ALS Breakthroughs

Amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease) is a rapidly progressive motor neuron disease that attacks nerve cells (motor neurons) in the brain and spinal cord leading to paralysis.

In 2007, Tanz scientists developed the first antibody that specifically labels misfolded superoxide dismutase-1 (SOD1). Mutations in SOD1 are the only known cause of ALS. 

The antibody has potential utility for use in biomarker studies, drug discovery and development of immunization strategies for the treatment of ALS caused by mutations in SOD1.

Tanz researchers were also the first to show that there is deregulated expression of alternatively spliced variants of the peripherin protein in ALS, and that these may play a role in the neurodegenerative process.