Compositions and methods for treating gaucher disease

Abstract

Methods and compositions for treating Gaucher disease are described.

1 . A method of treating a subject with Gaucher disease, the method comprising
 administering a glucocerebrosidase enzyme replacement therapy by intravenous infusion to the subject over a period of less than 2 hours, to thereby treat the subject.   
     
     
         2 . The method of  claim 1 , wherein the glucocerebrosidase enzyme replacement therapy is administered over a period of 90 minutes or less. 
     
     
         3 . The method of  claim 1 , wherein the glucocerebrosidase enzyme replacement therapy is administered over a period of 60 minutes or less. 
     
     
         4 . The method of  claim 1 , further comprising administering a second glucocerebrosidase enzyme replacement therapy by intravenous infusion to the subject over a period of less than 2 hours. 
     
     
         5 . The method of  claim 1 , wherein the glucocerebrosidase enzyme replacement therapy is selected from the group consisting of: velaglucerase, imiglucerase and uplyso. 
     
     
         6 . The method of  claim 1 , wherein the glucocerebrosidase enzyme replacement therapy is administered at a dose of 15 to 60 U/kg. 
     
     
         7 . A method for identifying a subject as suitable for treatment with a glucocerebrosidase enzyme replacement therapy, comprising:
 determining if neutralizing antibodies to a glucocerebrosidase enzyme replacement therapy are present in a sample from the subject to obtain a measured value of antibody to the therapy, wherein the subject is currently being administered a first glucocerebrosidase enzyme replacement therapy or has previously received a first glucocerebrosidase enzyme replacement therapy, and   comparing the measured value of antibody to the therapy to a standard,   wherein if the measured antibody value is greater than the standard, the subject is identified as having antibodies to the glucocerebrosidase enzyme replacement therapy and as a candidate for glucocerebrosidase enzyme replacement therapy with velaglucerase, and if the measured antibody value is not greater than the standard, identifying the subject as a candidate for glucocerebrosidase enzyme replacement therapy with the first glucocerebrosidase enzyme replacement therapy or an alternative glucocerebrosidase enzyme replacement therapy.   
     
     
         8 . The method of  claim 7 , wherein the first glucocerebrosidase enzyme replacement therapy is imiglucerase or uplyso. 
     
     
         9 . The method of  claim 7 , wherein the sample is a blood or serum sample. 
     
     
         10 . The method of  claim 9 , wherein the blood or serum sample has been modified. 
     
     
         11 . The method of  claim 10 , wherein the sample has been contacted with an analytical reagent or substrate. 
     
     
         12 . The method of  claim 10 , wherein the sample is a concentrated portion of a blood or serum sample. 
     
     
         13 . The method of  claim 7 , wherein the subject is identified as a candidate for glucocerebrosidase enzyme replacement therapy with velaglucerase, and the method further comprises administering velaglucerase to the subject. 
     
     
         14 . A method for treating a subject with Gaucher disease that is currently taking or has previously received a glucocerebrosidase enzyme replacement therapy, comprising:
 selecting a subject on the basis that the subject has tested positive for the production of antibodies to the therapy the subject is currently taking or has previously taken for Gaucher disease, and   administering velaglucerase to the subject.   
     
     
         15 . The method of  claim 14 , wherein the subject tested positive for the production of IgE antibodies to the therapy the subject is currently taking or has previously taken for Gaucher disease. 
     
     
         16 . The method of  claim 14 , wherein the subject tested positive for the production of IgM antibodies to the therapy the subject is currently taking or has previously taken for Gaucher disease. 
     
     
         17 . The method of  claim 14 , wherein the subject tested positive for the production of IgG antibodies to the therapy the subject is currently taking or has previously taken for Gaucher disease. 
     
     
         18 . The method of  claim 14 , wherein the therapy the subject is currently taking or has previously taken for Gaucher disease is imiglucerase. 
     
     
         19 . The method of  claim 14 , wherein the therapy the subject is currently taking or has previously taken for Gaucher disease is uplyso. 
     
     
         20 . The method of  claim 14 , wherein velaglucerase is administered at a dose of 15 to 60 U/kg. 
     
     
         21 . The method of  claim 14 , wherein the velaglucerase is administered to the subject by intravenous infusion over 90 minutes or less. 
     
     
         22 . The method of  claim 1 , further comprising administering an oral therapy comprising a compound that reduces glucosylceramide. 
     
     
         23 . The method of  claim 1 , further comprising administering a pharmacological chaperone molecule.