Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose

Abstract

Disclosed is a method of treatment of a disease associated with abnormal protein aggregation comprising parenterally administering pharmaceutical formulations comprising trehalose. Also disclosed is an injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose.

1 - 59 . (canceled) 
     
     
         60 . A method of treating progressive supranuclear palsy (PSP), comprising parenterally administering to a subject a pharmaceutical formulation comprising trehalose as the sole active ingredient;
 wherein the concentration of trehalose in the formulation is between about 0.1% (w/v) to about 50% (w/v);   wherein the formulation has an osmolality of from about 280 to about 330 mOsm/Kg; and   wherein the subject is administered about 10 to about 50 grams of trehalose per week.   
     
     
         61 . The method of  claim 60 , wherein the pharmaceutical formulation is an injectable solution for parenteral administration. 
     
     
         62 . The method of  claim 61 , wherein the parenteral administration is any one of intravenous, intramuscular, or intraperitoneal administration. 
     
     
         63 . The method of  claim 60 , wherein the pharmaceutical formulation has a pH about 4.5 to 7.0. 
     
     
         64 . The method of  claim 60 , wherein the pharmaceutical formulation contains less than 0.75 endotoxin units per mL. 
     
     
         65 . A kit comprising:
 (a) trehalose;   (b) a sterile solution comprising water;   (c) a means for preparing an injectable aqueous solution of the trehalose by mixing (a) with (b);   (d) a means for parenterally administering the injectable solution to a subject; and   (e) instructions for use   
     
     
         66 . The kit of  claim 65 , wherein the sterile solution comprising water is water. 
     
     
         67 . The kit of  claim 65 , further comprising a trehalase inhibitor. 
     
     
         68 . The kit of  claim 65 , wherein the injectable aqueous solution (c) comprises about 10% trehalose (w/v). 
     
     
         69 . A method of treating frontotemporal dementia, comprising parenterally administering to a subject a pharmaceutical formulation comprising trehalose as the sole active ingredient;
 wherein the concentration of trehalose in the formulation is between about 0.1% (w/v) to about 50% (w/v);   wherein the formulation has an osmolality of from about 280 to about 330 mOsm/Kg; and   wherein the subject is administered about 10 to about 50 grams of trehalose per week.   
     
     
         70 . The method of  claim 69 , wherein the pharmaceutical formulation is an injectable solution for parenteral administration. 
     
     
         71 . The method of  claim 70 , wherein the parenteral administration is any one of intravenous, intramuscular, or intraperitoneal administration. 
     
     
         72 . The method of  claim 69 , wherein the pharmaceutical formulation has a pH about 4.5 to 7.0. 
     
     
         73 . The method of  claim 69 , wherein the pharmaceutical formulation contains less than 0.75 endotoxin units per mL. 
     
     
         74 . The method of  claim 60 , wherein the trehalose comprises less than about 0.5% maltotriose (w/w). 
     
     
         75 . The kit of  claim 65 , wherein the trehalose comprises less than about 0.5% maltotriose (w/w). 
     
     
         76 . The method of  claim 69 , wherein the trehalose comprises less than about 0.5% maltotriose (w/w). 
     
     
         77 . The method of  claim 60 , wherein the trehalose comprises less than about 0.5% glucose (w/w). 
     
     
         78 . The kit of  claim 65 , wherein the trehalose comprises less than about 0.5% glucose (w/w). 
     
     
         79 . The method of  claim 69 , wherein the trehalose comprises less than about 0.5% glucose (w/w).