) before initiating treatment and regularly during therapy 1
• Riluzole is contraindicated in patients with baseline serum aminotransferases greater than 5 times the upper limit of normal 1
• Discontinue riluzole if evidence of liver dysfunction develops during treatment 1
• Advise patients to report any febrile illness due to potential neutropenia risk 1
• Monitor for interstitial lung disease and discontinue if it develops 1

Additional FDA-Approved Options

• Edaravone (60 mg IV infusion over 60 minutes in 28-day cycles) is approved as a second disease-modifying agent, shown to slow loss of physical function by 33% compared to placebo 3
• Sodium phenylbutyrate and taurursodiol (PB/TURSO) represents a third FDA-approved option for slowing ALS progression 4
• A fourth therapy has received accelerated FDA approval contingent upon confirmatory trial verification 4

Multidisciplinary Supportive Care Framework

Core Principle

Equitable access to affordable multidisciplinary care is essential from diagnosis onward, as supportive care is known to improve both quantity and quality of life in ALS 5

Nutritional Management Strategy

• Assess nutritional status (BMI, weight loss) every 3 months to detect early malnutrition 6
• For patients with muscular fatigue and prolonged mealtimes, fractionate and enrich meals with energy or deficient nutrients 7
• For moderate dysphagia, adapt food texture (soft, semisolid, or semiliquid) to facilitate swallowing and prevent aspiration 7
• Implement chin-tuck posture during swallowing to protect the airway 7
• Use thicker liquids and semisolid foods with high water content (like jellified water) instead of thin liquids for patients with delayed swallowing 7
• Enteral nutrition via feeding tubes (preferably gastrostomy) is preferred over parenteral nutrition when oral intake becomes insufficient, with PEG placement considered before respiratory function significantly deteriorates 7, 6

Physical Activity Guidelines

• Recommend low to moderate physical activity as long as it doesn't worsen the patient's condition, as endurance and resistance exercises may slow disease progression and improve functionality 7
• Avoid excessive physical exertion that could lead to fatigue and symptom worsening 7

Equipment and Services Access

• All requests for equipment and services for ALS should be considered urgent and handled expeditiously (within 72 hours), as delays can result in catastrophic safety risks rather than simple inconvenience given the 2-5 year average life expectancy from onset 5

Palliative Care Integration

A palliative care approach should be adopted from the time of diagnosis, with early referral to palliative services to establish relationships with staff and address end-of-life issues 6

Common Pitfalls and Caveats

Riluzole-Specific Concerns

• The most frequent adverse events are asthenia (18% of patients) and nausea (15% of patients) 8
• Serum alanine aminotransferase elevations more than three times the upper limit of normal occur in 10-15% of patients, requiring strict liver enzyme monitoring 8
• Overall tolerability is good, and riluzole can be used in all ALS patients except those with elevated transaminases or active liver disease 8

Dysphagia Management

• Dysphagia management techniques effective for other conditions may not work in ALS due to the specific pathophysiology of progressive muscle atrophy and fatigue 7
• Use videofluoroscopy (VFS) or fiberoptic endoscopic evaluation of swallowing (FEES) for instrumental assessment to guide texture modifications 7

Realistic Expectations

• There is currently no cure for ALS, and disease-modifying treatments provide modest survival benefits rather than halting progression 5
• Riluzole does not significantly improve functional assessments, though it extends survival 2