Excitotoxicity in the Motor System
20-50% of ALS patients show hyperexcitability in their motor system, where the neurons that control their muscles are overactive. This process leads to a fast progression of the disease in patients, largely due to excess production of calcium and toxic proteins that cannot be cleared. By studying motor neurons generated from ALS patient stem cells, we have discovered that the potassium channel Kv7.2/7.3 is downregulated in the motor system leading to hyperexcitability and excitotoxicity. With further investigation, we identified a drug target in the KV7.2/7.3 ion channel and we are currently developing a drug candidate to treat the motor neuron hyperexcitability characteristic of ALS.