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Ezogabine Treatment Decreases Motor Neuron Excitability in ALS Patients

In This Article

  • Brian Wainger, MD, PhD, presented findings of a completed phase 2 clinical trial at the Motor Neuron Disease Association annual meeting
  • Ezogabine activates the KCNQ family of potassium channel subunits, Kv7.2 through 5, reducing the excitability of neurons that can lead to seizures
  • Researchers used magnetic and electrophysiological techniques to evaluate the effects of ezogabine on motor neuron excitability
  • They found that the treatment reduced the excitability of motor neurons

Brian Wainger, MD, PhD, a physician-scientist at the Sean M. Healey and AMG Center for ALS, presented the findings of completed phase 2 clinical trial of ezogabine for treatment of amyotrophic lateral sclerosis (ALS) at the annual meeting of the Motor Neuron Disease Association in December 2018.

At present, there are only three drugs approved to treat ALS in the United States. These drugs have limited effects, and more therapies are needed to lengthen lifespan. Better understanding the disease at a molecular level and identifying new targets for intervention are crucial to advancing treatment.

Ezogabine activates Kv7.2-Kv7.5 potassium channels, which reduces neuronal excitability in neurons that can cause seizures. Researchers with the Neurological Clinical Research Institute (NCRI) at sites of the Northeast ALS Consortium wanted to understand if the "hyperexcitability" of motor neurons may play a role in ALS.

They used transcranial magnetic stimulation and threshold tracking nerve conduction studies on 65 ALS patients to evaluate the effects of ezogabine on upper and lower neuron excitability and found that the treatment reduced the excitability of motor neurons.

Though the study cannot assess clinical outcomes, it does provide more knowledge about neuron excitability and understanding about an important disease pathway.

Learn more about the Healey Center for ALS

Learn more about the Wainger Lab

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The Healey Center at Massachusetts General Hospital is a worldwide leader in ALS research that speeds new therapies to patients.


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