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 . An aqueous formulation for intravenous injection comprising a single active ingredient consisting of trehalose, wherein the formulation has a pH about 4.5 to 7.0 and contains less than 0.75 endotoxin units per ml. 
     
     
         2 . The aqueous formulation of  claim 1 , wherein the trehalose is at 10% (w/v). 
     
     
         3 . The aqueous formulation of  claim 1 , wherein the formulation has an osmolality of about 280-330 mOsm/kg. 
     
     
         4 . A method of alleviating a sign or symptom of oculopharyngeal muscular dystrophy (OPMD) by intravenously administering to a subject in need thereof the aqueous formulation according to  claim 1 . 
     
     
         5 . The method of  claim 4 , wherein the aqueous formulation is administered once weekly. 
     
     
         6 . The method of  claim 5 , where the aqueous formulation is administered at a per administration dose of about 0.5 gram trehalose per kilogram body weight. 
     
     
         7 . The method of  claim 5 , wherein the aqueous formulation is administered at a per administration dose of between 5 to 35 grams trehalose. 
     
     
         8 . The method of  claim 7 , wherein the per administration dose is 8, 15 or 30 grams. 
     
     
         9 . The method of  claim 4 , wherein the administration is completed within less than 120 minutes. 
     
     
         10 . The method of  claim 4 , wherein the rate of administration is such that the maximum endotoxin level is less than 5 EU per kilogram of body weight per hour. 
     
     
         11 . A method for treating or alleviating any one of oculopharengeal muscular dystrophy (OPMD), spinocerebellar ataxia (SCA), Friedreich's ataxia, spinal and bulbar muscular atrophy (SBMA), Huntington's disease, Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), dentatombral-pailidoluyssan atrophy (DRPLA), Pick's disease, Corticobasal degeneration (CBD), Progressive supranuclear palsy (PSP), Frontotemporal dementia, or parkinsonism linked to chromosome 17 (FTDP-17), or at least one symptom associated therewith, in a human subject in need thereof comprising administering to said subject a pharmaceutical formulation comprising trehalose as sole active ingredient. 
     
     
         12 . The method of  claim 11 , wherein the concentration of trehalose in the pharmaceutical formulation is between about 0.1% (w/v) to about 50% (w/v). 
     
     
         13 . The method of  claim 11 , wherein the pharmaceutical formulation has a pH about 4.5 to 7.0 and contains less than 0.75 endotoxin units per ml. 
     
     
         14 . The formulation of  claim 11 , wherein the pharmaceutical formulation has an osmolality of about 280-330 mOsm/kg. 
     
     
         15 . The method of  claim 11 , wherein the administering is once weekly. 
     
     
         16 . The method of  claim 11 , where the administering is at a per administration dose of about 0.5 gram trehalose per kilogram body weight. 
     
     
         17 . The method of  claim 11 , wherein the administering is at a per administration dose of between 5 to 35 grams trehalose. 
     
     
         18 . The method of  claim 11 , wherein the administering is completed within less than 120 minutes. 
     
     
         19 . The method of  claim 11 , wherein the rate of administration is such that the maximum endotoxin level is less than 5 EU per kilogram of body weight per hour. 
     
     
         20 . The method of  claim 11 , wherein the administering is any one of intravenous, intramuscular, or intraperitoneal.