First ALS Patient Receives Isis-SOD1_RX 

Antisense Treatment in Phase 1 Clinical Trial

Isis Pharmaceuticals, Inc. has initiated a Phase 1 study of ISIS-SOD1_Rx in patients with an inherited, aggressive form of Lou Gehrig’s disease also known as familial amyotrophic lateral sclerosis (ALS).  Approximately 20 percent of all familial ALS cases are caused by a mutant form of superoxide dismutase, or SOD1.  The ALS Association and the Muscular Dystrophy Association are providing funding for the development of ISIS-SOD1_Rx.

ISIS-SOD1_Rx is an antisense drug designed to inhibit the production of SOD1.  Antisense techniques are used to deactivate disease-causing or undesirable genes so that they cannot produce harmful or unwanted proteins.

"This therapy will be the first treatment for ALS that is specifically aimed at the target, SOD1, known to cause ALS. The development of new treatments for ALS is an extremely challenging and costly process.  The ALS Association has been pleased to co-fund this study and partner with Isis on this extremely promising treatment approach. It is only through the support of our generous donors that this type of advancement is made possible," commented Lucie Bruijn, Ph.D., Chief Scientist, The ALS Association.