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

Primary Treatment for Amyotrophic Lateral Sclerosis (ALS)

The primary treatments for ALS are the FDA-approved disease-modifying medications riluzole and edaravone, which should be initiated as early as possible after diagnosis to slow disease progression and improve survival. 1, 2, 3

Disease-Modifying Treatments

First-Line Medications

1. Riluzole (oral)

   • FDA-approved for ALS treatment 1
   • Mechanism: Anti-excitotoxic properties
   • Dosage: 100 mg daily (50 mg twice daily)
   • Benefits: Extends survival by approximately 2-3 months 4, 5
   • Side effects: Asthenia, dizziness, gastrointestinal disorders, elevated liver enzymes 4

2. Edaravone (IV)

   • FDA-approved for ALS treatment 2
   • Mechanism: Free radical scavenger
   • Administration: Intravenous infusion
   • Benefits: Slows functional decline 6

3. Newer options

   • Sodium phenylbutyrate and taurursodiol (PB/TURSO) - recently approved 3
   • Tofersen - approved under accelerated approval for SOD1-ALS 3

Multidisciplinary Care Approach

Multidisciplinary care is essential alongside disease-modifying treatments and has been shown to improve survival and quality of life 7:

1. Respiratory management

   • Regular assessment of respiratory function
   • Non-invasive ventilation when indicated
   • Discussion about invasive ventilation options

2. Nutritional support

   • Regular nutritional status assessment (BMI, weight loss) 7
   • Early consideration of gastrostomy for patients with dysphagia
   • Body composition analysis using DEXA or BIA when available 7

3. Symptom management

   • Medications for symptom control (muscle cramps, spasticity, sialorrhea, etc.)
   • Gastrointestinal management for issues like constipation 8
   • Pain management

Early Palliative Care Integration

Palliative care should be integrated from diagnosis, not just at end-of-life 7:

• Early referral to palliative services is recommended to establish relationships before communication becomes limited 7
• Advance care planning discussions should be initiated early
• Caregiver support including counseling and support groups 7

Timing of Interventions

Early diagnosis and treatment initiation are critical 9:

• The diagnostic process should be expedited (currently takes approximately 12 months from symptom onset to diagnosis) 9
• Disease-modifying treatments show better efficacy when started earlier 9
• Early multidisciplinary care results in cost savings and prolonged survival 9

Common Pitfalls to Avoid

1. Delayed treatment initiation

   • Waiting too long to start riluzole or edaravone reduces potential benefits
   • Early referral to specialized ALS centers improves outcomes

2. Inadequate symptom management

   • Failing to address symptoms like dysphagia, respiratory insufficiency, and pain
   • Not considering quality of life alongside disease modification

3. Neglecting nutritional status

   • Weight loss is associated with worse prognosis 7
   • At diagnosis, patients with >5% weight loss compared to usual weight have twice the risk of death 7

4. Delayed palliative care referral

   • Palliative care should not be reserved for end-of-life
   • Only 30% of palliative units in the UK provide ALS care, and only 8% are involved from diagnosis 7

5. Equipment and service delays

   • The rapidly progressive nature of ALS requires expedited approval for equipment and services 7
   • Delays can result in catastrophic safety risks rather than simple inconvenience 7

While there is currently no cure for ALS, the combination of disease-modifying treatments and comprehensive supportive care can significantly impact both survival and quality of life for patients with this devastating disease.