Antisense Oligonucleotides (ASO) for ALS targeting C9 and SOD1 genes

The UCSD ALS Clinical Research Group is proud to be one of the first sites in the US to be selected to participate in this innovative concept that has drastically changed the gene therapy landscape of antisense technology. This new potential treatment targets the first and second most common cause of inherited ALS, mutations in the C9 and SOD1 genes respectively. Developed by Biogen and Ionis Pharmaceuticals, in collaboration with UC San Diego ALS Clinical Research Program, SOD1 antisense oligonucleotide (ASO) therapy for ALS has initiated Phase III clinical trial stage to further test the safety and efficacy of BIIB67 (now called tofersen) in those living with ALS that carry SOD1 gene mutations.  At the same time, the first treatment targeting C9orf72 gene mutation in ALS is ongoing. UC San Diego ALS Clinical Research Program has successfully enrolled patients in this clinical trial in cohorts 1, 2 and 3. Cohort 4 is scheduled to begin enrollment during the first quarter of year 2020. We are excited to be a part of this ground breaking concept.

Small molecule BIIB100 (XPO1 inhibitor) Therapy

UC San Diego ALS Clinical Research group is also an active site in clinical trials evaluating the small molecule BIIB100. BIIB100 is being evaluated to see if it can slow the progression of sporadic ALS by normalizing the nuclear traffic of proteins and RNA. Dysfunction in nucleo-cytoplasmic transport (the process by which proteins and RNA molecules move in and out of the nucleus) appears to play a role in ALS. UC San Diego ALS Clinical Trials program has successfully enrolled subjects for cohorts 1 and 2 in this new and exciting small molecule ALS therapy. Cohort 3 is scheduled to open for enrollment in early 2020. We are excited to be a part of this exciting clinical development.