A new candidate therapy for ALS based on copper delivery to the brain and spinal cord extends lifespan and improves locomotor function in ALS mice. Mutations in copper, zinc superoxide dismutase (SOD1) are thought to cause familial ALS through a toxic gain-of-function mechanism, although precisely how SOD1 mutations lead to selective motor neuron death is not fully understood. A recent report published June 4 online in Journal of Neuroscience and led by Peter Crouch and colleagues at the University of Melbourne, Australia and Joseph Beckman at Oregon State University, demonstrates that restoring the copper content of mutant SOD1 by oral delivery of a an organic molecular containing copper, called Cu-ATSM, improved locomotor function and extended lifespan by 26% in a mutant SOD1 mouse model of ALS. Intriguingly, the treated mice express higher levels of misfolded mutant SOD1 than untreated mice, challenging the accepted view that the abundance of mutant SOD1 determines disease severity. Cu-ATSM has also shown therapeutic promise in Parkinson’s disease (see April 2011 Conference News). Click here to read more.
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