Over 10 years ago, researchers discovered that people with ALS many times had clumps of the protein TDP-43 outside of the nucleus of their brain cells, but they never knew if it was the cause or the result of having ALS.
With the money raised, the scientists were able to do experiments with mice and they made a protein that mimics TDP-43 and put it into nerve cells. When the cells came back to life, this let them know that the protein problem is part of the reason the nervous system slowly dies off in ALS.
Due to the success of the ice bucket challenge, the scientists now have funding to begin clinical trials on humans. This could potentially lead to cure of the disease or potentially slow down the progressive disease. See their announcement below:
