Research Aims to Understand the Mechanism of Neuron Death in Amyotrophic Lateral Sclerosis

According to a story from, the process that causes the neurons responsible for voluntary muscle movement in amyotrophic lateral sclerosis (also known as Lou Gehrig’s disease) to die is not well understood. An international team of scientists from the US, Germany, the Netherlands, and Italy are hoping to change that. Data from their latest study suggests that the team is starting to make significant progress.

About Amyotrophic Lateral Sclerosis (ALS)

Amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease, is a rare, degenerative disease that causes the death of nerve cells associated with the voluntary muscles. Little is known about the origins of amyotrophic lateral sclerosis, with no definitive cause in about 95 percent of cases. The remaining five percent appear to inherit the disease from their parents. Symptoms initially include loss of coordination, muscle weakness and atrophy, muscle stiffness and cramping, and trouble speaking, breathing, or swallowing. These symptoms worsen steadily over time; most patients die because of respiratory complications. Treatment is mostly symptomatic and the medication riluzole can prolong life. Life expectancy after diagnosis ranges from two to four years, but some patients can survive for substantially longer. To learn more about amyotrophic lateral sclerosis, click here.

About The Research

One factor that the team is observing is changes to the behavior of the FUS protein, which plays a role in RNA binding. Mutations of this protein has previously been linked to aggressive variants of amyotrophic lateral sclerosis. However, the team is finding that interactions of RNA binding proteins may be more central to the progression of the disease than has been understood in the past.

Impaired FUS interactions can completely throw off the balance of RNA binding protein activity, which contributes to the death of neurons. This theory was tested when the scientists used drugs to trigger the degradation of proteins. This process was able to slow the pace of neuron death.

These findings could mean that drugs that induce or increase the autophagy process (protein degradation) could be a viable therapeutic approach to treating amyotrophic lateral sclerosis. 

The team is using induced pluripotent stem cells (essentially stem cells from patients that have been reprogrammed) to conduct research not only on amyotrophic lateral sclerosis, but also other degenerative illnesses, such Parkinson’s disease.


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