Methods for slowing familial als disease progression

Abstract

Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS.

1 . A method of treating amyotrophic lateral sclerosis (ALS) comprising administering to the cerebrospinal fluid of a subject in need thereof a therapeutically or prophylactically effective amount of a composition comprising:
 a modified oligonucleotide consisting of 12 to 30 linked nucleosides that is specifically hybridizable with SEQ ID NO: 1, and   a pharmaceutically acceptable diluents or carrier, wherein the treatment increases lifespan in the subject.   
     
     
         2 . The method of  claim 1 , wherein administering is intrathecal administration. 
     
     
         3 . The method of  claim 1 , wherein administering is intraventricular administration. 
     
     
         4 . The method of  claim 1 , wherein the modified oligonucleotide consists of 12 to 30 linked nucleosides having a nucleobase sequence comprising an 8-nucleobase portion of SEQ ID NO: 3. 
     
     
         5 . A method of slowing progression of amyotrophic lateral sclerosis (ALS) comprising administering to the cerebrospinal fluid of a subject in need thereof a therapeutically or prophylactically effective amount of a composition comprising:
 a modified oligonucleotide consisting of 12 to 30 linked nucleosides that is specifically hybridizable with SEQ ID NO: 1, and   a pharmaceutically acceptable diluents or carrier, wherein the treatment slows disease progression.   
     
     
         6 . The method of  claim 5 , wherein administering is intrathecal administration. 
     
     
         7 . The method of  claim 5 , wherein administering is intraventricular administration. 
     
     
         8 . The method of  claim 5 , wherein the modified oligonucleotide consists of 12 to 30 linked nucleosides having a nucleobase sequence comprising an 8-nucleobase portion of SEQ ID NO: 3.