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news.Isis Pharmaceuticals, a company focused on discovery and development of RNA-based drugs, has started a Phase 1 study of ISIS-SOD1Rx in patients with an inherited, aggressive form of Lou Gehrig's disease, known as familial amyotrophic lateral sclerosis (ALS).
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Isis claimed that ISIS-SOD1Rx is an anti-sense drug designed to restrict the production of SOD1. The ALS Association and the Muscular Dystrophy Association are providing funding for the development of the drug.
The Phase 1 study of ISIS-SOD1Rx is a placebo-controlled, dose-escalation study designed to evaluate 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 the study, ISIS-SOD1Rx will be administered intrathecally 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 US.
Merit Cudkowicz, co-chair of the Northeast ALS Consortium, said: “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-SOD1Rx in people with familial ALS.”
Timothy Miller, director of the Christopher Wells Hobler Laboratory for ALS Research at the Hope Center for Neurological Disorders, said: “It is evident that certain cases 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.
“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.”
C Frank Bennett, senior vice president of research at Isis Pharmaceuticals, said: “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 into the central nervous system. Based on our earlier preclinical studies, we believe that ISIS-SOD1Rx could offer an effective treatment for patients with familial ALS.
“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.”