A recent study suggests that a known copper compound may hold promise as the basis for therapy to address amyotrophic lateral sclerosis (ALS). According to a news release issued by Oregon State University (OSU), researchers from Australia, the US (Oregon), and the United Kingdom demonstrated in laboratory animal tests that oral intake of the compound significantly extended the lifespan and improved the locomotor function of transgenic mice genetically engineered to develop ALS.
Joseph Beckman, distinguished professor of biochemistry and biophysics in the OSU College of Science, states in the release, “We believe that with further improvements, and following necessary human clinical trails for safety and efficacy, this could provide a valuable therapy for ALS and perhaps Parkinson’s disease.”
The release notes that in ALS, when SOD1 is lacking its metal co-factors, it “unfolds” and becomes toxic, ultimately killing motor neurons. Copper and zinc are key in stabilizing this protein, researchers say, and can help it remain folded more than 200 years.
Beckman explains that damage from ALS primarily occurs in the spinal cord; one of the most difficult places in the body to absorb copper, “Copper itself is necessary but it 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. Otherwise, the compound keeps copper inert. This is a safe way to deliver a micronutrient like copper exactly where it is needed,” Beckman adds.
Restoring a proper balance of copper into the brain and spinal cord, scientists say, may help stabilize the superoxide dismutase in its mature form, while improving the function of mitochondria. The results suggest that the technique has extended the lifespan of affected mice by 26%, and with continued research the scientists state they hope to achieve additional extension.
The compound, copper (ATSM), has reportedly been studied for use in some cancer treatments.
Blaine Roberts, lead study author, research fellow at the University of Melbourne, explains that the treatment “increased the amount of mutant SOD, and by accepted dogma this means the animals should get worse. But in every case, they got a lot better. This is because we’re making a targeted delivery of copper just to the cells that need it.”
Roberts adds that the study opens up previously neglected avenues for new disease therapies for ALS and other neurodegenerative diseases.
Source: Oregon State University