Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disease, with an average life expectancy of 2–5 years after diagnosis and no available cures.

We have discovered a mechanism to reduce insoluble TDP-43 aggregates, a toxic protein found in all ALS cases. Our in vivo studies show that reducing NLK levels decreases toxic TDP-43, improves motor function, and extends survival in ALS mouse models.

Using an in silico screen, we have identified potential NLK inhibitors and are seeking funds to advance these candidates in our small molecule development pipeline.