What is the primary treatment for amyotrophic lateral sclerosis (ALS)?

Primary Treatment for Amyotrophic Lateral Sclerosis (ALS)

The primary treatment for amyotrophic lateral sclerosis (ALS) consists of riluzole and edaravone, with riluzole being the first-line medication that has demonstrated a modest but significant prolongation of survival by approximately 2-3 months. 1, 2

FDA-Approved Medications

Riluzole

• Riluzole 100 mg/day is the standard dose that provides the best benefit-to-risk ratio, decreasing the risk of death by approximately 35% compared to placebo 3
• Prolongs survival by about 2-3 months and increases the probability of surviving one year by approximately 9% (from 57% in placebo to 66% in the riluzole group) 1
• Most effective when initiated early in the disease course, with greater benefits observed in patients with bulbar-onset ALS compared to limb-onset ALS 2
• Common adverse effects include asthenia, dizziness, gastrointestinal disorders, and elevated liver enzyme levels 4

Edaravone

• Administered as an intravenous infusion of 60 mg given over a 60-minute period according to a specific schedule:
  • Initial treatment cycle: daily dosing for 14 days, followed by a 14-day drug-free period
  • Subsequent treatment cycles: daily dosing for 10 days out of 14-day periods, followed by 14-day drug-free periods 5
• Significantly slows the decline in ALSFRS-R scores (ALS Functional Rating Scale-Revised) compared to placebo 5
• Most beneficial in patients who still have relatively preserved function (ALSFRS-R scores ≥2 on each item and FVC ≥80%) 5

Supportive Care Interventions

Respiratory Support

• Non-invasive ventilation (NIV) significantly improves survival in ALS patients and should be implemented when respiratory insufficiency develops 6
• Combined therapy with both NIV and riluzole provides greater survival benefit (16.61 months) compared to either therapy alone (NIV: 13.45 months; riluzole: 11.19 months) or supportive care only (10.69 months) 6

Nutritional Management

• Regular nutritional status assessment (BMI, weight loss) every 3 months to detect early malnutrition 7
• For patients with dysphagia:
  • Adapt food texture (soft, semisolid, or semiliquid) to facilitate swallowing 8
  • Implement postural maneuvers, particularly chin-tuck posture 8
  • Use thicker liquids and semisolid foods with high water content instead of thin liquids 8
• Enteral nutrition via feeding tubes (preferably gastrostomy) when oral intake becomes insufficient 7

Multidisciplinary Approach

• A palliative care approach should be adopted from the time of diagnosis, with early referral to palliative services 7
• Low to moderate physical activity should be maintained as long as it doesn't worsen the patient's condition 8
• Regular assessment of swallowing function through videofluoroscopy (VFS) or fiberoptic endoscopic evaluation of swallowing (FEES) to guide interventions 7, 8

Common Pitfalls and Considerations

• Delaying riluzole treatment reduces its efficacy - initiate as soon as diagnosis is confirmed 2
• Riluzole's beneficial effect may be transient and more pronounced in the first 12 months of treatment 2
• Elderly patients (>70 years) may particularly benefit from riluzole treatment with an 8-month longer median survival time compared to untreated elderly patients 2
• Edaravone is most effective in patients with early-stage disease who still have good functional status 5
• Despite limited evidence for efficacy of some nutritional interventions, they remain crucial for maintaining quality of life and preventing complications 8