What are the treatment options for Amyotrophic Lateral Sclerosis (ALS)?

Treatment Options for Amyotrophic Lateral Sclerosis (ALS)

The treatment of ALS requires a comprehensive approach including FDA-approved medications like riluzole, nutritional support, and management of dysphagia, as these interventions have been shown to improve survival and quality of life.

Pharmacological Treatments

• Riluzole is the primary FDA-approved medication for ALS treatment, administered at 50 mg twice daily, and has been shown to extend survival by approximately 2-3 months by decreasing the risk of death or tracheostomy by 35% compared to placebo 1, 2
• Riluzole should be taken at least 1 hour before or 2 hours after meals to ensure proper absorption 1
• Regular monitoring of liver enzymes is mandatory as riluzole can cause elevation of serum aminotransferases in 10-15% of patients 1, 3
• The most common adverse effects include asthenia (18%), nausea (15%), dizziness, decreased lung function, and abdominal pain 1, 3
• Riluzole is contraindicated in patients with baseline elevations of serum aminotransferases greater than 5 times the upper limit of normal and in those with a history of severe hypersensitivity reactions to the drug 1

Nutritional Management

For Patients with Dysphagia

• For patients with muscular fatigue and prolonged mealtimes: Fractionate and enrich meals with energy or deficient nutrients; if weight loss progresses, oral nutritional supplementation is recommended 4
• For moderate dysphagia: Dietetic counseling to adapt food texture (soft, semisolid, or semiliquid) to facilitate swallowing and prevent aspiration 4
• Instrumental assessment of swallowing function through videofluoroscopy (VFS), fiberoptic endoscopic evaluation of swallowing (FEES), or VFS-manometry can guide texture modifications 4
• Postural maneuvers, particularly chin-tuck posture, should be implemented to protect the airway during swallowing 4
• For patients with delayed swallowing, thicker liquids and semisolid foods with high water content (like jellified water) are better alternatives to thin liquids 4

Advanced Nutritional Support

• Enteral nutrition (tube feeding) is preferred over parenteral nutrition for ALS patients requiring nutritional therapy 4
• Parenteral nutrition should only be considered in acute settings when enteral nutrition is contraindicated or not feasible 4
• Home parenteral nutrition is generally not indicated in ALS patients due to high complication rates (3.11 per 1000 catheter days) 4

Physical Activity Recommendations

• Low to moderate physical activity should be advised as long as it doesn't worsen the patient's condition, as some evidence suggests that endurance and resistance exercises may slow disease progression and improve functionality and quality of life 4
• Avoid excessive physical exertion that could lead to fatigue and worsen symptoms 4

Management of Complications

High White Blood Cell Count

• In ALS patients presenting with very high white blood cell counts, monitor for signs of leukostasis (unexplained hypoxia, neurological symptoms, renal failure) 4
• Prompt initiation of therapy is essential in cases of symptomatic leukostasis 4

Infection Prevention

• Implement antibacterial, antifungal, and antiviral prophylaxis as infections are a major cause of death in ALS patients 4
• Consider prophylactic use of G-CSF (granulocyte colony-stimulating factor) to shorten neutropenia duration and improve adherence to chemotherapy schedules 4

Special Considerations

• For female patients of reproductive age, continuous administration of progestational agents is preferred over combined oral contraception to suppress menorrhagia during thrombocytopenic periods 4
• Fertility preservation options should be discussed with patients of reproductive age before starting treatment 4

Monitoring and Follow-up

• Regular assessment of liver function is essential for patients on riluzole 1, 3
• Monitor for neutropenia, as riluzole may rarely cause this condition 3
• Discontinue riluzole if there is evidence of interstitial lung disease 1

Pitfalls and Caveats

• Riluzole should not be used in patients with pre-existing liver disease or elevated transaminases 1, 3
• The benefit of riluzole is modest (2-3 months survival extension), and patients should have realistic expectations about treatment outcomes 2, 5
• Dysphagia management techniques that work for other conditions may not be effective in ALS due to the specific pathophysiology of muscle atrophy and fatigue 4
• Despite limited evidence for efficacy, nutritional interventions are crucial for maintaining quality of life and preventing complications 4