Oregon State University researchers were able to stop the progression of amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) for nearly two years in a specific type of mouse model. The treatment allowed the animals to live for 650 days – 500 days longer than any other previous treatment has managed. The study, “Copper delivery to the CNS by CuATSM effectively treats motor neuron disease in SODG93A mice co-expressing the copper-chaperone-for-SOD,” was published in the journal Neurobiology of Disease.
Despite years of research efforts, there is no cure for ALS, and available treatments prolong survival for less than a month. ALS is caused by the deterioration and death of in the spinal cord, which has been associated to mutations in copper, zinc superoxide dismutase, which is essential to life but can become toxic when damaged.
Researchers used an ALS mouse model to deliver a novel treatment called copper-ATSM, which helps deliver copper to cells with damaged mitochondria. This strategy allows the drug to penetrate the spinal cord of patients with ALS. It can then selectively deliver copper to the damaged organelles, and ultimately reduce the damage caused by the disease.
The treatment should not be confused with taking copper supplements, which can be toxic at even moderate doses. Scientists say copper supplements do not help people with ALS.
After treatment with the new drug, researchers were able to stop ALS progression in one type of ALS mouse model that survived more than 650 days, compared to two weeks without treatment.
After treatment had been stopped for two months, the animals again began exhibiting symptoms of ALS. But once treatment was restarted, mice gained weight, the progression of the disease was halted, and mice survived for another six to 12 months.