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3d model of calprotectin protein

New therapy halts progression of Lou Gehrig’s disease in mice

By Dave Stauth

Human Argonaute 1 protein (cyan) bound to let-7 microRNA (magenta)

A team of researchers led by Joseph Beckman, distinguished professor in biochemistry and biophysics, have essentially stopped the progression of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, for nearly two years in one type of mouse model used to study the disease – allowing the mice to approach their normal lifespan.

The findings are some of the most compelling ever produced in the search for a therapy for ALS, a debilitating and fatal disease, according to the scientists. The study was published in the journal Neurobiology of Disease.

"We are shocked at how well this treatment can stop the progression of ALS,” said Beckman, lead author on this study, principal investigator and holder of the Burgess and Elizabeth Jamieson Chair in OSU’s Linus Pauling Institute.

Beckman is also the director of OSU’s Environmental Health Sciences Center, funded by the National Institutes of Health to support research on the role of the environment in causing disease.

Joe Beckman in his lab with colleagues

Joseph Beckman, Distinguished Professor in Biochemistry and Biophysics

In decades of work, no treatment has been discovered for ALS that can do anything but prolong human survival for less than a month. The mouse model used in this study is one that scientists believe may more closely resemble the human reaction to treatment, which consists of a compound called copper-ATSM.

It’s not yet known if humans will have the same response, but researchers are moving as quickly as possible toward human clinical trials, testing first for safety and then efficacy of the new approach.

Copper-ATSM is a known compound that helps deliver copper specifically to cells with damaged mitochondria, and reaches the spinal cord where it’s needed to treat ALS. This compound has low toxicity, easily penetrates the blood-brain barrier, is already used in human medicine at much lower doses for some purposes, and is well tolerated in laboratory animals at far higher levels. Any copper not needed after use of copper-ATSM is quickly flushed out of the body.

Experts caution, however, that this approach is not as simple as taking a nutritional supplement of copper, which can be toxic at even moderate doses. Such supplements would be of no value to people with ALS, they said.

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The study is available online at ScienceDirect.