Treatment of proteinopathies
The present disclosure provides technologies relating to lysosomal activation. The disclosure provides several strategies for increasing level and/or activity of lysosomal enzyme, and furthermore demonstrates the surprising applicability of such strategies in the treatment and/or prophylaxis of certain proteinopathies. Among other things, the present invention provides methods and compositions for the treatment and/or prophylaxis of proteinopathies other than lysosomal storage diseases through lysosomal activation. In particular, the present disclosure provides methods and compositions for the treatment and/or prophylaxis of neurodegenerative proteinopathies, and in particular those associated with accumulation of α-synuclein. The present disclosure specifically provides methods and compositions for the treatment and/or prophylaxis of Parkinson's disease.
1. A method of treating a neurodegenerative proteinopathic disease in a subject in need thereof, the method comprising:
administering to the subject a therapeutically effective amount of the compound NCGC00188758 having the structure of Formula I:
(Formula I), or a salt thereof, thereby to treat the neurodegenerative proteinopathic disease in the subject.
2. The method of claim 1 , wherein the disease is Parkinson's disease.