Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) – Treatment Overview

Introduction
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disorder involving the loss of upper and lower motor neurons. This leads to muscle weakness, spasticity, atrophy, dysarthria, dysphagia, and ultimately respiratory failure. Cognitive and behavioral changes may occur in up to 50% of patients (frontotemporal dementia overlap). While there is no cure, treatment focuses on slowing disease progression, managing symptoms, and improving quality of life.

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Disease-Modifying Therapy

1. Riluzole (glutamate release inhibitor)

• Dose: 50 mg orally twice daily.

• Extends survival by 2–3 months and delays need for tracheostomy or ventilation.

2. Edaravone (free radical scavenger / antioxidant)

• IV regimen: 60 mg daily for 14 days, then 14 days off; subsequent cycles: 60 mg daily for 10 of 14 days.

• Oral suspension: Equivalent efficacy, easier administration.

• Slows functional decline in early-stage ALS.

3. Sodium phenylbutyrate + Taurursodiol (AMX0035 / Relyvrio*)

• Dose: 1 sachet orally or via feeding tube twice daily.

• May slow progression and improve survival; however, its availability is limited in some regions.

4. Tofersen (for SOD1 mutation ALS – antisense oligonucleotide)

• Dose: 100 mg intrathecal injection (3 doses every 14 days, then every 28 days).

• Reduces SOD1 protein levels, FDA-approved for SOD1-mutant ALS.

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Symptomatic Management

1. Spasticity and Cramps

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

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

• Diazepam for refractory spasticity.

2. Sialorrhea (drooling)

• Amitriptyline: 25–75 mg daily.

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

• Botulinum toxin injections into salivary glands for resistant cases.

3. Dysphagia & Nutrition

• Swallowing assessment with modified diet.

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

4. Respiratory Support

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

• Invasive ventilation via tracheostomy: Considered in advanced disease, based on patient preference.

5. Communication Support

• Speech therapy for dysarthria.

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

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Multidisciplinary and Supportive Care

• Team approach including neurologists, physiotherapists, occupational therapists, respiratory specialists, dietitians, psychologists, and palliative care.

• Early advance care planning for end-of-life decisions.

• Physical therapy to prevent contractures; mobility aids for independence.

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Prognosis

• Median survival: 3–5 years from symptom onset (longer in limb-onset vs. bulbar-onset ALS).

• Prognosis improved with multidisciplinary care and early initiation of riluzole, edaravone, and respiratory support.