The 'Smoking Gun' in ALS

A spate of reports buttresses the case for TDP-43 as a key player in amyotrophic lateral sclerosis. But even though the protein is mislocalized in a majority of patients, drug developers are holding off on targeting TDP-43 until more is known about its function. Instead, most companies are sticking with better-studied targets such as neuronal receptors and another mutated protein, superoxide dismutase 1.

Amyotrophic lateral sclerosis (ALS) is a debilitating neurodegenerative disorder that causes loss of sensation, motor difficulties and paralysis. ALS is usually sporadic, but a small number of cases have previously been linked to hereditary mutations in superoxide dismutase 1 (SOD1)...