An ALS breakthrough announced by scientists was partially funded by money raised through the Ice Bucket Challenge. A team of researchers identified a protein linked to cell death in the brain or spinal cord of patients – providing a target for therapeutic intervention in the future.
Scientists at Baltimore's Johns Hopkins University announced their findings at the 14th International Conference on Endothelin: Physiology, Pathophysiology and Therapeutics in Georgia. The protein Endothelin (ET)-1 is produced by blood vessel cells and a powerful vessel constrictor. It is also produced by astrocytes – brain cells that play a large role in health and disease.
In patients with Amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), progression of the disease is associated with dysfunctional astrocytes. Previous research has shown ET-1 influences several cellular pathways implicated in its progression, and that ET-1 and the receptor it binds to, ET-B, are elevated in ALS sufferers.
ALS is a motor neurone disease where cells of the nervous system die. This leads to muscle weakness, impacting speech, movement, swallowing and breathing. There is no cure and current treatments only slow disease progression by a few months.
In their latest study, researchers looked at whether ET and ET-B are altered in the areas where nerve cells die in ALS. They looked at tissue samples and cells of patients along with a mouse model of ALS. Findings showed higher levels of ET-1 in astrocytes and a higher level of the ET-B receptor in the nerve cells in regions affected by the disease.
"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," researchers said.
ALS made headlines around the globe following the launch of the Ice Bucket Challenge, where people filmed themselves pouring ice over their heads and nominating others to do the same while donating to the ALS Association. Millions was raised as a result and Jonathan Ling, lead author of the study published in the journal Science, said it helped further their research.
He told the New York Times that while the research was already under way, the money helped them to speed up their work and conduct more experiments: "The funding certainly facilitated the results we obtained," he said.