In a new study published by the Neurobiology of Disease journal, researchers working with mice claim to have halted the progression of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, a fatal neurodegenerative disorder.
Named after the famous New York Yankee baseballer who developed the disease, ALS has baffled researchers for decades.
“We are shocked at how well this treatment can stop the progression of ALS,” study author Joseph Beckman, a professor of biochemistry at Oregon State University, said in a statement.
ALS is known to be a result of the death and degeneration of motor neurons in the spine. The breakdown has been linked to mutations in an enzyme called copper, zinc superoxide dismutase.
Showing great promise
The study team used a mouse model thought to closely mimic the human reaction to treatment with a compound called copper-ATSM. The compound is a known to help deliver copper expressly to cells with impaired mitochondria, and reaches the spinal cord where it can treat ALS. Copper-ATSM has low toxicity, readily permeates the blood-brain barrier, is already utilized in human medicine, at much lower doses, and is well-tolerated in laboratory animals at far greater amounts. Any copper not required after use of copper-ATSM is rapidly eliminated from the body, the researchers said.
Copper, zinc superoxide dismutase. (Credit: Oregon State University)
Using the treatment, scientists stopped the advancement of ALS in one kind of transgenic mouse model, which normally would die within two weeks without care. Many of these mice have survived for more than 650 days, 500 days longer than any previous study has been able to accomplish.
In some trials, the regimen was begun, and then withheld. In this scenario the mice started to show ALS symptoms within two months after medical care was stopped, and would die within a month. But if medical care was resumed, the mice gained weight, advancement of the disease once again was halted, and the mice lived another 6 to 12 months longer.
“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.”
The researchers noted a potential treatment is not likely to lead to recovery from neuronal loss already brought on by ALS. However, it could hamper further disease development when started after a diagnosis. It could also possibly treat carriers of SOD mutant genes that induce ALS.
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