The Linus Pauling Institute has some very exciting research to report on potential therapies for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. Dr. Joseph Beckman from the LPI with colleagues in Australia and the United Kingdom have shown that a copper compound, called CuATSM, delayed the onset of symptoms and extended lifespan in mouse models of ALS.

Put simply, this is groundbreaking.

Currently, treatment options for ALS are few and not very effective. This disease hits a very specific part of the body: motor neurons – cells that control every movement. However, motor neurons lie in the protected environment of the spinal cord, making ALS extremely difficult to treat.

 Another key to the disease is thought to be superoxide dismutase 1 (SOD1). This antioxidant enzyme normally removes harmful oxygen radicals in the body – at least when it’s working properly.

When SOD1 is lacking one of its metal atoms (it has one copper and one zinc atom), it “unfolds” and starts causing damage. Instead of helping to protect motor neurons, it becomes toxic.

Laboratory animals with ALS express mutant forms of SOD1 – mutations that have been found in humans with the disease. These mutant forms of SOD1 easily lose one of their metal ions, giving animals the same progressive paralysis and wasting seen in ALS patients.

One approach to restore SOD1 function is to try forcing the metals back into the enzyme. This is where CuATSM comes into play. Since copper is carefully controlled in the body for a variety of reasons, including potential toxicity, most copper therapies are very difficult to use. CuATSM, however, is capable of delivering copper atoms in hard-to-reach places in the body.

As described by Dr. Beckman and his colleagues in the The Journal of Neuroscience, CuATSM fed to laboratory animals with ALS actually delivered copper to motor neurons of the spinal cord. Although the animals wound up producing more mutant SOD1, the extra copper allowed SOD1 to stay in its properly folded, protective form.

Best of all, the CuATSM delayed the progression of ALS in these animals, and they lived longer than animals that did not receive it.

So for the first time, a promising therapy may exist for ALS – and since CuATSM has already been used in humans, it’s not expected to have serious negative effects.

As Dr. Beckman said, “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.”

While much more work still needs to be done, these findings are already being heralded as a breakthrough.

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