ALS (Amyotrophic Lateral Sclerosis)

Amyotrophic Lateral Sclerosis (ALS) – Treatment Overview

Introduction
Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig’s disease, is a progressive neurodegenerative disorder characterized by the loss of upper and lower motor neurons. This leads to progressive muscle weakness, spasticity, fasciculations, dysarthria, dysphagia, and eventually respiratory failure. Cognitive and behavioral changes may also occur in some patients (frontotemporal dementia overlap). Although there is currently no cure, treatment focuses on slowing disease progression, managing symptoms, and maintaining quality of life.

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Disease-Modifying Therapy

1. Riluzole – glutamate release inhibitor

• Dose: 50 mg orally twice daily.

• Shown to prolong survival by 2–3 months, especially in patients with bulbar-onset disease.

2. Edaravone – free radical scavenger (antioxidant)

• IV infusion regimen: 60 mg once daily for 14 consecutive days, followed by 14 days off; then 60 mg daily for 10 of 14 days in subsequent cycles.

• Oral suspension: Equivalent dosing now available for easier use.

• May slow functional decline in early-stage ALS.

3. Sodium phenylbutyrate/taurursodiol (AMX0035, Relyvrio*)

• Dose: One sachet orally or via feeding tube twice daily.

• Slows disease progression and may extend survival. (Note: Withdrawn in some regions due to limited efficacy data, but may still be available in others.)

4. Investigational therapies

• Gene therapies, antisense oligonucleotides (e.g., tofersen for SOD1 mutations), and stem cell approaches are under clinical study.

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Symptomatic and Supportive Treatment

1. Spasticity and cramps

• Baclofen: 5–20 mg orally three times daily.

• Tizanidine: 2–4 mg orally three times daily.

• Diazepam may be used in resistant cases.

2. Sialorrhea (excessive drooling)

• Amitriptyline: 25–75 mg/day orally.

• Glycopyrrolate: 1–2 mg orally 2–3 times daily.

• Botulinum toxin injections into salivary glands for refractory cases.

3. Dysphagia and nutrition

• Swallowing assessment and modification of food texture.

• PEG (percutaneous endoscopic gastrostomy) feeding when oral intake becomes inadequate.

4. Respiratory support

• Non-invasive ventilation (NIV, e.g., BiPAP) improves survival and quality of life.

• Tracheostomy with invasive ventilation in advanced stages (based on patient preference).

5. Communication aids

• Speech therapy.

• Augmentative and alternative communication devices (eye-tracking technology, voice banking).

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Multidisciplinary Care

• Neurologists, physiotherapists, occupational therapists, respiratory specialists, speech therapists, dietitians, and psychologists all play crucial roles.

• Palliative care should be introduced early to address symptoms and advance care planning.