About 5,000 people each year will be diagnosed with amyotrophic lateral sclerosis (ALS), a neurodegenerative condition commonly known as Lou Gehrig's Disease in which the nerves stop sending impulses to the muscles, causing paralysis. The prognosis isn't favorable, with an average life expectancy of 3-5 years after diagnosis, though a small percentage live another 20 years or more.
Treatment options have historically been mostly palliative, but there could be a brighter future on the horizon. A study published in Neurobiology of Disease has described a new treatment that was able to stop the progression of ALS in mice, allowing them to live to their normal life expectancy.
The secret behind this treatment is copper-ATSM, a compound that delivers copper to damaged cells. The normal survival rate of a mouse genetically engineered to have ALS-like symptoms is about 2 weeks, but using this treatment, the mice were able to live a full 2 years.This is far and away the longest that mice with this condition have survived. If the mice stopped receiving treatment, however, the progression of the disease would begin again.
"We are shocked at how well this treatment can stop the progression of ALS," lead author Joseph Beckman admitted in a news release.
Copper is essential for a number of processes that keep the body healthy, including formation of red blood cells, embryonic development, and cellular functions that keep nerves healthy. When an enzyme called copper, zinc superoxide dismutase becomes mutated, however, it can cause ALS.
The answer to ALS does not lie in copper supplements, as it can become toxic at certain levels. The beauty of copper-ATSM is that it targets cells in the spinal cord with damaged mitochondria, the powerhouse of the cell. The treatment works exactly where it needs to and the rest is simply filtered out with other waste products in the body.
It's important to note that this treatment didn't reverse the effects of the disease. It merely stopped the disease from getting any worse. Currently, there are no ways to repair the damage once it's been done.
"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 stated, which suggests that it could work for those both with and without a genetic predisposition to developing ALS. "But we won't know until we try."
The researchers hope that gaining a better understanding of this mechanism could also help with other neurodegenerative diseases, including Parkinson's disease.
There are a number of steps that need to be taken before the researchers can move forward with their findings and bring the copper-ATSM to human clinical trials. But given the poor prognosis for individuals with the disease, the researchers are highly motivated to get a treatment to those who are suffering.
"We want people to understand that we are moving to human trials as quickly as we can," Beckman concluded. "In humans who develop ALS, the average time from onset to death is only three to four years."
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