BREAKING THE ICE WITH ALS

ALS awareness is on the rise thanks to the ubiquitous 'ice bucket challenges' making worldwide headlines. This social media phenomena prompts us here at BioTech Primer to wonder where does the biotech industry stand?

ALS TREATMENTS IN DEVELOPMENT
There is plenty of interest in developing ALS treatments with Biogen Idec (Cambridge, MA) announcing an 'ALS Innovation Hub' last month - an effort to better understand the disease mechanism with the goal of identifying new drug targets. And Prize4Life (Berkeley, CA) is inviting academic and industry contenders to compete to find a treatment for ALS - a 'no-strings attached' $1 million dollars given to the winner. Founded in 2006, Prize4Life's goal is to 'accelerate the discovery of treatments and a cure for ALS.' Registration is still open - could we have a winner from one of the treatments listed below?

ISIS Pharmaceuticals (Carlsbad, CA) announced positive results from a Phase I study of an antisense drug targeting the SOD1 gene last year. The drug is aimed at FALS - approximately 20% of FALS cases are tied to the mutated SOD1 gene.

Neuralstem (Germantown, MD) is currently conducting Phase II studies on a stem cell treatment for ALS. Human spinal cord stem cells are surgically implanted into ALS patients, where the cells differentiate into motor neuron cells – essentially repairing the damaged nerves that are the source of the disease. Phase II results have not yet been announced, but stem cell treatment showed promising results in an animal model of the disease.

GlaxoSmithKline is in Phase II trials with monoclonal antibody Ozanezumab, aimed to maintain existing muscle function in ALS patients with the possibility of slowing the progression of the disease.

Lastly, ALS Therapy Development Institute (ALS TDI) (Cambridge, MA) screens over 25 different therapies targeting ALS, check out their active pipeline

Click here to read more about ALS in today's BioTech Primer WEEKLY