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A new treatment for ALS could be on the way, as researchers say they have stopped the progression of the disease in mice for two years. The treatment allowed the mice to live for 650 days – 500 days longer than any other previous treatment has managed.
The results from Oregon State University, published in Neurobiology of Disease, were unexpectedly promising, say the scientists. The treatment, known as Copper-ATSM, was dissolved in the solvent dimethyl sulfoxide and applied directly to the skin of the mice on the back of their necks. It was absorbed within ten minutes, and scientists say the difference was noticeable almost immediately.
"We are shocked at how well this treatment can stop the progression of ALS," said Joseph Beckman, lead author of the study. "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."
ALS, or Lou Gehrig's disease, is a fatal disease whereby motor neurons in the spinal cord begin to deteriorate. Roughly 10% of ALS patients live for longer than 10 years after diagnosis, and current treatments can only extend their lives by just a few months. While the researchers are aware this new treatment is not designed to be a cure, they believe it can extend the lives of ALS patients greatly compared to currently available therapies.
Usually, the ALS-positive mice used in the trial would have died in less than two weeks without any treatment, as a copper deficiency – linked to ALS – would have damaged the mitochondria in their development.
After Copper-ATSM was used, the mice survived for much longer; at least 650 days.
To test the treatment further, the scientists actually stopped the treatment at some points in the study. Within two months, the mice began to show symptoms again, and died not long after. Similarly, if they were put back on the treatment, they gained weight and the disease appeared to recede once more.
Copper-ATSM is a compound that is used to deliver copper to cells that have damaged mitochondria – the part of the cell that changes glucose to energy. In this case, it can be sent directly to the spinal cord of ALS sufferers. It can then selectively hand out copper to these damaged organelles, and ultimately reduce the damage the disease causes.
The compound is already currently being used for cancer treatments in humans, albeit in much smaller doses that what the scientists are suggesting for ALS. That said, it is not very toxic, so they believe the positives for ALS patients would definitely outweigh the negatives.
"In humans who develop ALS, the average time from onset to death is only three to four years," said Beckman. "We want people to understand that we are moving to human trials as quickly as we can."
Before that happens though, Lucie Bruijin, Chief Scientist at the ALS Association, says the treatment has a long way to go, and it will be tested on a small group of dogs, too.
"Currently The ALS Association is supporting the investigator to develop biomarkers using this treatment approach, so that we can more readily determine whether this treatment approach is beneficial in human clinical trials," she told IBTimes UK. "In addition, the investigator is planning studies in a canine model."
Researchers believe Copper-ATSM could prevent death in ALS patients for years, as opposed to months. Its official effect on humans is as yet unknown, but the treatment is being rushed to human trials right now, after the trials in mice were successful.
"I believe it is important to pursue these exciting findings as they represent an opportunity to develop meaningful therapies for people living with the disease." said Bruijin. "However, it is unclear whether this approach will impact all forms of ALS, or whether these significant findings in the mice will translate to humans."