Compound aids in the delivery of copper to cells with damaged mitochondria, specifically in the spinal cord where amyotrophic lateral sclerosis treatment is needed.
Researchers were able to stop the progression of amyotrophic lateral sclerosis (ALS) in mice for almost 2 years in a recent study.
Although it’s not yet known if humans will have the same response, scientists believe that the mouse model used more closely resembles the human reaction to treatment that consists of copper-ATSM.
Copper-ATSM is a compound that aids in the delivery of copper to cells with damaged mitochondria, specifically in the spinal cord where ALS treatment is needed.
This compound is able to easily penetrate the blood-brain barrier and has a low toxicity level. Currently, it is used in medicines for humans at a much lower dose, as well as in lab animals at higher levels. Any copper that is not needed is flushed out of the body.
A copper supplement can be toxic at even moderate doses and has no value for patients with ALS.
The study, published in Neurobiology of Disease, used transgenic mice that have been engineered to carry the human gene copper chaperon for superoxide dismutase (CCS gene). CCS inserts copper into superoxide dismutase (SOD) and the mice that carry these genes die much more quickly without treatment.
The copper helps stabilizes the antioxidant protein SOD, but without its metal co-factors SOD can become toxic, which results in the death of motor neurons.
The results of the current study showed that ALS progression was halted in 1 type of transgenic mouse model, which typically died within 2 weeks without treatment. Some of the mice were able to survive more than 650 days, which is 500 days longer than any prior research that has been done before.
During certain experiments within the study, researchers had the mice start treatment and then withheld it. Those mice starting showing symptoms of ALS within 2 months after treatment stopped and eventually died within a month. Treatment that was continued caused the mice to gain weight and the disease progression stopped.
"We are shocked at how well this treatment can stop the progression of ALS," said lead author Joseph Beckman.
These mice were able to live for another 6 to 12 months.
"We have a solid understanding of why the treatment works in the mice, and we predict it should work in both familial and possibly sporadic human patients," Beckman said. "But we won't know until we try."
Researchers are looking to move as quickly as possible to human trials. Although the may not help patients recover from neuronal loss, it could slow further progression when started after diagnosis.