According to The Times of Israel, a group of researchers believe they have found the origin of what could lead to a treatment for amyotrophic lateral sclerosis. Given the debilitating effect of ALS on its patients, this could be a major step forward.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that progressively affects nerve cells in the spinal cord and brain. Because ALS causes nerve cells to wither away, muscles in those affected eventually weaken and many experience a loss of voluntary movement and control. Due to the nature of the disease, ALS is progressive and eventually fatal as those who have it ultimately lose the ability to breathe. Unfortunately, there is currently not a cure for ALS, but there are a few treatments available that focus on relieving the patient. To learn more about the neurological disorder, click here.
Given that there are no substantial treatments for ALS, the news that scientists from Tel Aviv University may have found one is big.
“While we are not claiming we have found the cure for ALS, we have certainly moved the field forward,” said leader of the study Dr. Eran Perlson. “The findings may be the basis of a future drug,” although “it will take time.”
The research, which can be found in Journal of Neuroscience, revealed that people with ALS have muscles that secrete toxic factors which ultimately cause damage. This is groundbreaking because it is the first time that research has emphasized the importance of these toxic molecules in ALS.
Perlson noted that people with ALS showed higher levels of toxicity and also an increase in the number of receptors. So how does this lead to a solution?
The cherry on top is that researchers also found that high toxicity occurred when levels of microRNA (miRs) were reduced. These are small molecules that help with the translation of proteins. In addition, microRNAs are incredibly important for many other cellular functions and processes.
Perlson believes that miR can be used to eventually treat ALS. The team has already seen results in mice—the lab work has shown that symptoms of ALS are improved by manipulating miR-126-5p (the specific type of miR that is reduced in ALS models).
When the research team injected mice with a viral vector carrying miR126-5p, they found that the injected mice showed substantial improvement in muscle degeneration, neuromuscular functionality and walking ability. This injection greatly slowed the neuron degeneration process.
Dr. Perlson is very hopeful about the research. “We demonstrated in lab work and on mouse models that we can successfully ameliorate ALS symptoms using this miR as a potential drug,” he said. Additionally, “We further demonstrated that muscle tissue — not only motor neurons — are undoubtedly involved in the progression of ALS.”
Perlson and his team have even bigger plans to continue ALS research into the non-muscular tissues damaged by the disease.
