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ALS-Related Protein Causes Disease in Animal Model, Suggesting Therapy Target beyond Idebenone

By Idebenone Admin July 27, 2019 0 comments

Recent studies by the Amyotrophic Lateral sclerosis (ALS) Association has demonstrated the loss of a closely related ALS protein that caused the degeneration of the motor neurons that are affected by ALS. Published in the Proceedings of the National Academy of Sciences, ALS, AKA Lou Gehrig’s Disease is a neurodegenerative disease that negatively impacts nerve cells in the spine and brain resulting in the loss of control of muscles. There is no cure and the best modern science has been able to treat it is with various mitochondrial management antioxidants such as idebenone.

Aggregates of the TDP-43 protein were found in failing motor neurons in almost all cases of ALS. Though the mechanism is not fully understood, on theory is that the increase of TDP-43 protein in the aggregates lessens the volume of functional protein, and that decrease contributes to the degradation and eventual death of motor neurons.

Zuoshang Xu, M.D., Ph.D., of the University of Massachusetts Medical School in Worcester, partially quieted the TDP-43 gene in rodents chiefly in support cells called astrocytes. Diseased mice developed an advanced neurodegenerative disease that eventually led to paralysis and death. The mitigation of TDP-43 led to variations in the levels of numerous other proteins in the spines of ALS sufferers.

Conclusion, TDP-43 may add to the disease progression and magnify previous findings on the importance of astrocytes in the disease process,” said Lucie Bruijn, Ph.D., MBA, Chief Scientist for The Association. “If more trials confirm the role of loss of TDP-43, therapies that thwart TDP-43 aggregation, or trade its lost function, could be a valuable treatment approach.”

Until such time as that idebenone is the safest most benign protocol for most mitochondrial issues not just related to ALS. A one year Italian study involving 29 patients found significant effects in cardiac issues like left ventricular mass in the idebenone versus the placebo group.


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