The anti-epileptic drug ezogabine reduced pathologic excitability of cortical and spinal motor neuron cells that are early signs of clinical dysfunction in people with amyotrophic lateral sclerosis (ALS), according to a study. The study was conducted by the Neurological Clinical Research Institute of Massachusetts General Hospital (MGH).In addition to providing a clearer understanding of motor neuron excitability as an important disease pathway for ALS, the multi-site study, published in JAMA Neurology, involves the first clinical investigation of ALS (also known as Lou Gehrig’s disease) using a drug identified through an induced pluripotent stem cell (iPSC) model. ALS is a progressive neurodegenerative disorder that leads to the death of neurons in the brain and spinal cord that control speech, swallowing and limb movements. Named after the famous baseball player Lou Gehrig, who was diagnosed with the disease in 1939, there are around 20,000 people in the US with ALS, and another 5,000 newly diagnosed cases each year. Currently, there are three approved drugs in the US for treating ALS, each with limited benefit, creating an urgent need for new therapies that could change the course of the fatal disease. The MGH study of ezogabine was not designed to assess the long-term effects of the drug on neurodegenerative disorder, but rather to unravel the biological processes that go awry and identify novel molecular targets for drug intervention.