Biogen and Isis Pharmaceuticals have announced the initiation of a phase I/II clinical trial of ISIS-SOD1Rx (BIIB067), an antisense oligonucleotide (ASO) therapy targeting superoxide dismutase (SOD1). ASOs recognize and target specific RNAs for destruction (see Jan 2013 news, Oct 2013 news), ultimately reducing expression of their protein products. ISIS-SOD1Rx is specifically designed to inhibit production of mutant SOD1, which underlies approximately 20% of familial ALS cases and confers toxicity through a gain-of-function mechanism. The ASOs to be tested in this trial are a second generation, more potent version of ASOs previously tested and shown to be safe in phase I trials (see May 2013 news). The current trial will test not only safety, but also treatment effect on SOD1 protein levels in the cerebrospinal fluid, as a measure of drug activity. The study, entitled “Single and Multiple Dose Study of BIIB067 (Isis-SOD1Rx) in Adults With Amyotrophic Lateral Sclerosis (ALS)” will be conducted in two phases, the first of which will enroll both SOD1-ALS patients and patients without SOD1 mutations.
Read more here and here.
antisense disease-als SOD1 topic-clinical topic-randd