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In Australia, amyotrophic lateral sclerosis (ALS) — or Lou Gehrig’s disease — kills roughly two people every day.
Using animal models, an international team including researchers from the University of Melbourne and the Florey Institute, have shown that swallowing a cheap copper compound can improve movement and significantly extend life.
In humans, no therapy for ALS has ever been discovered that could extend lifespan more than a few additional months.
Dr Peter Crouch, from the Department of Pathology at the University of Melbourne, said the new approach has great potential.
“We believe that with further improvements, and following necessary human clinical trials for safety and efficacy, this could provide a valuable new therapy for ALS and perhaps Parkinson’s disease also,” he said.
The compound has previously been studied for use in some cancer treatments, and is inexpensive to produce.
ALS was first identified as a progressive and fatal neurodegenerative disease in the late 1800s and gained international recognition in 1939 when it was diagnosed in American baseball legend Lou Gehrig.
It’s known to be caused by motor neurons in the spinal cord deteriorating and dying, and has been traced to mutations in copper, zinc superoxide dismutase, or SOD1. When SOD1 is lacking its metal co-factors, it “unfolds” and becomes toxic, leading to the death of motor neurons.
Dr Crouch said ALS occurs in a sensitive part of the human body, making direct treatments difficult.
“The damage from ALS is happening primarily in the spinal cord and that’s also one of the most difficult places in the body to absorb copper,” Dr Crouch said.
“Copper itself can be toxic, so its levels are tightly controlled in the body. The therapy we’re working toward delivers copper selectively into the cells in the spinal cord that actually need it."
“This is a safe way to deliver a micronutrient like copper exactly where it is needed,” he said.
By restoring a proper balance of copper into the brain and spinal cord, scientists believe they are stabilising the superoxide dismutase in its mature form.
“In this case, the result was just the opposite of what one might have expected,” said Blaine Roberts, lead author on the study and a research fellow at the Florey Institute of Neuroscience and Mental Health.
“The treatment increased the amount of mutant SOD, and by accepted procedures, this means the animals should get worse,” he said.
“But in this case, they got a lot better. This is because we’re making a targeted delivery of copper just to the cells that need it."
“This study opens up a previously neglected avenue for new disease therapies, for ALS and other neurodegenerative disease,” Roberts said.
The research has been published in the current edition of the Journal of Neuroscience.