Scientists at the University of North Carolina School of Medicine have been successful in identifying the structure of the neuronal protein clumps associated with ALS, which is also known as Lou Gehrig’s disease. Their research, which was published online, has given the scientific community the evidence it needs to conclude that these toxic clumps play a significant part in the development of this degenerative disease. It also gives them hope that they are one step nearer to finding ways to prevent the formation of these clumps, and thus halt the progression of ALS.
So far, it has proved difficult to find successful treatments for ALS and other neurodegenerative diseases, as scientists have been unable to pinpoint the causes. This has proved to be one of the biggest set-backs for researchers looking to come up with suitable drugs and treatments. However, as this new study has identified the structure of the toxic clumps which are responsible for the death of the motor neurons, it will now be possible to develop new drugs to combat this.
A study based around patients with variations in a specific protein
The study was based around a group of patients who exhibited variations in a specific protein known as SOD1, which has been shown to form potentially lethal clumps. This particular protein is very unstable, and the researchers believe that it is this instability that makes it so toxic. Due to its instability it has proved to be extremely difficult to study in the past, therefore for this study they team used a combination of both computational modelling and experiments using live cells.
The researchers saw that the clumps formed in groups of three, and that these clumps were able to kill lab-grown motor neuron cells. By being able to see what these clumps looked like and how they formed, the researchers now hope to be able to develop drugs that will either break up the clumps, or stop their formation before they cause the fatal damage. They also plan to look more closely at what actually holds the clumps together. Furthermore, it’s hoped that this new information will not only help researchers to develop new treatments for ALS, but that it may also provide an insight into ways in which to combat other neurodegenerative diseases such as Alzheimer’s and Parkinson’s.
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