S100A10 and SP1 identified as ALS targets

INDICATION: Amyotrophic lateral sclerosis (ALS)

Inhibiting the adaptor protein S100A10 or SP1, a transcription factor that controls S100A10 expression, could treat ALS and injury-induced motor neuron degeneration. In mice with ALS, siRNA against S100A10 or Mithracin plicamycin -- which interferes with SP1's ability to bind DNA -- reduced motor neuron loss and weight loss, improved motor performance, delayed symptom onset and extended survival. Also in the ALS model, neuron-specific S100A10 knockdown or dominant-negative expression of truncated SP1 reduced motor neuron loss. The therapies led to similar effects in a mouse model of injury-induced motor neuron degeneration...