New Therapy Shows Promise in Treating ALS in Mice


News Brief by Akash Pillai


            Oregon State University scientists published promising findings in the journal Neurobiology of Disease in late January regarding a potential treatment for Amyotrophic Lateral Sclerosis (ALS), also commonly referred to as Lou Gehrig’s Disease. This debilitating neurological disorder is caused by the death of motor neurons in the brain and spinal cord. ALS’s progression is believed to be linked to the loss of function of superoxide dismutase, an important enzyme which becomes toxic in motor neurons in the absence of proper regulation by copper co-factors. This results in a loss of control over voluntary muscles, which in humans typically results in a greatly decreased lifespan and death in 3-4 years on average. Thus far, there have been very little advances made in ALS therapies, with most treatments only increasing a person’s lifespan by about one month at best. However, scientists developed a therapy based on a compound known as Copper-ATSM, which delivers the deficient copper into the cell. In an ALS mouse model, the scientists found that the therapy prevented the disease’s progression for two years. This allowed the ALS mice to survive far longer than their usual two weeks, and much closer to their normal lifespan. The longer the mice were kept on the therapy, the longer it took for the mice to die from their ALS when taken off the medication. This strongly suggested that the therapy was successful in halting ALS’s progression, although the specific mechanisms by which this occurred are not well understood. Nevertheless, future experiments may be able to develop new therapies for ALS based on these new findings.

Jared R. Williams, Emiliano Trias, Pamela R. Beilby, Nathan I. Lopez, Edwin M. Labut, C.

Samuel Bradford, Blaine R. Roberts, Erin J. McAllum, Peter J. Crouch, Timothy W. Rhoads, Cliff Pereira, Marjatta Son, Jeffrey L. Elliott, Maria Clara Franco, Alvaro G. Estévez, Luis Barbeito, Joseph S. Beckman. Copper delivery to the CNS by CuATSM effectively treats motor neuron disease in SODG93A mice co-expressing the copper-chaperone-for-SOD. Neurobiology of Disease, 2016; DOI: 10.1016/j.nbd.2016.01.020