Therapeutic targets
- Functional improvement
- Relief of discomfort
- Life extension
Therapy recommendations
- Causal therapy of ALS is not yet possible.
- Symptomatic therapy:
- Bulbar symptoms (concerning pharyngeal/throat muscles): methanthelinium bromide (anticholinergics); trihexyphenidyl (muscarinic receptor anatgonists); glycopyrronium (parasympatholytics).
- Anxiety: benzodiazepines (e.g., lorazepam).
- Depression (see under depression): preferential use of serotonin reuptake inhibitors (e.g., citalopram); tricyclics (e.g., amitryptiline).
- Hypersalivation (synonyms: sialorrhea, sialorrhea; ptyalism; increased salivation): scopolamine patch (alkaloid); atropine drops (parasympatholytics); amitriptyline (tricyclic antidepressants); botulinum toxin (incobotulinum toxin A).
- Convulsions: magnesium (first-line agent); carbamazepine, gabapentin, phenytoin (antiepileptic agents); diphenhydramine (H1 antihistamines); quinine sulfate (200-400 mg/d; quinoline alkaloids; for nocturnal convulsions).
- Muscle tone elevation: baclofen, tizanidine; slow-onset L-dopa.
- Fasciculations (involuntary movements of very small muscle groups): anticonvulsants (e.g., carbamazepine 2 × 200 mg/d); usually no treatment is required
- Pain: nonsteroidal anti-inflammatory drugs (NSAIDs) and opiates according to WHO staging scheme.
- Spasticity: diazepam (benzodiazepines); baclofen, tizanidine (myotonolytics/antispasmodics).
- Tough mucus: acetylcysteine (ACC; 300-600 mg/d).
- A small life-prolonging effect (on average by 2-3 months) in the early stage shows therapy with the glutamate antagonist riluzole. However, this effect could not be demonstrated in patients older than 75 years and in the advanced stage.
- Delaying the progression of neurodegeneration: antioxidant edaravone (MCI-184, 3-methyl-1-phenyl-2-pyrazolin-5-one; “free radical scavenger”; one-hour infusion therapy).
- Significant reduction in disease progression as measured by change in ALS-FRS-R score.
- Antibiotics are administered for infections.
Therapies in the trial phase
In ALS, which is caused by mutations in the gene encoding superoxide dismutase 1 (SOD1), a Phase I/II trial has seen promising results from treatment with an antisense oligonucleotide that prevents production of a protein responsible for the disease. Whether this has an impact on the progression of the disease is currently being investigated in a phase III trial.
