New ALS research results from Oregon State University scientists may offer hope for a new kind of treatment for ALS. The treatment uses a chemical compound called copper-ATSM. The study recently published in the journal of Neurobiology of Disease shows significant results in treating laboratory mice with ALS. The treatment has not been tested yet in humans.
The copper-ATSM treatments were able to extend the life of mice for 20 months, which is nearly a full lifespan for the genetically engineered ALS mice. In contrast, the mice without treatment died within 8-13 days of birth.
The study also showed that progression of the motor neuron disease could be restarted and stopped by withholding or giving the copper-ATSM treatments. The mice seemed to respond within hours of treatment.
Joseph S Beckman, Ph.D with OSU’s Linus Pauling Institute has been studying ALS for 23 years. He patterned this study after promising work being done at the University of Melbourne. He knew that copper plays an important role in ALS, but it must be delivered in the right way.
Copper nutritional supplements cannot be used to treating ALS symptoms. The published study states, “Copper supplements can damage the liver and are generally toxic at high dosages.” But the copper-ATSM compound, used in this case, can prevent copper from being released in most tissues, and only be delivered to to hypoxic tissues, or areas with low oxygen supply. The copper-ATSM can cross the blood-brain barrier and release the copper to cells with damaged mitochondria.
Although copper-ATSM has not been tried as a treatment in ALS patients, it is used as a PET-imaging agent for hypoxic tumors in humans. The copper-ATSM has a low toxicity and can penetrate to the central nervous system within minutes.
If results in humans are similar to the results seem in mice, this would be a huge breakthrough for the ALS community. ALS is a condition that progressively degenerates nerve cells that control all muscle movement. It is always fatal and most patients are told they have 3-5 years to live after diagnosis. Each year 3,000-4,000 people are newly diagnosed with ALS in the U.S.
It typically takes 10 years for a pharmaceutical drug to be developed and ready for patients. The process starts in laboratory test tubes, progresses to testing in animal models such as mice, then goes to human testing. The next step for the copper-ATSM treatments would be to 1) optimize the copper-ATSM dosage in mice, and 2) verify treatment effectiveness in mice, through repeated studies using the same protocol. After these steps the treatment could proceed to human clinical trials.
This copper-ATSM treatment has such promising results for halting the progression of ALS. It will no doubt be watched closely and hopefully by ALS patients and families. .
The full study seen in Neurobiology of Disease is titled: Copper delivery to the CNS by CuATSM effectively treats motor neuron disease in SOCG93A mice co-expressing the Copper-Chaperone-for-SOD.