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 FDA-approved disease-modifying treatment for ALS, with proven survival benefit. 1

Disease-Modifying Pharmacotherapy

First-Line Treatment

• Riluzole remains the cornerstone of ALS pharmacotherapy, demonstrating a 35% decreased risk of death compared to placebo when adjusted for baseline prognostic factors over 18 months 2
• The FDA-approved dosing is 50 mg orally twice daily, administered on an empty stomach (at least 1 hour before or 2 hours after meals) 1
• Riluzole extends tracheostomy-free survival rates to 56.8% compared to 50.4% with placebo at 18 months 2

Additional FDA-Approved Options

• Edaravone (60 mg IV over 60 minutes in 28-day cycles) is the second FDA-approved 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

Critical Monitoring Requirements

Hepatic Surveillance

• Measure serum aminotransferases before initiating riluzole and monitor regularly during treatment 1
• Riluzole is contraindicated in patients with baseline transaminase elevations greater than 5 times the upper limit of normal 1
• Discontinue riluzole if evidence of liver dysfunction develops, as 10-15% of patients experience alanine aminotransferase elevations more than 3 times the upper limit of normal 5

Hematologic Monitoring

• Advise patients to report any febrile illness, as riluzole may rarely cause neutropenia 1
• Physicians should maintain vigilance for neutropenia risk 5

Multidisciplinary Supportive Care Framework

Nutritional Management

• 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; progress to oral nutritional supplementation if weight loss continues 7
• Implement videofluoroscopy (VFS) at diagnosis to detect early dysphagia, even in asymptomatic patients 6
• For moderate dysphagia, adapt food texture (soft, semisolid, or semiliquid) and implement chin-tuck posture to protect the airway 7
• Consider enteral nutrition via gastrostomy (preferably PEG) before respiratory function significantly deteriorates 6

Physical Activity Guidelines

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

Palliative Care Integration

• Adopt a palliative care approach from the time of diagnosis, with early referral to palliative services to establish relationships and address end-of-life issues 6

Equipment and Service Access

• All requests for equipment and services for ALS patients should be considered urgent and handled expeditiously, given the rapidly progressive nature of ALS with average life expectancy of 2-5 years from onset 8
• Delays in approval and delivery can result in catastrophic safety risks rather than simple inconvenience 8

Common Adverse Effects and Management

Riluzole-Specific Concerns

• The most common adverse reactions (≥5% and greater than placebo) are asthenia (18%), nausea (15%), dizziness, decreased lung function, and abdominal pain 1, 5
• Discontinue riluzole if interstitial lung disease develops 1
• Avoid coadministration with strong to moderate CYP1A2 inhibitors (may increase adverse reactions) or inducers (may decrease efficacy) 1

Critical Pitfalls to Avoid

• Do not delay riluzole initiation while awaiting genetic testing results, as approximately 90% of ALS is sporadic without a known genetic mutation 8
• Do not use the traditional reactive reimbursement model for ALS equipment, as the rapidly progressive nature requires proactive planning 8
• Do not assume dysphagia management techniques from other conditions will work in ALS, due to the specific pathophysiology of muscle atrophy and fatigue 7