Compositions and methods for treating an aggregation disease or disorder

Abstract

The present invention alleviates a sign or symptom of an aggregation disease or disorder by administering an aqueous formulation comprising trehalose.

What is claimed is: 
     
         1 .- 20 . (canceled) 
     
     
         21 . A method for treating a Parkinson's disease or Alzheimer's disease, or alleviating at least one symptom associated therewith, in a human subject in need thereof, comprising intravenously administering to said subject a pharmaceutical formulation comprising trehalose as sole active ingredient. 
     
     
         22 . The method of  claim 21 , wherein the formulation has a pH of about 4.5 to about 7.0. 
     
     
         23 . The method of  claim 21 , wherein the formulation contains less than 0.75 endotoxin units per mL. 
     
     
         24 . The method of  claim 21 , wherein the administering is at a per administration dose of between 5 to 50 grams trehalose. 
     
     
         25 . The method of  claim 21 , wherein the administration is completed within less than 120 minutes. 
     
     
         26 . The method of  claim 21 , wherein the concentration of trehalose in the pharmaceutical formulation is between about 0.1% (w/v) to about 50% (w/v). 
     
     
         27 . The formulation of  claim 21 , wherein the pharmaceutical formulation has an osmolality of about 280-330 mOsm/kg. 
     
     
         28 . The method of  claim 21 , wherein the administering is once weekly. 
     
     
         29 . The method of  claim 21 , wherein the administering is at a per administration dose of about 0.5 gram trehalose per kilogram body weight of the subject. 
     
     
         30 . The method of  claim 24 , wherein the administering is at a per administration dose of between 5 to 35 grams trehalose. 
     
     
         31 . The method of  claim 21 , wherein the rate of administration is such that the maximum endotoxin level is less than 5 endotoxin units per kilogram of body weight of the subject per hour. 
     
     
         32 . The method of  claim 26 , wherein the concentration of trehalose in the pharmaceutical formulation is between about 0.1% (w/v) to about 20% (w/v). 
     
     
         33 . The method of  claim 32 , wherein the concentration of trehalose in the pharmaceutical formulation is between about 5% (w/v) to about 10% (w/v). 
     
     
         34 . The method of  claim 21 , wherein the administering is completed within less than 90 minutes. 
     
     
         35 . The method of  claim 21 , wherein the disease or disorder is Parkinson's disease. 
     
     
         36 . The method of  claim 21 , wherein the disease or disorder is Alzheimer's disease. 
     
     
         37 . A method for inhibiting aggregation of poly (A) binding protein nuclear 1 protein (PABPN 1), in a human subject in need thereof, comprising intravenously administering to said subject a pharmaceutical formulation comprising trehalose as sole active ingredient. 
     
     
         38 . The method of  claim 37 , wherein the formulation has a pH of about 4.5 to about 7.0 and the formulation contains less than 0.75 endotoxin units per mL. 
     
     
         39 . The method of  claim 37 , wherein the pharmaceutical formulation has an osmolality of about 280-330 mOsm/kg. 
     
     
         40 . The method of  claim 37 , wherein the administering is at a per administration dose of between 5 to 50 grams trehalose.