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Remarkable New Therapy Stops ALS Development In Mice


Robin Andrews

Science & Policy Writer

1009 Remarkable New Therapy Stops ALS Development In Mice
Could this therapy eventually work on people with ALS? crazymedia007/Shutterstock

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig's diesease, is a neurodegenerative disorder that causes the death of neurons within the brain and spinal cord. This condition was thought to be incurable; however, a new study published in the journal Neurobiology of Disease has demonstrated that, using a stabilizing chemical called Copper-ATSM, ALS can be effectively stopped within mice for nearly two years, a substantial portion of their lifespan.

In most cases, the underlying causes of this disorder are unknown; only a fraction of ALS cases can be linked to genetic inheritance. Typified by the disruption of the connections between the nervous system and the body’s muscles, ALS quickly reducers a sufferer’s ability to control their muscles. On average, it causes death within three to four years after its onset.


Previous research has linked the destruction of motor, or movement neurons to a malfunctioning enzyme known as copper-zinc superoxide dismutase, or CuZnSOD. This enzyme chemically alters negatively charged oxygen molecules known as superoxides into either ordinary molecular oxygen or hydrogen peroxide. Superoxides are highly reactive molecules, and can destroy proteins and cause cellular damage if left unconverted by CuZnSOD.

Unfortunately, the gene that codes for CuZnSOD production, SOD1, can mutate with negative consequences. For example, mice with the mutant SOD1 gene have been shown to produce toxic CuZnSODs, as the copper, a key component, cannot be properly integrated within these enzymes.

Without this integration, these enzymes unfold and become harmful to cells. Along with all the reactive, unconverted superoxides, these unfolded enzymes can lead to the death of motor neurons and induce ALS.

The mice lived 500 percent longer than their untreated brethren. anyaivanova/Shutterstock


Compounds known as copper chaperones are used by animals to deliver and integrate copper into CuZnSOD. Oddly, when human copper chaperones are given to mice with the mutant SOD1 gene – in an attempt to make working CuZnSOD – they experience a major copper deficiency in their spinal cord.

According to the authors of this study, this indicates that the CuZnSOD within the mice’s central nervous system could not obtain enough copper, and could not properly mature in order to perform its protective role. Therefore, a way to effectively deliver copper to the spinal cord, and allowing it to integrate into the CuZnSOD, was required.

Copper-ATSM is a compound that is known to chaperone copper specifically into the central nervous system. It has low toxicity, easily penetrates the blood-brain barrier, and is tolerated by both humans and animals.

This was injected into mice possessing the mutant SOD1 gene, and within weeks the mice began to form CuZnSOD within their spinal cord. Their ALS symptoms were dramatically reduced, and the mice gained control over their muscles. The treatment was so remarkably effective that these mice lived on average for an extra 20 months, a 500 percent increase in lifespan.


“We are shocked at how well this treatment can stop the progression of ALS,” said Joseph Beckman, lead author on this study, and a professor of biochemistry and biophysics in the College of Science at Oregon State University, in a statement. “We want people to understand that we are moving to human trials as quickly as we can.”

The researchers note that Copper-ATSM’s effects in ALS sufferers cannot be replicated by taking copper supplements, which even in moderate concentrations can be highly toxic within the human body.


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