Treatment for familial amyotrophic lateral sclerosis/motor neuron disease

Benatar M, Kurent J, Moore DH

Summary

Treatment for familial amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) and Lou Gehrig's disease, is a rare disease in which degeneration of motor nerves leads to progressive weakness and wasting of muscles. For the most part, the cause of ALS is unknown. In a small proportion of cases there is a family history of ALS/MND and in an even smaller proportion, the disease is known to result from a change in a gene known as SOD1. An understanding of the genetic basis for one familial form of ALS/MND has permitted the construction of an animal model of ALS/MND (the SOD1 mouse) that has been used extensively to study potential therapeutic agents for the human disease. None of the drugs found to be effective in the mouse have translated into therapeutic benefits for humans with ALS/MND. There are several possible explanations for this finding, one of which is that successful treatments in the mouse model (based on the genetic form of ALS/MND) are more likely to be effective in patients with the familial form of the disease. In an effort to begin to address this question, this review was undertaken in order to ask whether or not people with the familial form of the disease respond differently to treatment than patients with the sporadic (or non-familial) form of ALS/MND.

We identified all randomized controlled trials (RCTs) in ALS/MND and wrote to the authors to request the data needed to complete this review. Although many more studies were eligible for inclusion, only four authors were willing to share the data from their individual RCTs. Based on the analyses of these data, we find no evidence to support a statistically significant difference in the response to treatment between patients with the familial and sporadic forms of ALS/MND.