Endothelin 1 Levels Found to be Elevated in ALS Patients: Possible Treatment Avenue

Johns Hopkins University researchers have announced that they will present the latest results of a study that reveals increased levels of endothelin 1 (ET-1) in astrocytes in the brain, along with increased levels of ET-B binding protein (the protein that binds to the ET-1 receptor) are associated with amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease. The results will be presented at the 14th International Conference on Endothelin: Physiology, Pathophysiology and Therapeutics.

Progressive nerve cell death is the hallmark of Lou Gehrig’s disease. It is almost always a fatal disease and represents an ongoing loss of muscle function that eventually progresses to the muscles of the respiratory tract so that ALS patients cannot walk, use their upper extremities or the muscles that control respiration. The patient generally dies from respiratory failure or another respiratory complication. There is no known cure for Lou Gehrig’s disease and the treatments currently available can only prolong the person’s life by a few months at the most.

The recent findings from Johns Hopkins University researchers looked at the levels of ET-1, a tiny protein produced by cells of the blood vessels that causes extreme vasoconstriction, in the astrocytes of the brain. ET-1 has a binding protein called endothelin binding protein, or ET-B, both of which have been found to be elevated in ALS patients. Researchers observed that ET-1 along with ET-B is active and involved in several different pathways underlying ALS. However, the exact manner in which ET-1 impacts the development of Lou Gehrig’s disease is still not understood.

“These experiments demonstrate striking abnormalities in the central nervous system endothelin system, [suggesting that] the endothelin system may represent a largely unexplored and potentially significant target for therapeutic intervention in ALS,” the researchers stated.

Other brain diseases have been successfully managed with ET-1 blockers that currently exist on the market. The researchers believe that it is possible that the same medications, or alternative therapeutics yet to be discovered, may impact Lou Gehrig’s disease treatment as well. To date, no studies have used ET-1 blockers in ALS. However, the medications do exist and the researchers believe that future research should attempt to use ET-1 blocker therapy in the treatment of ALS in order to understand if such an approach could reverse some of the nerve cell death found in these patients.