The effect of available treatment for any of the motor neuron diseases is modest. A small number of drugs, however, have been developed: riluzole, edaravone, and masitinib. Supportive measures, however, are exceedingly important. In initial office visits, it has been our practice to give the patient some idea of the seriousness of the condition; but in early discussions we avoid the devastating statement that ALS is invariably fatal. Typically, patients and family members will ask explicitly about these matters in subsequent visits; such data as are appropriate to the patient’s circumstances and character can be conveyed at that time, usually with the caveat that any individual may outlive the standard survival statistics.
The antiglutamate agent riluzole, when given orally, was shown by Bensimon and colleagues to slow the progression of ALS and improve survival in patients with disease of bulbar onset; it prolonged survival by 3 months. Antioxidants have slowed the clinical progress of ALS in limited and unconfirmed trials. Masitinib is a tyrosine kinase inhibitor that has been tried in a number of degenerative disorders and is under investigation in ALS. Some of these claims have been confirmed in several followup studies, although again, the benefit has been marginal. Several additional agents are reported to have been effective in genetic models of ALS. Among the long list of agents that have claimed, but ultimately failed, to help ALS are guanidine hydrochloride, injections of cobra venom, gangliosides, interferons, high-dose intravenous cyclophosphamide, and thyrotropin-releasing hormone.
With regard to symptomatic treatment, an attempt can be made to reduce spasticity with medications, such as baclofen or tizanidine, or by subarachnoid infusions of baclofen via an implanted lumbar pump. Initial intrathecal test doses are given to predict a response to the pump infusions of baclofen, but this test may fail; consequently, in severe cases it may be advisable to proceed with a constant infusion for several days. Some degree of improved comfort from a reduction in the extreme rigidity is usually the most that can be expected. Partial relief from spasticity may also be afforded by the use of benzodiazepines or sometimes dantrolene. These approaches are most suitable for cases of primary lateral sclerosis, which can be expected to progress slowly and for a long period. The pseudobulbar syndrome can be ameliorated with dextromethorphan-quinidine compounds.
At all stages of ALS, physical therapy is useful in maintaining mobility, but overwork of the muscles leading to fatigue and cramps should be avoided. Physical therapy is invaluable, for example, for avoiding contractures of the fingers and shoulders. Occupational therapy is likewise helpful, particularly assessments of the patient’s function in the home.