Restoration of autophagy pathways
Autophagy, a process in which the cell selectively recycles parts of itself as a way to dispose of waste and regain important building blocks, is malfunctioning in a subpopulation of ALS patients, leading to cell death in the patients’ motor neurons. This malfunction can be caused by a mutation in one of the genes on the autophagy pathway. We are targeting a protein early on the autophagy pathway, hoping its reduction will lead to normalization of autophagy levels and reduce the buildup of toxic waste. Our discovery work is showing a possible drug candidate to be a potentially safe, targeted therapeutic to correct this faulty autophagy pathway and restore neuronal function.