The ALS Association

ALS Ice Bucket Challenge Progress

First ALS Patient Receives Isis-SOD1RX Antisense Treatment in Phase 1 Clinical Trial

March 5, 2010

Isis Pharmaceuticals, Inc. has initiated a Phase 1 study of ISIS-SOD1Rx 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-SOD1Rx.

ISIS-SOD1Rx 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.

The Phase 1 study of ISIS-SOD1Rx is a placebo-controlled, dose-escalation study designed to assess the safety, tolerability and pharmacokinetic profile of ISIS-SOD1Rx in patients with familial ALS that is caused by mutations within the SOD1 gene. The study consists of four cohorts with eight patients each. In this study, ISIS-SOD1Rx will be administered intrathecally (in the spinal canal) using an external pump to deliver the drug directly into the spinal fluid during a single, 12-hour infusion. The study will be conducted in multiple centers within the United States.

“There is a desperate need for new and more substantive treatments for ALS patients. The mission of the Northeast ALS Consortium is to help advance new therapeutic options for patients with ALS, and I am very pleased to be a co-chair on this important study evaluating ISIS-SOD1 Rx in people with familial ALS,” said Merit Cudkowicz, M.D., Co-Chair of the Northeast ALS Consortium and a Professor of Neurology at the Massachusetts General Hospital of Harvard Medical School.

“It is evident that certain forms of familial ALS are related to mutant forms of SOD1. Therefore, the selective inhibition of SOD1 production could provide a way to improve the outcomes of these patients with ALS,” said Timothy Miller, M.D., Ph.D., Assistant Professor of Neurology at Washington University School of Medicine and Director of the Christopher Wells Hobler Laboratory for ALS Research at the Hope Center for Neurological Disorders. “This is the first study to administer an inhibitor of SOD1 directly into the central nervous system. Having been involved with Isis in the research and now the clinical development of ISIS-SOD1Rx, I am looking forward to co-chairing this study with Dr. Cudkowicz and the Northeast ALS Consortium.”

“ISIS-SOD1Rx is our first antisense drug to enter clinical trials to treat a neurodegenerative disease, and our first antisense drug to be administered directly to the central nervous system. Based on our earlier preclinical studies, we believe that ISIS-SOD1 Rx could offer an effective treatment for patients with familial ALS,” said C. Frank Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals. “This study is the first step in demonstrating the applicability of antisense drugs to treat severe neurodegenerative diseases. We also continue to advance our earlier-stage programs toward human studies.”

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